Feature

Fantine Giap, MD, sat across from a 21-year-old with a rare sarcoma at the base of her skull. 

Despite the large tumor, nestled in a sensitive area, the Boston-based radiation oncologist could envision a bright future for her patient. 

She and the other members of the patient’s care team had an impressive cancer-fighting arsenal at her fingertips. The team had recommended surgery, followed by proton therapy — a sophisticated tool able to deliver concentrated, razor-focused radiation to the once apple-sized growth, while sparing the fragile brain stem, optic nerve, and spinal cord. 

Surgery went as planned. But as the days and weeks wore on and insurance prior authorization for the proton therapy never came, the tumor roared back, leading to more surgeries and more complications. Ultimately, the young woman needed a tracheostomy and a feeding tube. 

By the time insurance said yes, more than 1 year from her initial visit, the future the team had envisioned seemed out of reach. 

“Unfortunately for this patient, it went from a potentially curable situation to a likely not curable situation,” recalled Dr. Giap, a clinician at Massachusetts General Hospital and instructor at Harvard Medical School, Boston. “I wanted to cry every day that she waited.’’ 

While a stark example, such insurance delays are not uncommon, according to new research published in JAMA Network Open.

The study of 206 denials in radiation oncology concluded that more than two-thirds were ultimately approved on appeal without changes, but often these approvals came only after costly delays that potentially compromised patient care.

Other studies have found that number to be even higher, with more than 86% of prior authorization requests ultimately approved with few changes.

‘’It gives you the idea that this entire process might be a little futile — that it’s just wasting people’s time,’’ said Fumiko Chino, MD, coauthor on the JAMA study and now an assistant professor in radiation oncology at MD Anderson Cancer Center in Houston. ‘’The problem is cancer doesn’t wait for bureaucracy.’’
 

Barriers at Every Step

As Dr. Chino and her study coauthors explained, advancements like intensity-modulated radiation therapy and stereotactic radiosurgery have allowed a new generation of specialists to treat previously untreatable cancers in ways that maximize tumor-killing power while minimizing collateral damage. But these tools require sophisticated planning, imaging, simulations and execution — all of which are subject to increased insurance scrutiny.

‘’We face barriers pretty much every step of the way for every patient,’’ said Dr. Chino.

To investigate how such barriers impact care, Dr. Chino and colleagues at Memorial Sloan Kettering Cancer Center — where she worked until July — looked at 206 cases in which payers denied prior authorization for radiation therapy from November 1, 2021 to December 8, 2022. 

The team found that 62% were ultimately approved without any change to technique or dose, while 28% were authorized, but with lower doses or less sophisticated techniques. Four people, however, never got authorization at all — three abandoned treatment altogether, and one sought treatment at another institution.

Treatment delays ranged from 1 day to 49 days. Eighty-three patients died.

Would some of them have lived if it weren’t for prior authorization?

Dr. Chino cannot say for sure, but did note that certain cancers, like cervical cancer, can grow so quickly that every day of delayed treatment makes them harder to control. 

Patients with metastatic or late-stage cancers are often denied more aggressive treatments by insurers who, in essence, “assume that they are going to die from their disease anyway,” Dr. Chino said. 

She views this as tragically shortsighted.

‘’There’s actually a strong body of evidence to show that if you treat even metastatic stage IV diseases aggressively, you can prolong not just quality of life but also quantity,’’ she said. 

In cases where the cancer is more localized and insurance mandates lower doses or cheaper techniques, the consequences can be equally heartbreaking.

‘’It’s like saying instead of taking an extra-strength Tylenol you can only have a baby aspirin,’’ she said. ‘’Their pain is less likely to be controlled, their disease is less likely to be controlled, and they are more likely to need retreatment.’’

Prior authorization delays can also significantly stress patients at the most vulnerable point of their lives.

In another recent study, Dr. Chino found that 69% of patients with cancer reported prior authorization-related delays in care, with one-third waiting a month or longer. One in five never got the care their doctors recommended, and 20% reported spending more than 11 hours on the phone haggling with their insurance companies. 

Most patients rated the process as ‘’bad’’ or ‘’horrible,’’ and said it fueled anxiety.

Such delays can be hard on clinicians and the healthcare system too. 

One 2022 study found that a typical academic radiation oncology practice spent about a half-million dollars per year seeking insurance preauthorization. Nationally, that number exceeds $40 million.

Then there is the burnout factor. 

Dr. Giap, an early-career physician who specializes in rare, aggressive sarcomas, works at an institution that helped pioneer proton therapy. She says it pains her to tell a desperate patient, like the 21-year-old, who has traveled to her from out of state that they have to wait. 

‘’Knowing that the majority of the cases are ultimately approved and that this wait is often unnecessary makes it even tougher,’’ she said.

Dr. Chino, a breast cancer specialist, has taken to warning patients before the alarming insurance letter arrives in the mail that their insurance may delay authorizing their care. But she tells patients that she will do everything she can to fight for them and develops a back-up plan to pivot to quickly, if needed.

‘’No one goes into medicine to spend their time talking to insurance companies,’’ said Dr. Chino.

The national trade group, America’s Health Insurance Plans (AHIP), did not return repeated requests for an interview for this story. But their official position, as stated on their website, is that “prior authorization is one of many tools health insurance providers use to promote safe, timely, evidence-based, affordable, and efficient care.”

Both Dr. Giap and Dr. Chino believe that prior authorization was developed with good intentions: to save healthcare costs and rein in treatments that don’t necessarily benefit patients. 

But, in their specialty, the burden has proliferated to a point that Dr. Chino characterizes as ‘’unconscionable.’’

She believes that policy changes like the proposed Improving Seniors’ Timely Access to Care Act — which would require real-time decisions for procedures that are routinely approved — could go a long way in improving patient care.

Meanwhile, Dr. Giap said, more research and professional guidelines are necessary to bolster insurance company confidence in newer technologies, particularly for rare cancers.

Her patient ultimately got her proton therapy and is ‘’doing relatively well, all things considered.’’

But not all the stories end like this.

Dr. Chino will never forget a patient with a cancer growing so rapidly she could see it protruding through her chest wall. She called for an urgent PET scan to see where else in the body the cancer might be brewing and rushed the planning process for radiation therapy, both of which faced prior authorization barriers. That scan — which ultimately showed the cancer had spread — was delayed for months.*

If the team had had those imaging results upfront, she said, they would have recommended a completely different course of treatment.

And her patient might be alive today.

‘’Unfortunately,” Dr. Chino said, “the people with the very worst prior authorization stories aren’t here anymore to tell you about them.”

*Correction,  10/4/24: An earlier version of this article erroneously stated that Dr. Chino called for surgery for her patient. She actually called for a PET scan and an urgent radiation start.

A version of this article first appeared on Medscape.com.

Fantine Giap, MD, sat across from a 21-year-old with a rare sarcoma at the base of her skull. 

Despite the large tumor, nestled in a sensitive area, the Boston-based radiation oncologist could envision a bright future for her patient. 

She and the other members of the patient’s care team had an impressive cancer-fighting arsenal at her fingertips. The team had recommended surgery, followed by proton therapy — a sophisticated tool able to deliver concentrated, razor-focused radiation to the once apple-sized growth, while sparing the fragile brain stem, optic nerve, and spinal cord. 

Surgery went as planned. But as the days and weeks wore on and insurance prior authorization for the proton therapy never came, the tumor roared back, leading to more surgeries and more complications. Ultimately, the young woman needed a tracheostomy and a feeding tube. 

By the time insurance said yes, more than 1 year from her initial visit, the future the team had envisioned seemed out of reach. 

“Unfortunately for this patient, it went from a potentially curable situation to a likely not curable situation,” recalled Dr. Giap, a clinician at Massachusetts General Hospital and instructor at Harvard Medical School, Boston. “I wanted to cry every day that she waited.’’ 

While a stark example, such insurance delays are not uncommon, according to new research published in JAMA Network Open.

The study of 206 denials in radiation oncology concluded that more than two-thirds were ultimately approved on appeal without changes, but often these approvals came only after costly delays that potentially compromised patient care.

Other studies have found that number to be even higher, with more than 86% of prior authorization requests ultimately approved with few changes.

‘’It gives you the idea that this entire process might be a little futile — that it’s just wasting people’s time,’’ said Fumiko Chino, MD, coauthor on the JAMA study and now an assistant professor in radiation oncology at MD Anderson Cancer Center in Houston. ‘’The problem is cancer doesn’t wait for bureaucracy.’’
 

Barriers at Every Step

As Dr. Chino and her study coauthors explained, advancements like intensity-modulated radiation therapy and stereotactic radiosurgery have allowed a new generation of specialists to treat previously untreatable cancers in ways that maximize tumor-killing power while minimizing collateral damage. But these tools require sophisticated planning, imaging, simulations and execution — all of which are subject to increased insurance scrutiny.

‘’We face barriers pretty much every step of the way for every patient,’’ said Dr. Chino.

To investigate how such barriers impact care, Dr. Chino and colleagues at Memorial Sloan Kettering Cancer Center — where she worked until July — looked at 206 cases in which payers denied prior authorization for radiation therapy from November 1, 2021 to December 8, 2022. 

The team found that 62% were ultimately approved without any change to technique or dose, while 28% were authorized, but with lower doses or less sophisticated techniques. Four people, however, never got authorization at all — three abandoned treatment altogether, and one sought treatment at another institution.

Treatment delays ranged from 1 day to 49 days. Eighty-three patients died.

Would some of them have lived if it weren’t for prior authorization?

Dr. Chino cannot say for sure, but did note that certain cancers, like cervical cancer, can grow so quickly that every day of delayed treatment makes them harder to control. 

Patients with metastatic or late-stage cancers are often denied more aggressive treatments by insurers who, in essence, “assume that they are going to die from their disease anyway,” Dr. Chino said. 

She views this as tragically shortsighted.

‘’There’s actually a strong body of evidence to show that if you treat even metastatic stage IV diseases aggressively, you can prolong not just quality of life but also quantity,’’ she said. 

In cases where the cancer is more localized and insurance mandates lower doses or cheaper techniques, the consequences can be equally heartbreaking.

‘’It’s like saying instead of taking an extra-strength Tylenol you can only have a baby aspirin,’’ she said. ‘’Their pain is less likely to be controlled, their disease is less likely to be controlled, and they are more likely to need retreatment.’’

Prior authorization delays can also significantly stress patients at the most vulnerable point of their lives.

In another recent study, Dr. Chino found that 69% of patients with cancer reported prior authorization-related delays in care, with one-third waiting a month or longer. One in five never got the care their doctors recommended, and 20% reported spending more than 11 hours on the phone haggling with their insurance companies. 

Most patients rated the process as ‘’bad’’ or ‘’horrible,’’ and said it fueled anxiety.

Such delays can be hard on clinicians and the healthcare system too. 

One 2022 study found that a typical academic radiation oncology practice spent about a half-million dollars per year seeking insurance preauthorization. Nationally, that number exceeds $40 million.

Then there is the burnout factor. 

Dr. Giap, an early-career physician who specializes in rare, aggressive sarcomas, works at an institution that helped pioneer proton therapy. She says it pains her to tell a desperate patient, like the 21-year-old, who has traveled to her from out of state that they have to wait. 

‘’Knowing that the majority of the cases are ultimately approved and that this wait is often unnecessary makes it even tougher,’’ she said.

Dr. Chino, a breast cancer specialist, has taken to warning patients before the alarming insurance letter arrives in the mail that their insurance may delay authorizing their care. But she tells patients that she will do everything she can to fight for them and develops a back-up plan to pivot to quickly, if needed.

‘’No one goes into medicine to spend their time talking to insurance companies,’’ said Dr. Chino.

The national trade group, America’s Health Insurance Plans (AHIP), did not return repeated requests for an interview for this story. But their official position, as stated on their website, is that “prior authorization is one of many tools health insurance providers use to promote safe, timely, evidence-based, affordable, and efficient care.”

Both Dr. Giap and Dr. Chino believe that prior authorization was developed with good intentions: to save healthcare costs and rein in treatments that don’t necessarily benefit patients. 

But, in their specialty, the burden has proliferated to a point that Dr. Chino characterizes as ‘’unconscionable.’’

She believes that policy changes like the proposed Improving Seniors’ Timely Access to Care Act — which would require real-time decisions for procedures that are routinely approved — could go a long way in improving patient care.

Meanwhile, Dr. Giap said, more research and professional guidelines are necessary to bolster insurance company confidence in newer technologies, particularly for rare cancers.

Her patient ultimately got her proton therapy and is ‘’doing relatively well, all things considered.’’

But not all the stories end like this.

Dr. Chino will never forget a patient with a cancer growing so rapidly she could see it protruding through her chest wall. She called for an urgent PET scan to see where else in the body the cancer might be brewing and rushed the planning process for radiation therapy, both of which faced prior authorization barriers. That scan — which ultimately showed the cancer had spread — was delayed for months.*

If the team had had those imaging results upfront, she said, they would have recommended a completely different course of treatment.

And her patient might be alive today.

‘’Unfortunately,” Dr. Chino said, “the people with the very worst prior authorization stories aren’t here anymore to tell you about them.”

*Correction,  10/4/24: An earlier version of this article erroneously stated that Dr. Chino called for surgery for her patient. She actually called for a PET scan and an urgent radiation start.

A version of this article first appeared on Medscape.com.

Fantine Giap, MD, sat across from a 21-year-old with a rare sarcoma at the base of her skull. 

Despite the large tumor, nestled in a sensitive area, the Boston-based radiation oncologist could envision a bright future for her patient. 

She and the other members of the patient’s care team had an impressive cancer-fighting arsenal at her fingertips. The team had recommended surgery, followed by proton therapy — a sophisticated tool able to deliver concentrated, razor-focused radiation to the once apple-sized growth, while sparing the fragile brain stem, optic nerve, and spinal cord. 

Surgery went as planned. But as the days and weeks wore on and insurance prior authorization for the proton therapy never came, the tumor roared back, leading to more surgeries and more complications. Ultimately, the young woman needed a tracheostomy and a feeding tube. 

By the time insurance said yes, more than 1 year from her initial visit, the future the team had envisioned seemed out of reach. 

“Unfortunately for this patient, it went from a potentially curable situation to a likely not curable situation,” recalled Dr. Giap, a clinician at Massachusetts General Hospital and instructor at Harvard Medical School, Boston. “I wanted to cry every day that she waited.’’ 

While a stark example, such insurance delays are not uncommon, according to new research published in JAMA Network Open.

The study of 206 denials in radiation oncology concluded that more than two-thirds were ultimately approved on appeal without changes, but often these approvals came only after costly delays that potentially compromised patient care.

Other studies have found that number to be even higher, with more than 86% of prior authorization requests ultimately approved with few changes.

‘’It gives you the idea that this entire process might be a little futile — that it’s just wasting people’s time,’’ said Fumiko Chino, MD, coauthor on the JAMA study and now an assistant professor in radiation oncology at MD Anderson Cancer Center in Houston. ‘’The problem is cancer doesn’t wait for bureaucracy.’’
 

Barriers at Every Step

As Dr. Chino and her study coauthors explained, advancements like intensity-modulated radiation therapy and stereotactic radiosurgery have allowed a new generation of specialists to treat previously untreatable cancers in ways that maximize tumor-killing power while minimizing collateral damage. But these tools require sophisticated planning, imaging, simulations and execution — all of which are subject to increased insurance scrutiny.

‘’We face barriers pretty much every step of the way for every patient,’’ said Dr. Chino.

To investigate how such barriers impact care, Dr. Chino and colleagues at Memorial Sloan Kettering Cancer Center — where she worked until July — looked at 206 cases in which payers denied prior authorization for radiation therapy from November 1, 2021 to December 8, 2022. 

The team found that 62% were ultimately approved without any change to technique or dose, while 28% were authorized, but with lower doses or less sophisticated techniques. Four people, however, never got authorization at all — three abandoned treatment altogether, and one sought treatment at another institution.

Treatment delays ranged from 1 day to 49 days. Eighty-three patients died.

Would some of them have lived if it weren’t for prior authorization?

Dr. Chino cannot say for sure, but did note that certain cancers, like cervical cancer, can grow so quickly that every day of delayed treatment makes them harder to control. 

Patients with metastatic or late-stage cancers are often denied more aggressive treatments by insurers who, in essence, “assume that they are going to die from their disease anyway,” Dr. Chino said. 

She views this as tragically shortsighted.

‘’There’s actually a strong body of evidence to show that if you treat even metastatic stage IV diseases aggressively, you can prolong not just quality of life but also quantity,’’ she said. 

In cases where the cancer is more localized and insurance mandates lower doses or cheaper techniques, the consequences can be equally heartbreaking.

‘’It’s like saying instead of taking an extra-strength Tylenol you can only have a baby aspirin,’’ she said. ‘’Their pain is less likely to be controlled, their disease is less likely to be controlled, and they are more likely to need retreatment.’’

Prior authorization delays can also significantly stress patients at the most vulnerable point of their lives.

In another recent study, Dr. Chino found that 69% of patients with cancer reported prior authorization-related delays in care, with one-third waiting a month or longer. One in five never got the care their doctors recommended, and 20% reported spending more than 11 hours on the phone haggling with their insurance companies. 

Most patients rated the process as ‘’bad’’ or ‘’horrible,’’ and said it fueled anxiety.

Such delays can be hard on clinicians and the healthcare system too. 

One 2022 study found that a typical academic radiation oncology practice spent about a half-million dollars per year seeking insurance preauthorization. Nationally, that number exceeds $40 million.

Then there is the burnout factor. 

Dr. Giap, an early-career physician who specializes in rare, aggressive sarcomas, works at an institution that helped pioneer proton therapy. She says it pains her to tell a desperate patient, like the 21-year-old, who has traveled to her from out of state that they have to wait. 

‘’Knowing that the majority of the cases are ultimately approved and that this wait is often unnecessary makes it even tougher,’’ she said.

Dr. Chino, a breast cancer specialist, has taken to warning patients before the alarming insurance letter arrives in the mail that their insurance may delay authorizing their care. But she tells patients that she will do everything she can to fight for them and develops a back-up plan to pivot to quickly, if needed.

‘’No one goes into medicine to spend their time talking to insurance companies,’’ said Dr. Chino.

The national trade group, America’s Health Insurance Plans (AHIP), did not return repeated requests for an interview for this story. But their official position, as stated on their website, is that “prior authorization is one of many tools health insurance providers use to promote safe, timely, evidence-based, affordable, and efficient care.”

Both Dr. Giap and Dr. Chino believe that prior authorization was developed with good intentions: to save healthcare costs and rein in treatments that don’t necessarily benefit patients. 

But, in their specialty, the burden has proliferated to a point that Dr. Chino characterizes as ‘’unconscionable.’’

She believes that policy changes like the proposed Improving Seniors’ Timely Access to Care Act — which would require real-time decisions for procedures that are routinely approved — could go a long way in improving patient care.

Meanwhile, Dr. Giap said, more research and professional guidelines are necessary to bolster insurance company confidence in newer technologies, particularly for rare cancers.

Her patient ultimately got her proton therapy and is ‘’doing relatively well, all things considered.’’

But not all the stories end like this.

Dr. Chino will never forget a patient with a cancer growing so rapidly she could see it protruding through her chest wall. She called for an urgent PET scan to see where else in the body the cancer might be brewing and rushed the planning process for radiation therapy, both of which faced prior authorization barriers. That scan — which ultimately showed the cancer had spread — was delayed for months.*

If the team had had those imaging results upfront, she said, they would have recommended a completely different course of treatment.

And her patient might be alive today.

‘’Unfortunately,” Dr. Chino said, “the people with the very worst prior authorization stories aren’t here anymore to tell you about them.”

*Correction,  10/4/24: An earlier version of this article erroneously stated that Dr. Chino called for surgery for her patient. She actually called for a PET scan and an urgent radiation start.

A version of this article first appeared on Medscape.com.

There’s a new morning ritual in Pinedale, Wyoming, a town of about 2000, nestled against the Wind River Mountains.

Friends and neighbors in the oil- and gas-rich community “take their morning coffee and pull up” to watch workers building the county’s first hospital, said Kari DeWitt, the project’s public relations director.

“I think it’s just gratitude,” Ms. DeWitt said.

Sublette County is the only one in Wyoming — where counties span thousands of square miles — without a hospital. The 10-bed, 40,000-square-foot hospital, with a similarly sized attached long-term care facility, is slated to open by the summer of 2025.

Ms. DeWitt, who also is executive director of the Sublette County Health Foundation, has an office at the town’s health clinic with a window view of the construction.

Pinedale’s residents have good reason to be excited. New full-service hospitals with inpatient beds are rare in rural America, where declining population has spurred decades of downsizing and closures. Yet, a few communities in Wyoming and others in Kansas and Georgia are defying the trend.

“To be honest with you, it even seems strange to me,” said Wyoming Hospital Association President Eric Boley. Small rural “hospitals are really struggling all across the country,” he said.

There is no official tally of new hospitals being built in rural America, but industry experts such as Mr. Boley said they’re rare. Typically, health-related construction projects in rural areas are for smaller urgent care centers or stand-alone emergency facilities or are replacements for old hospitals.

About half of rural hospitals lost money in the prior year, according to Chartis, a health analytics and consulting firm. And nearly 150 rural hospitals have closed or converted to smaller operations since 2010, according to data collected by the University of North Carolina’s Cecil G. Sheps Center for Health Services Research.

To stem the tide of closures, Congress created a new rural emergency hospital designation that allowed struggling hospitals to close their inpatient units and provide only outpatient and emergency services. Since January 2023, when the program took effect, 32 of the more than 1700 eligible rural hospitals — from Georgia to New Mexico — have joined the program, according to data from the Centers for Medicare & Medicaid Services.

Tony Breitlow is healthcare studio director for EUA, which has extensive experience working for rural health care systems. Mr. Breitlow said his national architecture and engineering firm’s work expands, replaces, or revamps older buildings, many of which were constructed during the middle of the last century.

The work, Mr. Breitlow said, is part of health care “systems figuring out how to remain robust and viable.”

Freeman Health System, based in Joplin, Missouri, announced plans last year to build a new 50-bed hospital across the state line in Kansas. Paula Baker, Freeman’s president and chief executive, said the system is building for patients in the southeastern corner of the state who travel 45 minutes or more to its bigger Joplin facilities for care.

Freeman’s new hospital, with construction on the building expected to begin in the spring, will be less than 10 miles away from an older, 64-bed hospital that has existed for decades. Kansas is one of more than a dozen states with no “certificate of need” law that would require health providers to obtain approval from the state before offering new services or building or expanding facilities.

Ms. Baker also said Freeman plans to operate emergency services and a small 10-bed outpost in Fort Scott, Kansas, opening early next year in a corner of a hospital that closed in late 2018. Residents there “cried, they cheered, they hugged me,” Ms. Baker said, adding that the “level of appreciation and gratitude that they felt and they displayed was overwhelming to me.”

Michael Topchik, executive director of the Chartis Center for Rural Health, said regional healthcare systems in the Upper Midwest have been particularly active in competing for patients by, among other things, building new hospitals.

And while private corporate money can drive construction, many rural hospital projects tap government programs, especially those supported by the US Department of Agriculture, Mr. Topchik said. That, he said, “surprises a lot of people.”

Since 2021, the USDA’s rural Community Facilities Programs have awarded $2.24 billion in loans and grants to 68 rural hospitals for work that was not related to an emergency or disaster, according to data analyzed by KFF Health News and confirmed by the agency. The federal program is funded through what is often known as the farm bill, which faces a September congressional renewal deadline.

Nearly all the projects are replacements or expansions and updates of older facilities.

The USDA confirmed that three new or planned Wyoming hospitals received federal funding. Hospital projects in Riverton and Saratoga received loans of $37.2 million and $18.3 million, respectively. Pinedale’s hospital received a $29.2 million loan from the agency.

Wyoming’s new construction is rare in a state where more than 80% of rural hospitals reported losses in the third quarter of 2023, according to Chartis. The state association’s Mr. Boley said he worries about several hospitals that have less than 10 days’ cash on hand “day and night.”

Pinedale’s project loan was approved after the community submitted a feasibility study to the USDA that included local clinics and a long-term care facility. “It’s pretty remote and right up in the mountains,” Mr. Boley said.

Pinedale’s Ms. DeWitt said the community was missing key services, such as blood transfusions, which are often necessary when there is a trauma like a car crash or if a pregnant woman faces severe complications. Local ambulances drove 94,000 miles last year, she said.

Ms. DeWitt began working to raise support for the new hospital after her own pregnancy-related trauma in 2014. She was bleeding heavily and arrived at the local health clinic believing it operated like a hospital.

“It was shocking to hear, ‘No, we’re not a hospital. We can’t do blood transfusions. We’re just going to have to pray you live for the next 45 minutes,’ ” Ms. DeWitt said.

Ms. DeWitt had to be airlifted to Idaho, where she delivered a few minutes after landing. When the hospital financing went on the ballot in 2020, Ms. DeWitt — fully recovered, with healthy grade-schoolers at home — began making five calls a night to rally support for a county tax increase to help fund the hospital.

“By improving health care, I think we improve everybody’s chances of survival. You know, it’s pretty basic,” Ms. DeWitt said.

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF—an independent source of health policy research, polling, and journalism. Learn more about KFF.

There’s a new morning ritual in Pinedale, Wyoming, a town of about 2000, nestled against the Wind River Mountains.

Friends and neighbors in the oil- and gas-rich community “take their morning coffee and pull up” to watch workers building the county’s first hospital, said Kari DeWitt, the project’s public relations director.

“I think it’s just gratitude,” Ms. DeWitt said.

Sublette County is the only one in Wyoming — where counties span thousands of square miles — without a hospital. The 10-bed, 40,000-square-foot hospital, with a similarly sized attached long-term care facility, is slated to open by the summer of 2025.

Ms. DeWitt, who also is executive director of the Sublette County Health Foundation, has an office at the town’s health clinic with a window view of the construction.

Pinedale’s residents have good reason to be excited. New full-service hospitals with inpatient beds are rare in rural America, where declining population has spurred decades of downsizing and closures. Yet, a few communities in Wyoming and others in Kansas and Georgia are defying the trend.

“To be honest with you, it even seems strange to me,” said Wyoming Hospital Association President Eric Boley. Small rural “hospitals are really struggling all across the country,” he said.

There is no official tally of new hospitals being built in rural America, but industry experts such as Mr. Boley said they’re rare. Typically, health-related construction projects in rural areas are for smaller urgent care centers or stand-alone emergency facilities or are replacements for old hospitals.

About half of rural hospitals lost money in the prior year, according to Chartis, a health analytics and consulting firm. And nearly 150 rural hospitals have closed or converted to smaller operations since 2010, according to data collected by the University of North Carolina’s Cecil G. Sheps Center for Health Services Research.

To stem the tide of closures, Congress created a new rural emergency hospital designation that allowed struggling hospitals to close their inpatient units and provide only outpatient and emergency services. Since January 2023, when the program took effect, 32 of the more than 1700 eligible rural hospitals — from Georgia to New Mexico — have joined the program, according to data from the Centers for Medicare & Medicaid Services.

Tony Breitlow is healthcare studio director for EUA, which has extensive experience working for rural health care systems. Mr. Breitlow said his national architecture and engineering firm’s work expands, replaces, or revamps older buildings, many of which were constructed during the middle of the last century.

The work, Mr. Breitlow said, is part of health care “systems figuring out how to remain robust and viable.”

Freeman Health System, based in Joplin, Missouri, announced plans last year to build a new 50-bed hospital across the state line in Kansas. Paula Baker, Freeman’s president and chief executive, said the system is building for patients in the southeastern corner of the state who travel 45 minutes or more to its bigger Joplin facilities for care.

Freeman’s new hospital, with construction on the building expected to begin in the spring, will be less than 10 miles away from an older, 64-bed hospital that has existed for decades. Kansas is one of more than a dozen states with no “certificate of need” law that would require health providers to obtain approval from the state before offering new services or building or expanding facilities.

Ms. Baker also said Freeman plans to operate emergency services and a small 10-bed outpost in Fort Scott, Kansas, opening early next year in a corner of a hospital that closed in late 2018. Residents there “cried, they cheered, they hugged me,” Ms. Baker said, adding that the “level of appreciation and gratitude that they felt and they displayed was overwhelming to me.”

Michael Topchik, executive director of the Chartis Center for Rural Health, said regional healthcare systems in the Upper Midwest have been particularly active in competing for patients by, among other things, building new hospitals.

And while private corporate money can drive construction, many rural hospital projects tap government programs, especially those supported by the US Department of Agriculture, Mr. Topchik said. That, he said, “surprises a lot of people.”

Since 2021, the USDA’s rural Community Facilities Programs have awarded $2.24 billion in loans and grants to 68 rural hospitals for work that was not related to an emergency or disaster, according to data analyzed by KFF Health News and confirmed by the agency. The federal program is funded through what is often known as the farm bill, which faces a September congressional renewal deadline.

Nearly all the projects are replacements or expansions and updates of older facilities.

The USDA confirmed that three new or planned Wyoming hospitals received federal funding. Hospital projects in Riverton and Saratoga received loans of $37.2 million and $18.3 million, respectively. Pinedale’s hospital received a $29.2 million loan from the agency.

Wyoming’s new construction is rare in a state where more than 80% of rural hospitals reported losses in the third quarter of 2023, according to Chartis. The state association’s Mr. Boley said he worries about several hospitals that have less than 10 days’ cash on hand “day and night.”

Pinedale’s project loan was approved after the community submitted a feasibility study to the USDA that included local clinics and a long-term care facility. “It’s pretty remote and right up in the mountains,” Mr. Boley said.

Pinedale’s Ms. DeWitt said the community was missing key services, such as blood transfusions, which are often necessary when there is a trauma like a car crash or if a pregnant woman faces severe complications. Local ambulances drove 94,000 miles last year, she said.

Ms. DeWitt began working to raise support for the new hospital after her own pregnancy-related trauma in 2014. She was bleeding heavily and arrived at the local health clinic believing it operated like a hospital.

“It was shocking to hear, ‘No, we’re not a hospital. We can’t do blood transfusions. We’re just going to have to pray you live for the next 45 minutes,’ ” Ms. DeWitt said.

Ms. DeWitt had to be airlifted to Idaho, where she delivered a few minutes after landing. When the hospital financing went on the ballot in 2020, Ms. DeWitt — fully recovered, with healthy grade-schoolers at home — began making five calls a night to rally support for a county tax increase to help fund the hospital.

“By improving health care, I think we improve everybody’s chances of survival. You know, it’s pretty basic,” Ms. DeWitt said.

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF—an independent source of health policy research, polling, and journalism. Learn more about KFF.

There’s a new morning ritual in Pinedale, Wyoming, a town of about 2000, nestled against the Wind River Mountains.

Friends and neighbors in the oil- and gas-rich community “take their morning coffee and pull up” to watch workers building the county’s first hospital, said Kari DeWitt, the project’s public relations director.

“I think it’s just gratitude,” Ms. DeWitt said.

Sublette County is the only one in Wyoming — where counties span thousands of square miles — without a hospital. The 10-bed, 40,000-square-foot hospital, with a similarly sized attached long-term care facility, is slated to open by the summer of 2025.

Ms. DeWitt, who also is executive director of the Sublette County Health Foundation, has an office at the town’s health clinic with a window view of the construction.

Pinedale’s residents have good reason to be excited. New full-service hospitals with inpatient beds are rare in rural America, where declining population has spurred decades of downsizing and closures. Yet, a few communities in Wyoming and others in Kansas and Georgia are defying the trend.

“To be honest with you, it even seems strange to me,” said Wyoming Hospital Association President Eric Boley. Small rural “hospitals are really struggling all across the country,” he said.

There is no official tally of new hospitals being built in rural America, but industry experts such as Mr. Boley said they’re rare. Typically, health-related construction projects in rural areas are for smaller urgent care centers or stand-alone emergency facilities or are replacements for old hospitals.

About half of rural hospitals lost money in the prior year, according to Chartis, a health analytics and consulting firm. And nearly 150 rural hospitals have closed or converted to smaller operations since 2010, according to data collected by the University of North Carolina’s Cecil G. Sheps Center for Health Services Research.

To stem the tide of closures, Congress created a new rural emergency hospital designation that allowed struggling hospitals to close their inpatient units and provide only outpatient and emergency services. Since January 2023, when the program took effect, 32 of the more than 1700 eligible rural hospitals — from Georgia to New Mexico — have joined the program, according to data from the Centers for Medicare & Medicaid Services.

Tony Breitlow is healthcare studio director for EUA, which has extensive experience working for rural health care systems. Mr. Breitlow said his national architecture and engineering firm’s work expands, replaces, or revamps older buildings, many of which were constructed during the middle of the last century.

The work, Mr. Breitlow said, is part of health care “systems figuring out how to remain robust and viable.”

Freeman Health System, based in Joplin, Missouri, announced plans last year to build a new 50-bed hospital across the state line in Kansas. Paula Baker, Freeman’s president and chief executive, said the system is building for patients in the southeastern corner of the state who travel 45 minutes or more to its bigger Joplin facilities for care.

Freeman’s new hospital, with construction on the building expected to begin in the spring, will be less than 10 miles away from an older, 64-bed hospital that has existed for decades. Kansas is one of more than a dozen states with no “certificate of need” law that would require health providers to obtain approval from the state before offering new services or building or expanding facilities.

Ms. Baker also said Freeman plans to operate emergency services and a small 10-bed outpost in Fort Scott, Kansas, opening early next year in a corner of a hospital that closed in late 2018. Residents there “cried, they cheered, they hugged me,” Ms. Baker said, adding that the “level of appreciation and gratitude that they felt and they displayed was overwhelming to me.”

Michael Topchik, executive director of the Chartis Center for Rural Health, said regional healthcare systems in the Upper Midwest have been particularly active in competing for patients by, among other things, building new hospitals.

And while private corporate money can drive construction, many rural hospital projects tap government programs, especially those supported by the US Department of Agriculture, Mr. Topchik said. That, he said, “surprises a lot of people.”

Since 2021, the USDA’s rural Community Facilities Programs have awarded $2.24 billion in loans and grants to 68 rural hospitals for work that was not related to an emergency or disaster, according to data analyzed by KFF Health News and confirmed by the agency. The federal program is funded through what is often known as the farm bill, which faces a September congressional renewal deadline.

Nearly all the projects are replacements or expansions and updates of older facilities.

The USDA confirmed that three new or planned Wyoming hospitals received federal funding. Hospital projects in Riverton and Saratoga received loans of $37.2 million and $18.3 million, respectively. Pinedale’s hospital received a $29.2 million loan from the agency.

Wyoming’s new construction is rare in a state where more than 80% of rural hospitals reported losses in the third quarter of 2023, according to Chartis. The state association’s Mr. Boley said he worries about several hospitals that have less than 10 days’ cash on hand “day and night.”

Pinedale’s project loan was approved after the community submitted a feasibility study to the USDA that included local clinics and a long-term care facility. “It’s pretty remote and right up in the mountains,” Mr. Boley said.

Pinedale’s Ms. DeWitt said the community was missing key services, such as blood transfusions, which are often necessary when there is a trauma like a car crash or if a pregnant woman faces severe complications. Local ambulances drove 94,000 miles last year, she said.

Ms. DeWitt began working to raise support for the new hospital after her own pregnancy-related trauma in 2014. She was bleeding heavily and arrived at the local health clinic believing it operated like a hospital.

“It was shocking to hear, ‘No, we’re not a hospital. We can’t do blood transfusions. We’re just going to have to pray you live for the next 45 minutes,’ ” Ms. DeWitt said.

Ms. DeWitt had to be airlifted to Idaho, where she delivered a few minutes after landing. When the hospital financing went on the ballot in 2020, Ms. DeWitt — fully recovered, with healthy grade-schoolers at home — began making five calls a night to rally support for a county tax increase to help fund the hospital.

“By improving health care, I think we improve everybody’s chances of survival. You know, it’s pretty basic,” Ms. DeWitt said.

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF—an independent source of health policy research, polling, and journalism. Learn more about KFF.

Gitanjali Srivastava, MD, professor of medicine, pediatrics, and surgery, and medical director of obesity medicine at Vanderbilt University School of Medicine in Nashville, Tennessee, was nearly 10 years into practicing pediatric medicine when she graduated from the obesity medicine fellowship at Massachusetts General Hospital in Boston in 2013. “We were the very first sort of fellows to speak of then; there were no standards or curriculum,” she said.

Obesity was already epidemic, but stigma and bias were still pervasive in the medical community and within the public. After graduating, Dr. Srivastava spent months vying for a position with hospital CEOs. She traveled across the country explaining the specialty and its value, going into detail about the budget, business model, space requirement, and revenue potential of obesity medicine. 

Today marks a very different era.

Obesity medicine is exploding. Patients are spilling into doctors’ offices looking for obesity treatment. Healthcare systems are seeking out obesity specialists and building metabolic health centers. Since 2020, the number of doctors board-certified by the American Board of Obesity Medicine has nearly doubled, and the number of obesity medicine fellowships across the country has more than doubled. Next month, another 2115 doctors from primary care, surgery, orthopedics, pediatrics, fertility, endocrinology, and beyond will sit for the 2024 exam. The once niche specialty is quickly becoming intertwined with most of modern medicine.
 

The Need to Treat

It’s no mystery that the rapid expansion of obesity medicine coincides with the US Food and Drug Administration’s approval of GLP-1 injections. The drugs’ radical weight loss properties have captured headlines and driven up patient demand. Meanwhile, doctors are finally able to offer effective treatment for a disease that affects 40% of US adults.

“We are finally treating it as a chronic disease, not as a lifestyle,” said Marcio Griebeler, MD, director of the obesity medicine fellowship at the Cleveland Clinic. And “I think it’s fulfilling for physicians,” he said. 

For so long, the advice for obesity was about lifestyle. Move more, eat less, and harness willpower, “which really is a fallacy,” said Kimberly Gudzune, MD, MPH, an obesity medicine specialist and chief medical officer for the American Board of Obesity Medicine (ABOM) Foundation. For people with obesity, “your brain is operating differently,” she said. “Your body really is set up to work against you.” 

Brianna Johnson-Rabbett, MD, medical director of the ABOM, told this news organization that with the advent of GLP-1s, “there’s a clearer recognition that obesity is a disease that needs to be treated like other diseases.” Some of that is thanks to clinical trial data showing that just as with other diseases such as high blood pressure or diabetes, obesity can be treated with medication and it resurges when the medication is stopped, she said.

Doctors don’t have to go looking for patients with obesity, dr. Griebeler added. Now that treatment options exist, they’re showing up in droves at the doctor’s office — all the doctors’ offices. In primary care, endocrinology, surgery, pediatrics — a wide variety of doctors are being asked about obesity drugs, Dr. Griebeler noted.

And while doctors are often just as excited as patients about the potential for treatment, many find themselves under-equipped when it comes to obesity. “More physicians are ... recognizing the value in treating this, and some are realizing, “Oh gosh, I never learned how to do this,” said Dr. Gudzune.
 

Information Patients Have Been Waiting For

Medical training has traditionally devoted minimal, if any, curriculum to obesity and metabolism. “To be honest, we didn’t really cover this at all in my training,” said Nina Paddu, MD, obesity medicine specialist at Maimonides Medical Center in New York City who finished her training only 2 years ago. “The guidance even in residency was ‘let’s send them to nutrition’ and ‘recommend exercising.’ ”

In addition to the medical education gap, until recently there was a “paucity of robust evidence,” Dr. Srivastava said. Leaders in the field wanted to establish standards and guidelines, but there wasn’t enough strong evidence on obesity and its treatments to build them, she said. 

Only in the last 5 years or so has the evidence-based understanding of obesity’s pathophysiology truly accelerated: The brain’s driving roles, its interplay with hormones, and its interactions with other diseases. “We are just at the cusp of understanding all the different factors,” Dr. Gudzune said.

But already endocrinologists, surgeons, fertility specialists, gynecologists, and oncologists, to name a few, see the critical overlap with their own field. “Conditions were once suspected of being intertwined [with obesity], and now we have data to connect them,” Dr. Srivastava said. For example, there’s now data connecting semaglutide to a 20% reduction in cardiovascular events for people with obesity. That’s a game changer for multiple specialties, she told this news organization. 
 

Getting Trained in Obesity Management

The recent uptick in obesity insights and increased patient need has doctors from every career stage seeking additional training.

The ABOM offers two board certification pathways: 60 hours of CME credits or a 12-month fellowship. Both paths require doctors to pass the board’s exam. 

Many doctors incorporate the training into their existing practice. The CME credit pathway, especially, is designed to help get doctors up to speed without requiring them to upend their lives for a fellowship.

Dr. Srivastava said that the fellowship is more consuming and immersive. While it’s often younger doctors just out of training who apply to fellowship, every year, “I’m astonished at the number of talented physicians with clinical and research experience who want to immerse themselves in a fellowship experience.”

Some doctors return to their previous specialties after fellowship. But many will go on to take obesity medicine–specific roles or set aside clinic hours for obesity medicine. Their credentials are “really attractive to institutions, especially those looking to open up obesity medicine or weight management programs,” said Dr. Srivastava.

Dr. Paddu, who finished her obesity medicine fellowship this year, said there are a variety of obesity medicine jobs to choose from — far different from Dr. Srivastava’s job search 15 years ago. Dr. Paddu’s new role combines 2 days of primary care with 2 days devoted to obesity medicine and 1 day each week set aside for administrative work so she can build up the hospital’s new metabolic health clinic. 
 

Still Not Enough Obesity Specialists

As with all things, rapid growth requires careful oversight. “Part of the responsibility of the board is to think critically of how the field is growing” and conduct ongoing monitoring, Dr. Gudzune said.

This is also why the board’s credentials are time-limited and must be recertified, Dr. Johnson-Rabbett added. 

But even with the rise in certified doctors and obesity medicine positions, the 8263 doctors certified by ABOM are only a tiny fraction of US physicians. As a result, there’s genuine likelihood that many patients seeking GLP-1s or other obesity treatment don’t yet have access to the holistic care they need. Plus, doctors may still not have obesity expertise within their networks.

“The field has grown rapidly, but it’s still such a small field relative to the patient need,” said Dr. Gudzune.
 

A version of this article appeared on Medscape.com.

Gitanjali Srivastava, MD, professor of medicine, pediatrics, and surgery, and medical director of obesity medicine at Vanderbilt University School of Medicine in Nashville, Tennessee, was nearly 10 years into practicing pediatric medicine when she graduated from the obesity medicine fellowship at Massachusetts General Hospital in Boston in 2013. “We were the very first sort of fellows to speak of then; there were no standards or curriculum,” she said.

Obesity was already epidemic, but stigma and bias were still pervasive in the medical community and within the public. After graduating, Dr. Srivastava spent months vying for a position with hospital CEOs. She traveled across the country explaining the specialty and its value, going into detail about the budget, business model, space requirement, and revenue potential of obesity medicine. 

Today marks a very different era.

Obesity medicine is exploding. Patients are spilling into doctors’ offices looking for obesity treatment. Healthcare systems are seeking out obesity specialists and building metabolic health centers. Since 2020, the number of doctors board-certified by the American Board of Obesity Medicine has nearly doubled, and the number of obesity medicine fellowships across the country has more than doubled. Next month, another 2115 doctors from primary care, surgery, orthopedics, pediatrics, fertility, endocrinology, and beyond will sit for the 2024 exam. The once niche specialty is quickly becoming intertwined with most of modern medicine.
 

The Need to Treat

It’s no mystery that the rapid expansion of obesity medicine coincides with the US Food and Drug Administration’s approval of GLP-1 injections. The drugs’ radical weight loss properties have captured headlines and driven up patient demand. Meanwhile, doctors are finally able to offer effective treatment for a disease that affects 40% of US adults.

“We are finally treating it as a chronic disease, not as a lifestyle,” said Marcio Griebeler, MD, director of the obesity medicine fellowship at the Cleveland Clinic. And “I think it’s fulfilling for physicians,” he said. 

For so long, the advice for obesity was about lifestyle. Move more, eat less, and harness willpower, “which really is a fallacy,” said Kimberly Gudzune, MD, MPH, an obesity medicine specialist and chief medical officer for the American Board of Obesity Medicine (ABOM) Foundation. For people with obesity, “your brain is operating differently,” she said. “Your body really is set up to work against you.” 

Brianna Johnson-Rabbett, MD, medical director of the ABOM, told this news organization that with the advent of GLP-1s, “there’s a clearer recognition that obesity is a disease that needs to be treated like other diseases.” Some of that is thanks to clinical trial data showing that just as with other diseases such as high blood pressure or diabetes, obesity can be treated with medication and it resurges when the medication is stopped, she said.

Doctors don’t have to go looking for patients with obesity, dr. Griebeler added. Now that treatment options exist, they’re showing up in droves at the doctor’s office — all the doctors’ offices. In primary care, endocrinology, surgery, pediatrics — a wide variety of doctors are being asked about obesity drugs, Dr. Griebeler noted.

And while doctors are often just as excited as patients about the potential for treatment, many find themselves under-equipped when it comes to obesity. “More physicians are ... recognizing the value in treating this, and some are realizing, “Oh gosh, I never learned how to do this,” said Dr. Gudzune.
 

Information Patients Have Been Waiting For

Medical training has traditionally devoted minimal, if any, curriculum to obesity and metabolism. “To be honest, we didn’t really cover this at all in my training,” said Nina Paddu, MD, obesity medicine specialist at Maimonides Medical Center in New York City who finished her training only 2 years ago. “The guidance even in residency was ‘let’s send them to nutrition’ and ‘recommend exercising.’ ”

In addition to the medical education gap, until recently there was a “paucity of robust evidence,” Dr. Srivastava said. Leaders in the field wanted to establish standards and guidelines, but there wasn’t enough strong evidence on obesity and its treatments to build them, she said. 

Only in the last 5 years or so has the evidence-based understanding of obesity’s pathophysiology truly accelerated: The brain’s driving roles, its interplay with hormones, and its interactions with other diseases. “We are just at the cusp of understanding all the different factors,” Dr. Gudzune said.

But already endocrinologists, surgeons, fertility specialists, gynecologists, and oncologists, to name a few, see the critical overlap with their own field. “Conditions were once suspected of being intertwined [with obesity], and now we have data to connect them,” Dr. Srivastava said. For example, there’s now data connecting semaglutide to a 20% reduction in cardiovascular events for people with obesity. That’s a game changer for multiple specialties, she told this news organization. 
 

Getting Trained in Obesity Management

The recent uptick in obesity insights and increased patient need has doctors from every career stage seeking additional training.

The ABOM offers two board certification pathways: 60 hours of CME credits or a 12-month fellowship. Both paths require doctors to pass the board’s exam. 

Many doctors incorporate the training into their existing practice. The CME credit pathway, especially, is designed to help get doctors up to speed without requiring them to upend their lives for a fellowship.

Dr. Srivastava said that the fellowship is more consuming and immersive. While it’s often younger doctors just out of training who apply to fellowship, every year, “I’m astonished at the number of talented physicians with clinical and research experience who want to immerse themselves in a fellowship experience.”

Some doctors return to their previous specialties after fellowship. But many will go on to take obesity medicine–specific roles or set aside clinic hours for obesity medicine. Their credentials are “really attractive to institutions, especially those looking to open up obesity medicine or weight management programs,” said Dr. Srivastava.

Dr. Paddu, who finished her obesity medicine fellowship this year, said there are a variety of obesity medicine jobs to choose from — far different from Dr. Srivastava’s job search 15 years ago. Dr. Paddu’s new role combines 2 days of primary care with 2 days devoted to obesity medicine and 1 day each week set aside for administrative work so she can build up the hospital’s new metabolic health clinic. 
 

Still Not Enough Obesity Specialists

As with all things, rapid growth requires careful oversight. “Part of the responsibility of the board is to think critically of how the field is growing” and conduct ongoing monitoring, Dr. Gudzune said.

This is also why the board’s credentials are time-limited and must be recertified, Dr. Johnson-Rabbett added. 

But even with the rise in certified doctors and obesity medicine positions, the 8263 doctors certified by ABOM are only a tiny fraction of US physicians. As a result, there’s genuine likelihood that many patients seeking GLP-1s or other obesity treatment don’t yet have access to the holistic care they need. Plus, doctors may still not have obesity expertise within their networks.

“The field has grown rapidly, but it’s still such a small field relative to the patient need,” said Dr. Gudzune.
 

A version of this article appeared on Medscape.com.

Gitanjali Srivastava, MD, professor of medicine, pediatrics, and surgery, and medical director of obesity medicine at Vanderbilt University School of Medicine in Nashville, Tennessee, was nearly 10 years into practicing pediatric medicine when she graduated from the obesity medicine fellowship at Massachusetts General Hospital in Boston in 2013. “We were the very first sort of fellows to speak of then; there were no standards or curriculum,” she said.

Obesity was already epidemic, but stigma and bias were still pervasive in the medical community and within the public. After graduating, Dr. Srivastava spent months vying for a position with hospital CEOs. She traveled across the country explaining the specialty and its value, going into detail about the budget, business model, space requirement, and revenue potential of obesity medicine. 

Today marks a very different era.

Obesity medicine is exploding. Patients are spilling into doctors’ offices looking for obesity treatment. Healthcare systems are seeking out obesity specialists and building metabolic health centers. Since 2020, the number of doctors board-certified by the American Board of Obesity Medicine has nearly doubled, and the number of obesity medicine fellowships across the country has more than doubled. Next month, another 2115 doctors from primary care, surgery, orthopedics, pediatrics, fertility, endocrinology, and beyond will sit for the 2024 exam. The once niche specialty is quickly becoming intertwined with most of modern medicine.
 

The Need to Treat

It’s no mystery that the rapid expansion of obesity medicine coincides with the US Food and Drug Administration’s approval of GLP-1 injections. The drugs’ radical weight loss properties have captured headlines and driven up patient demand. Meanwhile, doctors are finally able to offer effective treatment for a disease that affects 40% of US adults.

“We are finally treating it as a chronic disease, not as a lifestyle,” said Marcio Griebeler, MD, director of the obesity medicine fellowship at the Cleveland Clinic. And “I think it’s fulfilling for physicians,” he said. 

For so long, the advice for obesity was about lifestyle. Move more, eat less, and harness willpower, “which really is a fallacy,” said Kimberly Gudzune, MD, MPH, an obesity medicine specialist and chief medical officer for the American Board of Obesity Medicine (ABOM) Foundation. For people with obesity, “your brain is operating differently,” she said. “Your body really is set up to work against you.” 

Brianna Johnson-Rabbett, MD, medical director of the ABOM, told this news organization that with the advent of GLP-1s, “there’s a clearer recognition that obesity is a disease that needs to be treated like other diseases.” Some of that is thanks to clinical trial data showing that just as with other diseases such as high blood pressure or diabetes, obesity can be treated with medication and it resurges when the medication is stopped, she said.

Doctors don’t have to go looking for patients with obesity, dr. Griebeler added. Now that treatment options exist, they’re showing up in droves at the doctor’s office — all the doctors’ offices. In primary care, endocrinology, surgery, pediatrics — a wide variety of doctors are being asked about obesity drugs, Dr. Griebeler noted.

And while doctors are often just as excited as patients about the potential for treatment, many find themselves under-equipped when it comes to obesity. “More physicians are ... recognizing the value in treating this, and some are realizing, “Oh gosh, I never learned how to do this,” said Dr. Gudzune.
 

Information Patients Have Been Waiting For

Medical training has traditionally devoted minimal, if any, curriculum to obesity and metabolism. “To be honest, we didn’t really cover this at all in my training,” said Nina Paddu, MD, obesity medicine specialist at Maimonides Medical Center in New York City who finished her training only 2 years ago. “The guidance even in residency was ‘let’s send them to nutrition’ and ‘recommend exercising.’ ”

In addition to the medical education gap, until recently there was a “paucity of robust evidence,” Dr. Srivastava said. Leaders in the field wanted to establish standards and guidelines, but there wasn’t enough strong evidence on obesity and its treatments to build them, she said. 

Only in the last 5 years or so has the evidence-based understanding of obesity’s pathophysiology truly accelerated: The brain’s driving roles, its interplay with hormones, and its interactions with other diseases. “We are just at the cusp of understanding all the different factors,” Dr. Gudzune said.

But already endocrinologists, surgeons, fertility specialists, gynecologists, and oncologists, to name a few, see the critical overlap with their own field. “Conditions were once suspected of being intertwined [with obesity], and now we have data to connect them,” Dr. Srivastava said. For example, there’s now data connecting semaglutide to a 20% reduction in cardiovascular events for people with obesity. That’s a game changer for multiple specialties, she told this news organization. 
 

Getting Trained in Obesity Management

The recent uptick in obesity insights and increased patient need has doctors from every career stage seeking additional training.

The ABOM offers two board certification pathways: 60 hours of CME credits or a 12-month fellowship. Both paths require doctors to pass the board’s exam. 

Many doctors incorporate the training into their existing practice. The CME credit pathway, especially, is designed to help get doctors up to speed without requiring them to upend their lives for a fellowship.

Dr. Srivastava said that the fellowship is more consuming and immersive. While it’s often younger doctors just out of training who apply to fellowship, every year, “I’m astonished at the number of talented physicians with clinical and research experience who want to immerse themselves in a fellowship experience.”

Some doctors return to their previous specialties after fellowship. But many will go on to take obesity medicine–specific roles or set aside clinic hours for obesity medicine. Their credentials are “really attractive to institutions, especially those looking to open up obesity medicine or weight management programs,” said Dr. Srivastava.

Dr. Paddu, who finished her obesity medicine fellowship this year, said there are a variety of obesity medicine jobs to choose from — far different from Dr. Srivastava’s job search 15 years ago. Dr. Paddu’s new role combines 2 days of primary care with 2 days devoted to obesity medicine and 1 day each week set aside for administrative work so she can build up the hospital’s new metabolic health clinic. 
 

Still Not Enough Obesity Specialists

As with all things, rapid growth requires careful oversight. “Part of the responsibility of the board is to think critically of how the field is growing” and conduct ongoing monitoring, Dr. Gudzune said.

This is also why the board’s credentials are time-limited and must be recertified, Dr. Johnson-Rabbett added. 

But even with the rise in certified doctors and obesity medicine positions, the 8263 doctors certified by ABOM are only a tiny fraction of US physicians. As a result, there’s genuine likelihood that many patients seeking GLP-1s or other obesity treatment don’t yet have access to the holistic care they need. Plus, doctors may still not have obesity expertise within their networks.

“The field has grown rapidly, but it’s still such a small field relative to the patient need,” said Dr. Gudzune.
 

A version of this article appeared on Medscape.com.

Common complaints of abdominal pain, fever, shortness of breath, or rash can signal more serious disease that should be referred to specialty care or might be related to benign conditions.

Combine the vague nature of many patients’ descriptions and the pressure of short visits, and clinicians have a recipe for all manner of diagnostic error.

An estimated 5% of US adults who seek outpatient care each year experience a diagnostic error. Most Americans will eventually have this experience, according to a 2015 report from the National Academy of Medicine.

The most frequently missed diagnoses in primary care involve conditions such as pneumonia, decompensated heart failure, acute renal failure, cancer, urinary tract infection, and pyelonephritis.

“It’s not one or two or three types of diagnosis that are missed: We miss a lot of things, especially in primary care,” said Hardeep Singh, MD, MPH, a patient safety researcher at the Michael E. DeBakey VA Medical Center and the Baylor College of Medicine in Houston, Texas.

One of the most serious errors is to miss cancer, with failing to follow‐up on abnormal tests ranking consistently as one of the leading causes of malpractice claims. But most diagnostic errors do not lead to lawsuits, although they often result in missed and delayed opportunities for patients to get needed care.

In interviews with this news organization, experts who have studied the root causes of diagnostic error suggested primary care clinicians focus on several practices to avoid mistakes: Ask questions with humility and curiosity, use checklists, and brainstorm with patients the potential root cause of symptoms.
 

Humility and Curiosity 

Clinicians should remain aware of the potential for errors and reach out for assistance when needed, keeping an open mind that common symptoms may, in rare cases, signal serious illness, Dr. Singh said.

Dr. Singh recommends continual review with what he calls “byte-sized” learning through digital tools such as the Human Diagnosis app and podcasts and webinars offered by Clinical Problem Solvers.

Continuing education activities such as classes for Maintenance of Certification (MOC) can help keep physicians up to date and alert for cases where seemingly common symptoms may turn out to be something serious, said Richard M. Wardrop, III, MD, PhD, an internal medicine physician at the Cleveland Clinic in Ohio, and chair of the internal medicine board at the American Board of Internal Medicine.

“I’ve been in practice for 20 years. I’m double board certified in peds and medicine, and I regularly teach students and residents and mentor other physicians, but the further I go in my career and in practice, the more humble I become,” Dr. Wardrop told this news organization.

He said he recently spent a few hours on MOC for pediatrics and found the review was helpful in his practice in medicine.

“If I find myself taking care of a patient in a newborn nursery anytime soon, I’m going to understand the new hyperbilirubinemia guidelines,” Dr. Wardrop said. “That takes time and energy, but when I was done with the questions for this quarter, I felt good about myself.”
 

Checking It Twice

Clinicians should incorporate checklists into daily practice. Reviewing these with patients can not only help rule out an illness but also serve as a nonconfrontational method to inquire about issues patients may find uncomfortable, said John Ely, MD, MSPH, professor emeritus of family medicine at the University of Iowa Carver College of Medicine in Iowa City.

Clinicians could benefit from the approach used in aviation, where checklists are a required and routine part of a pilot’s job, Dr. Ely said.

Although clinicians may assume patients expect them to work from memory and knowledge without this aide, many will see using a checklist as a sign of providing thorough care, he said.

Checklists can also open a pathway for discussions about potentially difficult or touchy issues in short visits. For example, a patient might feel defensive if a clinician asks about depression during a visit for abdominal pain. But incorporating a question in a checklist allows for a different framing of the question.

“A clinician could say ‘I didn’t say you were depressed because of your abdominal pain, I brought it up because it’s on the list,’” Dr. Ely said. The checklist is “a very easy way to bring up those things.”

Dr. Ely said he has cared for a few patients who sought help for abdominal pain that turned out to be linked to sexual abuse in their past. Dr. Ely used a checklist with these patients to review possible causes for their illness. He recalled one of these patients who had suffered sexual abuse and had depression, neither of which was readily apparent.

“There was nothing about her affect that appeared to be depressed, and she had seen multiple physicians unable to make a diagnosis,” Dr. Ely said. “She had worked up for multiple other diseases and this had never come up before.”
 

Cooperation 

“Coproduction” is how Kathryn McDonald, PhD, describes an ideal path to getting an accurate diagnosis. The intent is for clinicians to enlist patients in helping them in finding the root cause of symptoms.

“It’s bringing the patient into knowing that they are in a partnership to coproduce, knowing that there is a process going on,” said Dr. McDonald, who is codirector of the Armstrong Institute Center for Diagnostic Excellence at Johns Hopkins University in Baltimore, Maryland.

In many cases, patients seek reassurance for ruling out a suspected condition, which the physician can sometimes provide. In others, clinicians may not be able to offer a concrete diagnosis.

“There are times when uncertainty is more pervasive and I will ask patients, ‘Let’s brainstorm this together,’” Dr. Wardrop said.

A version of this article appeared on Medscape.com.

Common complaints of abdominal pain, fever, shortness of breath, or rash can signal more serious disease that should be referred to specialty care or might be related to benign conditions.

Combine the vague nature of many patients’ descriptions and the pressure of short visits, and clinicians have a recipe for all manner of diagnostic error.

An estimated 5% of US adults who seek outpatient care each year experience a diagnostic error. Most Americans will eventually have this experience, according to a 2015 report from the National Academy of Medicine.

The most frequently missed diagnoses in primary care involve conditions such as pneumonia, decompensated heart failure, acute renal failure, cancer, urinary tract infection, and pyelonephritis.

“It’s not one or two or three types of diagnosis that are missed: We miss a lot of things, especially in primary care,” said Hardeep Singh, MD, MPH, a patient safety researcher at the Michael E. DeBakey VA Medical Center and the Baylor College of Medicine in Houston, Texas.

One of the most serious errors is to miss cancer, with failing to follow‐up on abnormal tests ranking consistently as one of the leading causes of malpractice claims. But most diagnostic errors do not lead to lawsuits, although they often result in missed and delayed opportunities for patients to get needed care.

In interviews with this news organization, experts who have studied the root causes of diagnostic error suggested primary care clinicians focus on several practices to avoid mistakes: Ask questions with humility and curiosity, use checklists, and brainstorm with patients the potential root cause of symptoms.
 

Humility and Curiosity 

Clinicians should remain aware of the potential for errors and reach out for assistance when needed, keeping an open mind that common symptoms may, in rare cases, signal serious illness, Dr. Singh said.

Dr. Singh recommends continual review with what he calls “byte-sized” learning through digital tools such as the Human Diagnosis app and podcasts and webinars offered by Clinical Problem Solvers.

Continuing education activities such as classes for Maintenance of Certification (MOC) can help keep physicians up to date and alert for cases where seemingly common symptoms may turn out to be something serious, said Richard M. Wardrop, III, MD, PhD, an internal medicine physician at the Cleveland Clinic in Ohio, and chair of the internal medicine board at the American Board of Internal Medicine.

“I’ve been in practice for 20 years. I’m double board certified in peds and medicine, and I regularly teach students and residents and mentor other physicians, but the further I go in my career and in practice, the more humble I become,” Dr. Wardrop told this news organization.

He said he recently spent a few hours on MOC for pediatrics and found the review was helpful in his practice in medicine.

“If I find myself taking care of a patient in a newborn nursery anytime soon, I’m going to understand the new hyperbilirubinemia guidelines,” Dr. Wardrop said. “That takes time and energy, but when I was done with the questions for this quarter, I felt good about myself.”
 

Checking It Twice

Clinicians should incorporate checklists into daily practice. Reviewing these with patients can not only help rule out an illness but also serve as a nonconfrontational method to inquire about issues patients may find uncomfortable, said John Ely, MD, MSPH, professor emeritus of family medicine at the University of Iowa Carver College of Medicine in Iowa City.

Clinicians could benefit from the approach used in aviation, where checklists are a required and routine part of a pilot’s job, Dr. Ely said.

Although clinicians may assume patients expect them to work from memory and knowledge without this aide, many will see using a checklist as a sign of providing thorough care, he said.

Checklists can also open a pathway for discussions about potentially difficult or touchy issues in short visits. For example, a patient might feel defensive if a clinician asks about depression during a visit for abdominal pain. But incorporating a question in a checklist allows for a different framing of the question.

“A clinician could say ‘I didn’t say you were depressed because of your abdominal pain, I brought it up because it’s on the list,’” Dr. Ely said. The checklist is “a very easy way to bring up those things.”

Dr. Ely said he has cared for a few patients who sought help for abdominal pain that turned out to be linked to sexual abuse in their past. Dr. Ely used a checklist with these patients to review possible causes for their illness. He recalled one of these patients who had suffered sexual abuse and had depression, neither of which was readily apparent.

“There was nothing about her affect that appeared to be depressed, and she had seen multiple physicians unable to make a diagnosis,” Dr. Ely said. “She had worked up for multiple other diseases and this had never come up before.”
 

Cooperation 

“Coproduction” is how Kathryn McDonald, PhD, describes an ideal path to getting an accurate diagnosis. The intent is for clinicians to enlist patients in helping them in finding the root cause of symptoms.

“It’s bringing the patient into knowing that they are in a partnership to coproduce, knowing that there is a process going on,” said Dr. McDonald, who is codirector of the Armstrong Institute Center for Diagnostic Excellence at Johns Hopkins University in Baltimore, Maryland.

In many cases, patients seek reassurance for ruling out a suspected condition, which the physician can sometimes provide. In others, clinicians may not be able to offer a concrete diagnosis.

“There are times when uncertainty is more pervasive and I will ask patients, ‘Let’s brainstorm this together,’” Dr. Wardrop said.

A version of this article appeared on Medscape.com.

Common complaints of abdominal pain, fever, shortness of breath, or rash can signal more serious disease that should be referred to specialty care or might be related to benign conditions.

Combine the vague nature of many patients’ descriptions and the pressure of short visits, and clinicians have a recipe for all manner of diagnostic error.

An estimated 5% of US adults who seek outpatient care each year experience a diagnostic error. Most Americans will eventually have this experience, according to a 2015 report from the National Academy of Medicine.

The most frequently missed diagnoses in primary care involve conditions such as pneumonia, decompensated heart failure, acute renal failure, cancer, urinary tract infection, and pyelonephritis.

“It’s not one or two or three types of diagnosis that are missed: We miss a lot of things, especially in primary care,” said Hardeep Singh, MD, MPH, a patient safety researcher at the Michael E. DeBakey VA Medical Center and the Baylor College of Medicine in Houston, Texas.

One of the most serious errors is to miss cancer, with failing to follow‐up on abnormal tests ranking consistently as one of the leading causes of malpractice claims. But most diagnostic errors do not lead to lawsuits, although they often result in missed and delayed opportunities for patients to get needed care.

In interviews with this news organization, experts who have studied the root causes of diagnostic error suggested primary care clinicians focus on several practices to avoid mistakes: Ask questions with humility and curiosity, use checklists, and brainstorm with patients the potential root cause of symptoms.
 

Humility and Curiosity 

Clinicians should remain aware of the potential for errors and reach out for assistance when needed, keeping an open mind that common symptoms may, in rare cases, signal serious illness, Dr. Singh said.

Dr. Singh recommends continual review with what he calls “byte-sized” learning through digital tools such as the Human Diagnosis app and podcasts and webinars offered by Clinical Problem Solvers.

Continuing education activities such as classes for Maintenance of Certification (MOC) can help keep physicians up to date and alert for cases where seemingly common symptoms may turn out to be something serious, said Richard M. Wardrop, III, MD, PhD, an internal medicine physician at the Cleveland Clinic in Ohio, and chair of the internal medicine board at the American Board of Internal Medicine.

“I’ve been in practice for 20 years. I’m double board certified in peds and medicine, and I regularly teach students and residents and mentor other physicians, but the further I go in my career and in practice, the more humble I become,” Dr. Wardrop told this news organization.

He said he recently spent a few hours on MOC for pediatrics and found the review was helpful in his practice in medicine.

“If I find myself taking care of a patient in a newborn nursery anytime soon, I’m going to understand the new hyperbilirubinemia guidelines,” Dr. Wardrop said. “That takes time and energy, but when I was done with the questions for this quarter, I felt good about myself.”
 

Checking It Twice

Clinicians should incorporate checklists into daily practice. Reviewing these with patients can not only help rule out an illness but also serve as a nonconfrontational method to inquire about issues patients may find uncomfortable, said John Ely, MD, MSPH, professor emeritus of family medicine at the University of Iowa Carver College of Medicine in Iowa City.

Clinicians could benefit from the approach used in aviation, where checklists are a required and routine part of a pilot’s job, Dr. Ely said.

Although clinicians may assume patients expect them to work from memory and knowledge without this aide, many will see using a checklist as a sign of providing thorough care, he said.

Checklists can also open a pathway for discussions about potentially difficult or touchy issues in short visits. For example, a patient might feel defensive if a clinician asks about depression during a visit for abdominal pain. But incorporating a question in a checklist allows for a different framing of the question.

“A clinician could say ‘I didn’t say you were depressed because of your abdominal pain, I brought it up because it’s on the list,’” Dr. Ely said. The checklist is “a very easy way to bring up those things.”

Dr. Ely said he has cared for a few patients who sought help for abdominal pain that turned out to be linked to sexual abuse in their past. Dr. Ely used a checklist with these patients to review possible causes for their illness. He recalled one of these patients who had suffered sexual abuse and had depression, neither of which was readily apparent.

“There was nothing about her affect that appeared to be depressed, and she had seen multiple physicians unable to make a diagnosis,” Dr. Ely said. “She had worked up for multiple other diseases and this had never come up before.”
 

Cooperation 

“Coproduction” is how Kathryn McDonald, PhD, describes an ideal path to getting an accurate diagnosis. The intent is for clinicians to enlist patients in helping them in finding the root cause of symptoms.

“It’s bringing the patient into knowing that they are in a partnership to coproduce, knowing that there is a process going on,” said Dr. McDonald, who is codirector of the Armstrong Institute Center for Diagnostic Excellence at Johns Hopkins University in Baltimore, Maryland.

In many cases, patients seek reassurance for ruling out a suspected condition, which the physician can sometimes provide. In others, clinicians may not be able to offer a concrete diagnosis.

“There are times when uncertainty is more pervasive and I will ask patients, ‘Let’s brainstorm this together,’” Dr. Wardrop said.

A version of this article appeared on Medscape.com.

The global withdrawal of voxelotor (Oxbryta, Pfizer) has left clinicians who treat sickle cell disease (SCD) with the urgent task of reaching patients taking the medicine, while trying to understand why it was taken off the market.

The National Alliance of Sickle Cell Centers issued a statement urging patients not to stop voxelotor abruptly. Instead, they should work out plans with their physicians and medical teams for weaning plans.

“Don’t lose faith. This a step backward, but we will stay on the path to better outcomes for everyone,” said the alliance in a statement to patients and clinicians.

On September 25, Pfizer said it would withdraw all lots of voxelotor in all markets where it is approved. The New York–based drugmaker also said it was discontinuing all active voxelotor clinical trials and expanded access programs worldwide. The cause was data that suggested “an imbalance in vaso-occlusive crises and fatal events which require further assessment.”

Pfizer told this news organization in an email exchange that it is focused on analyzing the data and will share updates in the future about presenting or publishing on this issue.

The withdrawal came amid increased scrutiny of the drug by the European Medicines Agency (EMA). The EMA in July began a review of voxelotor after data from a clinical trial showed that a higher number of deaths occurred with the drug than with placebo and another trial showed the total number of deaths was higher than anticipated.

On September 26, the EMA’s human medicines committee recommended suspending the marketing authorization of voxelotor, citing new safety data that emerged during the review. The drug had received marketing authorization for the European Union in 2022, the agency said.

The US Food and Drug Administration (FDA), which first cleared voxelotor for sale in 2019, also said it has been conducting a safety review of the drug. The agency continues to examine post-marketing clinical trial data for voxelotor, the real-world registry studies, and data from the FDA Adverse Event Reporting System. At the conclusion of this review, the FDA will communicate any additional findings, if necessary, the agency said.

The FDA said it appeared that more deaths and a higher rate of vaso-occlusive crisis occurred in patients taking voxelotor vs placebo in post-marketing clinical trials.

“Pfizer also observed a higher rate of vaso-occlusive crisis in patients with sickle cell disease receiving Oxbryta in two real-world registry studies,” the FDA said. “Based on the totality of clinical data, Pfizer has determined the benefit of Oxbryta does not outweigh the risk.”
 

Gene Therapy, Tried-and-True Hydroxyurea (HU)

As a field, SCD has drawn more interest in recent years, with significant gains made lately in cutting-edge projects.

The FDA in December approved two gene-editing treatments for patients aged 12 years or older. These are considered “milestone treatments” for a debilitating and potentially life-threatening blood disorder that affects about 100,000 people in the United States. Exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals and CRISPR Therapeutics) is the first to use the gene-editing tool CRISPR. And lovotibeglogene autotemcel (Lyfgenia, bluebird bio) uses a different gene-editing tool called a lentiviral vector.

These advances have been covered widely by the news media but are not expected to be widely available, with the cost of these extensive treatments estimated around $2-$3 million per patient.

“Gene therapy is amazing in that it can offer a cure, but it’s very expensive and not all patients are suitable for it. Some have so much existing organ damage that it’s not an option for them,” said John Wood, MD, PhD, director of cardiovascular MRI at Children’s Hospital Los Angeles, Los Angeles, who does research on SCD.

“So it really is a great treatment for a very few people,” he said in an interview.

The mainstay of treatment for SCD remains a drug that Lydia Pecker, MD, a pediatric hematologist at Johns Hopkins University in Baltimore, describes as the “first, oldest, and best”: HU.

The FDA approved this in 1998 for use in SCD. It reduces the frequency of painful crises and acute chest syndrome and other complications of SCD that otherwise could be serious or even lethal, Pecker said.

“Older doctors can tell you that what they experienced with sickle cell disease in the hospitals has been completely transformed because of the high uptake of the drug,” she said, adding that it made a “profound” change. “We just don’t have data for any other agent that’s quite like that.”

Voxelotor had been a good second drug to add for some patients, in addition to HU and blood transfusions, Dr. Pecker noted. It was a first-line drug for those for whom transfusion and HU were not options, which constitutes a relatively small number of patients, she said.

“So we have, in the last 5 years, felt more hopeful because we had something else to offer,” she said.

Alexis A. Thompson, MD, MPH, chief of the Division of Hematology at Children’s Hospital of Philadelphia in Pennsylvania, said in an interview that her organization also had patients who appeared to benefit from voxelotor, some of whom had been participants in clinical trials.

Dr. Thompson, who has been a top researcher involved in the study of gene therapy, urged the need for companies to keep seeking to expand the options for people with SCD, even after the setback with voxelotor.

“I hope that there’s an appreciation for the need for continued investment in this very serious condition, for which there are insufficient options for treatments,” Dr. Thompson said. “So ongoing investment is really needed if we expect to make progress.”

Dr. Pecker disclosed ties with Novartis, Afimmune, the American Society of Hematology, and the National Institutes of Health. Thompson reported relationships with bluebird bio, Beam, Editas, Novartis, and Novo Nordisk.
 

A version of this article first appeared on Medscape.com.

The global withdrawal of voxelotor (Oxbryta, Pfizer) has left clinicians who treat sickle cell disease (SCD) with the urgent task of reaching patients taking the medicine, while trying to understand why it was taken off the market.

The National Alliance of Sickle Cell Centers issued a statement urging patients not to stop voxelotor abruptly. Instead, they should work out plans with their physicians and medical teams for weaning plans.

“Don’t lose faith. This a step backward, but we will stay on the path to better outcomes for everyone,” said the alliance in a statement to patients and clinicians.

On September 25, Pfizer said it would withdraw all lots of voxelotor in all markets where it is approved. The New York–based drugmaker also said it was discontinuing all active voxelotor clinical trials and expanded access programs worldwide. The cause was data that suggested “an imbalance in vaso-occlusive crises and fatal events which require further assessment.”

Pfizer told this news organization in an email exchange that it is focused on analyzing the data and will share updates in the future about presenting or publishing on this issue.

The withdrawal came amid increased scrutiny of the drug by the European Medicines Agency (EMA). The EMA in July began a review of voxelotor after data from a clinical trial showed that a higher number of deaths occurred with the drug than with placebo and another trial showed the total number of deaths was higher than anticipated.

On September 26, the EMA’s human medicines committee recommended suspending the marketing authorization of voxelotor, citing new safety data that emerged during the review. The drug had received marketing authorization for the European Union in 2022, the agency said.

The US Food and Drug Administration (FDA), which first cleared voxelotor for sale in 2019, also said it has been conducting a safety review of the drug. The agency continues to examine post-marketing clinical trial data for voxelotor, the real-world registry studies, and data from the FDA Adverse Event Reporting System. At the conclusion of this review, the FDA will communicate any additional findings, if necessary, the agency said.

The FDA said it appeared that more deaths and a higher rate of vaso-occlusive crisis occurred in patients taking voxelotor vs placebo in post-marketing clinical trials.

“Pfizer also observed a higher rate of vaso-occlusive crisis in patients with sickle cell disease receiving Oxbryta in two real-world registry studies,” the FDA said. “Based on the totality of clinical data, Pfizer has determined the benefit of Oxbryta does not outweigh the risk.”
 

Gene Therapy, Tried-and-True Hydroxyurea (HU)

As a field, SCD has drawn more interest in recent years, with significant gains made lately in cutting-edge projects.

The FDA in December approved two gene-editing treatments for patients aged 12 years or older. These are considered “milestone treatments” for a debilitating and potentially life-threatening blood disorder that affects about 100,000 people in the United States. Exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals and CRISPR Therapeutics) is the first to use the gene-editing tool CRISPR. And lovotibeglogene autotemcel (Lyfgenia, bluebird bio) uses a different gene-editing tool called a lentiviral vector.

These advances have been covered widely by the news media but are not expected to be widely available, with the cost of these extensive treatments estimated around $2-$3 million per patient.

“Gene therapy is amazing in that it can offer a cure, but it’s very expensive and not all patients are suitable for it. Some have so much existing organ damage that it’s not an option for them,” said John Wood, MD, PhD, director of cardiovascular MRI at Children’s Hospital Los Angeles, Los Angeles, who does research on SCD.

“So it really is a great treatment for a very few people,” he said in an interview.

The mainstay of treatment for SCD remains a drug that Lydia Pecker, MD, a pediatric hematologist at Johns Hopkins University in Baltimore, describes as the “first, oldest, and best”: HU.

The FDA approved this in 1998 for use in SCD. It reduces the frequency of painful crises and acute chest syndrome and other complications of SCD that otherwise could be serious or even lethal, Pecker said.

“Older doctors can tell you that what they experienced with sickle cell disease in the hospitals has been completely transformed because of the high uptake of the drug,” she said, adding that it made a “profound” change. “We just don’t have data for any other agent that’s quite like that.”

Voxelotor had been a good second drug to add for some patients, in addition to HU and blood transfusions, Dr. Pecker noted. It was a first-line drug for those for whom transfusion and HU were not options, which constitutes a relatively small number of patients, she said.

“So we have, in the last 5 years, felt more hopeful because we had something else to offer,” she said.

Alexis A. Thompson, MD, MPH, chief of the Division of Hematology at Children’s Hospital of Philadelphia in Pennsylvania, said in an interview that her organization also had patients who appeared to benefit from voxelotor, some of whom had been participants in clinical trials.

Dr. Thompson, who has been a top researcher involved in the study of gene therapy, urged the need for companies to keep seeking to expand the options for people with SCD, even after the setback with voxelotor.

“I hope that there’s an appreciation for the need for continued investment in this very serious condition, for which there are insufficient options for treatments,” Dr. Thompson said. “So ongoing investment is really needed if we expect to make progress.”

Dr. Pecker disclosed ties with Novartis, Afimmune, the American Society of Hematology, and the National Institutes of Health. Thompson reported relationships with bluebird bio, Beam, Editas, Novartis, and Novo Nordisk.
 

A version of this article first appeared on Medscape.com.

The global withdrawal of voxelotor (Oxbryta, Pfizer) has left clinicians who treat sickle cell disease (SCD) with the urgent task of reaching patients taking the medicine, while trying to understand why it was taken off the market.

The National Alliance of Sickle Cell Centers issued a statement urging patients not to stop voxelotor abruptly. Instead, they should work out plans with their physicians and medical teams for weaning plans.

“Don’t lose faith. This a step backward, but we will stay on the path to better outcomes for everyone,” said the alliance in a statement to patients and clinicians.

On September 25, Pfizer said it would withdraw all lots of voxelotor in all markets where it is approved. The New York–based drugmaker also said it was discontinuing all active voxelotor clinical trials and expanded access programs worldwide. The cause was data that suggested “an imbalance in vaso-occlusive crises and fatal events which require further assessment.”

Pfizer told this news organization in an email exchange that it is focused on analyzing the data and will share updates in the future about presenting or publishing on this issue.

The withdrawal came amid increased scrutiny of the drug by the European Medicines Agency (EMA). The EMA in July began a review of voxelotor after data from a clinical trial showed that a higher number of deaths occurred with the drug than with placebo and another trial showed the total number of deaths was higher than anticipated.

On September 26, the EMA’s human medicines committee recommended suspending the marketing authorization of voxelotor, citing new safety data that emerged during the review. The drug had received marketing authorization for the European Union in 2022, the agency said.

The US Food and Drug Administration (FDA), which first cleared voxelotor for sale in 2019, also said it has been conducting a safety review of the drug. The agency continues to examine post-marketing clinical trial data for voxelotor, the real-world registry studies, and data from the FDA Adverse Event Reporting System. At the conclusion of this review, the FDA will communicate any additional findings, if necessary, the agency said.

The FDA said it appeared that more deaths and a higher rate of vaso-occlusive crisis occurred in patients taking voxelotor vs placebo in post-marketing clinical trials.

“Pfizer also observed a higher rate of vaso-occlusive crisis in patients with sickle cell disease receiving Oxbryta in two real-world registry studies,” the FDA said. “Based on the totality of clinical data, Pfizer has determined the benefit of Oxbryta does not outweigh the risk.”
 

Gene Therapy, Tried-and-True Hydroxyurea (HU)

As a field, SCD has drawn more interest in recent years, with significant gains made lately in cutting-edge projects.

The FDA in December approved two gene-editing treatments for patients aged 12 years or older. These are considered “milestone treatments” for a debilitating and potentially life-threatening blood disorder that affects about 100,000 people in the United States. Exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals and CRISPR Therapeutics) is the first to use the gene-editing tool CRISPR. And lovotibeglogene autotemcel (Lyfgenia, bluebird bio) uses a different gene-editing tool called a lentiviral vector.

These advances have been covered widely by the news media but are not expected to be widely available, with the cost of these extensive treatments estimated around $2-$3 million per patient.

“Gene therapy is amazing in that it can offer a cure, but it’s very expensive and not all patients are suitable for it. Some have so much existing organ damage that it’s not an option for them,” said John Wood, MD, PhD, director of cardiovascular MRI at Children’s Hospital Los Angeles, Los Angeles, who does research on SCD.

“So it really is a great treatment for a very few people,” he said in an interview.

The mainstay of treatment for SCD remains a drug that Lydia Pecker, MD, a pediatric hematologist at Johns Hopkins University in Baltimore, describes as the “first, oldest, and best”: HU.

The FDA approved this in 1998 for use in SCD. It reduces the frequency of painful crises and acute chest syndrome and other complications of SCD that otherwise could be serious or even lethal, Pecker said.

“Older doctors can tell you that what they experienced with sickle cell disease in the hospitals has been completely transformed because of the high uptake of the drug,” she said, adding that it made a “profound” change. “We just don’t have data for any other agent that’s quite like that.”

Voxelotor had been a good second drug to add for some patients, in addition to HU and blood transfusions, Dr. Pecker noted. It was a first-line drug for those for whom transfusion and HU were not options, which constitutes a relatively small number of patients, she said.

“So we have, in the last 5 years, felt more hopeful because we had something else to offer,” she said.

Alexis A. Thompson, MD, MPH, chief of the Division of Hematology at Children’s Hospital of Philadelphia in Pennsylvania, said in an interview that her organization also had patients who appeared to benefit from voxelotor, some of whom had been participants in clinical trials.

Dr. Thompson, who has been a top researcher involved in the study of gene therapy, urged the need for companies to keep seeking to expand the options for people with SCD, even after the setback with voxelotor.

“I hope that there’s an appreciation for the need for continued investment in this very serious condition, for which there are insufficient options for treatments,” Dr. Thompson said. “So ongoing investment is really needed if we expect to make progress.”

Dr. Pecker disclosed ties with Novartis, Afimmune, the American Society of Hematology, and the National Institutes of Health. Thompson reported relationships with bluebird bio, Beam, Editas, Novartis, and Novo Nordisk.
 

A version of this article first appeared on Medscape.com.

Demand for new weight loss drugs has surged over the past few years. 

Led by the antiobesity drugs semaglutide (Wegovy) and tirzepatide (Zepbound), these popular medications — more commonly known as glucagon-like peptide 1 (GLP-1) agonists — have become game changers for shedding excess pounds.

Aside from obesity indications, both drugs have been approved to treat type 2 diabetes under different brand names and have a growing list of other potential benefits, such as reducing inflammation and depression. 

These antiobesity drugs could even have a place in cancer care.

While there’s limited data to support the use of GLP-1 agonists for weight loss in cancer, some oncologists have begun carefully integrating the antiobesity agents into care and studying their effects in this patient population.

The reason: Research suggests that obesity can reduce the effectiveness of cancer therapies, especially in patients with breast cancer, and can increase the risk for treatment-related side effects. 

The idea is that managing patients’ weight will improve their cancer outcomes, explained Lajos Pusztai, MD, PhD, a breast cancer specialist and professor of medicine at Yale School of Medicine in New Haven, Connecticut. 

Although Dr. Pusztai and his oncology peers at Yale don’t yet use GPL-1 agonists, Neil Iyengar, MD, and colleagues have begun doing so to help some patients with breast cancer manage their weight. Dr. Iyengar estimates that a few hundred — almost 40% — of his patients are on the antiobesity drugs.

“For a patient who has really tried to reduce their weight and who is in the obese range, that’s where I think the use of these medications can be considered,” said Dr. Iyengar, a breast cancer oncologist at Memorial Sloan Kettering Cancer Center in New York City. 

Why GLP-1s in Cancer?

GLP-1 is a hormone that the small intestine releases after eating. GLP-1 agonists work by mimicking GLP-1 to trigger the release of insulin and reduce the production of glucagon — two processes that help regulate blood sugar. 

These agents, such as Wegovy (or Ozempic when prescribed for diabetes), also slow gastric emptying and can make people feel fuller longer. 

Zebound (or Mounjaro for type 2 diabetes) is considered a dual GLP-1 and glucose-dependent insulinotropic polypeptide agonist, which may enhance its weight loss benefits.

In practice, however, these drugs can increase nausea and vomiting from chemotherapy, so Dr. Iyengar typically has patients use them afterwards, during maintenance treatment.

Oncologists don’t prescribe the drugs themselves but instead refer patients to endocrinologists or weight management centers that then write the prescriptions. Taking these drugs involves weekly subcutaneous injections patients can administer themselves.

Endocrinologist Emily Gallagher, MD, PhD, of Mount Sinai Hospital in New York City, estimates she has prescribed the antiobesity drugs to a few hundred patients with cancer and, like Dr. Iyengar, uses the drugs during maintenance treatment with hormone therapy for breast cancer. She also has used these agents in patients with prostate and endometrial cancers and has found the drugs can help counter steroid weight gain in multiple myeloma. 

But, to date, the evidence for using GPL-1 agonists in cancer remains limited and the practice has not yet become widespread.

Research largely comes down to a few small retrospective studies in patients with breast cancer receiving aromatase inhibitors. Although no safety issues have emerged so far, these initial reports suggest that the drugs lead to significantly less weight loss in patients with cancer compared to the general population. 

Dr. Iyengar led one recent study, presented at the 2024 annual meeting of the American Society of Clinical Oncology, in which he and his team assessed outcomes in 75 women with breast cancer who received a GLP-1 agonist. Almost 80% of patients had diabetes, and 60% received hormone therapy, most commonly an aromatase inhibitor. Patients’ median body mass index (BMI) at baseline was 34 kg/m² (range, 23-50 kg/m²).

From baseline, patients lost 6.2 kg, on average, or about 5% of their total body weight, 12 months after initiating GLP-1 therapy. 

In contrast, phase 3 trials show much higher mean weight loss — about two times — in patients without cancer. 

Another recent study also reported modest weight loss results in patients with breast cancer undergoing endocrine therapy. The researchers reported that, at 12 months, Wegovy led to 4.34% reduction in BMI, compared with a 14% change reported in the general population. Zebound, however, was associated with a 2.31% BMI increase overall — though some patients did experience a decrease — compared with a 15% reduction in the general population. 

“These findings indicate a substantially reduced weight loss efficacy in breast cancer patients on endocrine therapy compared to the general population,” the authors concluded.

It’s unclear why the drugs appear to not work as well in patients with cancer. It’s possible that hormone therapy or metabolic changes interfere with their effectiveness, given that some cancer therapies lead to weight gain. Steroids and hormone therapies, for instance, often increase appetite, and some treatments can slow patients’ metabolism or lead to fatigue, which can make it harder to exercise.

Patients with cancer may need a higher dose of GLP-1 agonists to achieve similar weight loss to the general population, Dr. Iyengar noted.

However, Dr. Gallagher said, in her own experience, she hasn’t found the drugs to be less effective in patients with cancer, especially the newer agents, like Wegovy and Zepbound. 

As for safety, Wegovy and Zepbound both carry a black box warning for thyroid C-cell tumors, including medullary thyroid carcinoma. (Recent research, however, has found that GLP-1 agonists do not increase thyroid cancer risk). 

These antiobesity agents are also contraindicated in patients with a personal or family history of medullary thyroid carcinoma and in patients who have multiple endocrine neoplasia syndrome type 2, which is associated with medullary thyroid carcinoma.

Dr. Gallagher hasn’t seen any secondary tumors — thyroid or otherwise — in her patients with cancer, but she follows the labeling contraindications. Dr. Iyengar also noted that more recent and larger data sets have shown no impact on this risk, which may not actually exist, he said

Dr. Gallagher remains cautious about using GPL-1 agonists in patients who have had bariatric surgery because these agents can compound the slower gastric emptying and intestinal transit from surgery, potentially leading to gastrointestinal obstructions. 

Looking ahead, GPL-1 manufacturers are interested in adding cancer indications to the drug labeling. Both Dr. Iyengar and Dr. Gallagher said their institutions are in talks with companies to participate in large, multicenter, global phase 3 trials.

Dr. Iyengar welcomes the efforts, not only to test the effectiveness of GPL-1 agonists in oncology but also to “nail down” their safety in cancer. 

“I don’t think that there’s mechanistically anything that’s particularly worrisome,” and current observations suggest that these drugs are likely to be safe, Dr. Iyengar said. Even so, “GLP-1 agonists do a lot of things that we don’t fully understand yet.”

The bigger challenge, Dr. Iyengar noted, is that companies will have to show a sizable benefit to using these drugs in patients with cancer to get the Food and Drug Administration’s approval. And to move the needle on cancer-specific outcomes, these antiobesity drugs will need to demonstrate significant, durable weight loss in patients with cancer. 

But if these drugs can do that, “I think it’s going to be one of the biggest advances in medicine and oncology given the obesity and cancer epidemic,” Dr. Iyengar said. 

Dr. Iyengar has adviser and/or researcher ties with companies that make or are developing GPL-1 agonists, including AstraZeneca, Novartis, Gilead, and Pfizer. Dr. Gallagher is a consultant for Novartis, Flare Therapeutics, Reactive Biosciences, and Seagen.

A version of this article first appeared on Medscape.com.

Demand for new weight loss drugs has surged over the past few years. 

Led by the antiobesity drugs semaglutide (Wegovy) and tirzepatide (Zepbound), these popular medications — more commonly known as glucagon-like peptide 1 (GLP-1) agonists — have become game changers for shedding excess pounds.

Aside from obesity indications, both drugs have been approved to treat type 2 diabetes under different brand names and have a growing list of other potential benefits, such as reducing inflammation and depression. 

These antiobesity drugs could even have a place in cancer care.

While there’s limited data to support the use of GLP-1 agonists for weight loss in cancer, some oncologists have begun carefully integrating the antiobesity agents into care and studying their effects in this patient population.

The reason: Research suggests that obesity can reduce the effectiveness of cancer therapies, especially in patients with breast cancer, and can increase the risk for treatment-related side effects. 

The idea is that managing patients’ weight will improve their cancer outcomes, explained Lajos Pusztai, MD, PhD, a breast cancer specialist and professor of medicine at Yale School of Medicine in New Haven, Connecticut. 

Although Dr. Pusztai and his oncology peers at Yale don’t yet use GPL-1 agonists, Neil Iyengar, MD, and colleagues have begun doing so to help some patients with breast cancer manage their weight. Dr. Iyengar estimates that a few hundred — almost 40% — of his patients are on the antiobesity drugs.

“For a patient who has really tried to reduce their weight and who is in the obese range, that’s where I think the use of these medications can be considered,” said Dr. Iyengar, a breast cancer oncologist at Memorial Sloan Kettering Cancer Center in New York City. 

Why GLP-1s in Cancer?

GLP-1 is a hormone that the small intestine releases after eating. GLP-1 agonists work by mimicking GLP-1 to trigger the release of insulin and reduce the production of glucagon — two processes that help regulate blood sugar. 

These agents, such as Wegovy (or Ozempic when prescribed for diabetes), also slow gastric emptying and can make people feel fuller longer. 

Zebound (or Mounjaro for type 2 diabetes) is considered a dual GLP-1 and glucose-dependent insulinotropic polypeptide agonist, which may enhance its weight loss benefits.

In practice, however, these drugs can increase nausea and vomiting from chemotherapy, so Dr. Iyengar typically has patients use them afterwards, during maintenance treatment.

Oncologists don’t prescribe the drugs themselves but instead refer patients to endocrinologists or weight management centers that then write the prescriptions. Taking these drugs involves weekly subcutaneous injections patients can administer themselves.

Endocrinologist Emily Gallagher, MD, PhD, of Mount Sinai Hospital in New York City, estimates she has prescribed the antiobesity drugs to a few hundred patients with cancer and, like Dr. Iyengar, uses the drugs during maintenance treatment with hormone therapy for breast cancer. She also has used these agents in patients with prostate and endometrial cancers and has found the drugs can help counter steroid weight gain in multiple myeloma. 

But, to date, the evidence for using GPL-1 agonists in cancer remains limited and the practice has not yet become widespread.

Research largely comes down to a few small retrospective studies in patients with breast cancer receiving aromatase inhibitors. Although no safety issues have emerged so far, these initial reports suggest that the drugs lead to significantly less weight loss in patients with cancer compared to the general population. 

Dr. Iyengar led one recent study, presented at the 2024 annual meeting of the American Society of Clinical Oncology, in which he and his team assessed outcomes in 75 women with breast cancer who received a GLP-1 agonist. Almost 80% of patients had diabetes, and 60% received hormone therapy, most commonly an aromatase inhibitor. Patients’ median body mass index (BMI) at baseline was 34 kg/m² (range, 23-50 kg/m²).

From baseline, patients lost 6.2 kg, on average, or about 5% of their total body weight, 12 months after initiating GLP-1 therapy. 

In contrast, phase 3 trials show much higher mean weight loss — about two times — in patients without cancer. 

Another recent study also reported modest weight loss results in patients with breast cancer undergoing endocrine therapy. The researchers reported that, at 12 months, Wegovy led to 4.34% reduction in BMI, compared with a 14% change reported in the general population. Zebound, however, was associated with a 2.31% BMI increase overall — though some patients did experience a decrease — compared with a 15% reduction in the general population. 

“These findings indicate a substantially reduced weight loss efficacy in breast cancer patients on endocrine therapy compared to the general population,” the authors concluded.

It’s unclear why the drugs appear to not work as well in patients with cancer. It’s possible that hormone therapy or metabolic changes interfere with their effectiveness, given that some cancer therapies lead to weight gain. Steroids and hormone therapies, for instance, often increase appetite, and some treatments can slow patients’ metabolism or lead to fatigue, which can make it harder to exercise.

Patients with cancer may need a higher dose of GLP-1 agonists to achieve similar weight loss to the general population, Dr. Iyengar noted.

However, Dr. Gallagher said, in her own experience, she hasn’t found the drugs to be less effective in patients with cancer, especially the newer agents, like Wegovy and Zepbound. 

As for safety, Wegovy and Zepbound both carry a black box warning for thyroid C-cell tumors, including medullary thyroid carcinoma. (Recent research, however, has found that GLP-1 agonists do not increase thyroid cancer risk). 

These antiobesity agents are also contraindicated in patients with a personal or family history of medullary thyroid carcinoma and in patients who have multiple endocrine neoplasia syndrome type 2, which is associated with medullary thyroid carcinoma.

Dr. Gallagher hasn’t seen any secondary tumors — thyroid or otherwise — in her patients with cancer, but she follows the labeling contraindications. Dr. Iyengar also noted that more recent and larger data sets have shown no impact on this risk, which may not actually exist, he said

Dr. Gallagher remains cautious about using GPL-1 agonists in patients who have had bariatric surgery because these agents can compound the slower gastric emptying and intestinal transit from surgery, potentially leading to gastrointestinal obstructions. 

Looking ahead, GPL-1 manufacturers are interested in adding cancer indications to the drug labeling. Both Dr. Iyengar and Dr. Gallagher said their institutions are in talks with companies to participate in large, multicenter, global phase 3 trials.

Dr. Iyengar welcomes the efforts, not only to test the effectiveness of GPL-1 agonists in oncology but also to “nail down” their safety in cancer. 

“I don’t think that there’s mechanistically anything that’s particularly worrisome,” and current observations suggest that these drugs are likely to be safe, Dr. Iyengar said. Even so, “GLP-1 agonists do a lot of things that we don’t fully understand yet.”

The bigger challenge, Dr. Iyengar noted, is that companies will have to show a sizable benefit to using these drugs in patients with cancer to get the Food and Drug Administration’s approval. And to move the needle on cancer-specific outcomes, these antiobesity drugs will need to demonstrate significant, durable weight loss in patients with cancer. 

But if these drugs can do that, “I think it’s going to be one of the biggest advances in medicine and oncology given the obesity and cancer epidemic,” Dr. Iyengar said. 

Dr. Iyengar has adviser and/or researcher ties with companies that make or are developing GPL-1 agonists, including AstraZeneca, Novartis, Gilead, and Pfizer. Dr. Gallagher is a consultant for Novartis, Flare Therapeutics, Reactive Biosciences, and Seagen.

A version of this article first appeared on Medscape.com.

Demand for new weight loss drugs has surged over the past few years. 

Led by the antiobesity drugs semaglutide (Wegovy) and tirzepatide (Zepbound), these popular medications — more commonly known as glucagon-like peptide 1 (GLP-1) agonists — have become game changers for shedding excess pounds.

Aside from obesity indications, both drugs have been approved to treat type 2 diabetes under different brand names and have a growing list of other potential benefits, such as reducing inflammation and depression. 

These antiobesity drugs could even have a place in cancer care.

While there’s limited data to support the use of GLP-1 agonists for weight loss in cancer, some oncologists have begun carefully integrating the antiobesity agents into care and studying their effects in this patient population.

The reason: Research suggests that obesity can reduce the effectiveness of cancer therapies, especially in patients with breast cancer, and can increase the risk for treatment-related side effects. 

The idea is that managing patients’ weight will improve their cancer outcomes, explained Lajos Pusztai, MD, PhD, a breast cancer specialist and professor of medicine at Yale School of Medicine in New Haven, Connecticut. 

Although Dr. Pusztai and his oncology peers at Yale don’t yet use GPL-1 agonists, Neil Iyengar, MD, and colleagues have begun doing so to help some patients with breast cancer manage their weight. Dr. Iyengar estimates that a few hundred — almost 40% — of his patients are on the antiobesity drugs.

“For a patient who has really tried to reduce their weight and who is in the obese range, that’s where I think the use of these medications can be considered,” said Dr. Iyengar, a breast cancer oncologist at Memorial Sloan Kettering Cancer Center in New York City. 

Why GLP-1s in Cancer?

GLP-1 is a hormone that the small intestine releases after eating. GLP-1 agonists work by mimicking GLP-1 to trigger the release of insulin and reduce the production of glucagon — two processes that help regulate blood sugar. 

These agents, such as Wegovy (or Ozempic when prescribed for diabetes), also slow gastric emptying and can make people feel fuller longer. 

Zebound (or Mounjaro for type 2 diabetes) is considered a dual GLP-1 and glucose-dependent insulinotropic polypeptide agonist, which may enhance its weight loss benefits.

In practice, however, these drugs can increase nausea and vomiting from chemotherapy, so Dr. Iyengar typically has patients use them afterwards, during maintenance treatment.

Oncologists don’t prescribe the drugs themselves but instead refer patients to endocrinologists or weight management centers that then write the prescriptions. Taking these drugs involves weekly subcutaneous injections patients can administer themselves.

Endocrinologist Emily Gallagher, MD, PhD, of Mount Sinai Hospital in New York City, estimates she has prescribed the antiobesity drugs to a few hundred patients with cancer and, like Dr. Iyengar, uses the drugs during maintenance treatment with hormone therapy for breast cancer. She also has used these agents in patients with prostate and endometrial cancers and has found the drugs can help counter steroid weight gain in multiple myeloma. 

But, to date, the evidence for using GPL-1 agonists in cancer remains limited and the practice has not yet become widespread.

Research largely comes down to a few small retrospective studies in patients with breast cancer receiving aromatase inhibitors. Although no safety issues have emerged so far, these initial reports suggest that the drugs lead to significantly less weight loss in patients with cancer compared to the general population. 

Dr. Iyengar led one recent study, presented at the 2024 annual meeting of the American Society of Clinical Oncology, in which he and his team assessed outcomes in 75 women with breast cancer who received a GLP-1 agonist. Almost 80% of patients had diabetes, and 60% received hormone therapy, most commonly an aromatase inhibitor. Patients’ median body mass index (BMI) at baseline was 34 kg/m² (range, 23-50 kg/m²).

From baseline, patients lost 6.2 kg, on average, or about 5% of their total body weight, 12 months after initiating GLP-1 therapy. 

In contrast, phase 3 trials show much higher mean weight loss — about two times — in patients without cancer. 

Another recent study also reported modest weight loss results in patients with breast cancer undergoing endocrine therapy. The researchers reported that, at 12 months, Wegovy led to 4.34% reduction in BMI, compared with a 14% change reported in the general population. Zebound, however, was associated with a 2.31% BMI increase overall — though some patients did experience a decrease — compared with a 15% reduction in the general population. 

“These findings indicate a substantially reduced weight loss efficacy in breast cancer patients on endocrine therapy compared to the general population,” the authors concluded.

It’s unclear why the drugs appear to not work as well in patients with cancer. It’s possible that hormone therapy or metabolic changes interfere with their effectiveness, given that some cancer therapies lead to weight gain. Steroids and hormone therapies, for instance, often increase appetite, and some treatments can slow patients’ metabolism or lead to fatigue, which can make it harder to exercise.

Patients with cancer may need a higher dose of GLP-1 agonists to achieve similar weight loss to the general population, Dr. Iyengar noted.

However, Dr. Gallagher said, in her own experience, she hasn’t found the drugs to be less effective in patients with cancer, especially the newer agents, like Wegovy and Zepbound. 

As for safety, Wegovy and Zepbound both carry a black box warning for thyroid C-cell tumors, including medullary thyroid carcinoma. (Recent research, however, has found that GLP-1 agonists do not increase thyroid cancer risk). 

These antiobesity agents are also contraindicated in patients with a personal or family history of medullary thyroid carcinoma and in patients who have multiple endocrine neoplasia syndrome type 2, which is associated with medullary thyroid carcinoma.

Dr. Gallagher hasn’t seen any secondary tumors — thyroid or otherwise — in her patients with cancer, but she follows the labeling contraindications. Dr. Iyengar also noted that more recent and larger data sets have shown no impact on this risk, which may not actually exist, he said

Dr. Gallagher remains cautious about using GPL-1 agonists in patients who have had bariatric surgery because these agents can compound the slower gastric emptying and intestinal transit from surgery, potentially leading to gastrointestinal obstructions. 

Looking ahead, GPL-1 manufacturers are interested in adding cancer indications to the drug labeling. Both Dr. Iyengar and Dr. Gallagher said their institutions are in talks with companies to participate in large, multicenter, global phase 3 trials.

Dr. Iyengar welcomes the efforts, not only to test the effectiveness of GPL-1 agonists in oncology but also to “nail down” their safety in cancer. 

“I don’t think that there’s mechanistically anything that’s particularly worrisome,” and current observations suggest that these drugs are likely to be safe, Dr. Iyengar said. Even so, “GLP-1 agonists do a lot of things that we don’t fully understand yet.”

The bigger challenge, Dr. Iyengar noted, is that companies will have to show a sizable benefit to using these drugs in patients with cancer to get the Food and Drug Administration’s approval. And to move the needle on cancer-specific outcomes, these antiobesity drugs will need to demonstrate significant, durable weight loss in patients with cancer. 

But if these drugs can do that, “I think it’s going to be one of the biggest advances in medicine and oncology given the obesity and cancer epidemic,” Dr. Iyengar said. 

Dr. Iyengar has adviser and/or researcher ties with companies that make or are developing GPL-1 agonists, including AstraZeneca, Novartis, Gilead, and Pfizer. Dr. Gallagher is a consultant for Novartis, Flare Therapeutics, Reactive Biosciences, and Seagen.

A version of this article first appeared on Medscape.com.

Late last week, a US Food and Drug Administration (FDA) panel met to discuss whether to limit the use of nivolumab (Opdivo) and pembrolizumab (Keytruda) in patients with unresectable or metastatic esophageal squamous cell carcinoma and human epidermal growth factor receptor 2 (HER2)–negative, microsatellite stable gastric/gastroesophageal adenocarcinoma.

During the meeting, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted in favor of restricting the use of these immunotherapy agents to patients with programmed death-ligand 1 (PD-L1) expression of 1% or higher. 

The agency usually follows the ODAC’s advice.

The FDA had originally approved the two immune checkpoint inhibitors for both indications in combination with chemotherapy, regardless of patients’ PD-L1 status. The FDA had also approved nivolumab in combination with ipilimumab for esophageal cancer, regardless of PD-L1 expression. The approvals were based on overall benefit in intent-to-treat populations, not on specific PD-L1 expression subgroups.

Since then, additional studies — including the agency’s own pooled analyses of the approval trials — have found that overall survival benefits are limited to patients with PD-L1 expression of 1% or higher.

These findings have raised concerns that patients with no or low PD-L1 expression face the risks associated with immunotherapy, which include death, but minimal to no benefits.

In response, the FDA has considered changing the labeling for these indications to require a PD-L1 cutoff point of 1% or higher. The move would mirror guidelines from the American Society of Clinical Oncology and the National Comprehensive Cancer Network that already recommend use with chemotherapy only at certain PD-L1 cutoffs.

Before the agency acts, however, the FDA wanted the advice of the ODAC. It asked the 14-member panel whether the risk-benefit assessment is “favorable for the use of PD-1 inhibitors in first line” for the two indications among patients with PD-L1 expression below 1%.

In two nearly unanimous decisions for each indication, the panel voted that risk-benefit assessment was not favorable. In other words, the risks do outweigh the benefits for this patient population with no or low PD-L1 expression.

The determination also applies to tislelizumab (Tevimbra), an immune checkpoint inhibitor under review by the FDA for the same indications.

Voting came after hours of testimony from FDA scientists and the three drug companies involved in the decisions.

Merck, maker of pembrolizumab, was against any labeling change. Nivolumab’s maker, Bristol Myers Squibb (BMS), also wanted to stick with the current PD-L1 agnostic indications but said that any indication change should be class-wide to avoid confusion. BeiGene USA, maker of tislelizumab, had no problem with a PD-L1 cutoff of 1% because its approval trial showed benefit only in patients at that level or higher.

In general, Merck and BMS said the drug benefits correspond with higher PD-L1 expression but noted that patients with low or no PD-L1 expression can sometimes benefit from treatment. The companies had several patients testify to the benefits of the agents and noted patients like this would likely lose access. But an ODAC panelist noted that patients who died from immunotherapy complications weren’t there to respond.

The companies also expressed concern about taking treatment decisions out of the hands of oncologists as well as the need for additional biopsies to determine if tumors cross the proposed PD-L1 threshold at some point during treatment. With this potential new restriction, the companies were worried that insurance companies would be even less likely to pay for their checkpoint inhibitors in low or no PD-L1 expressors.

ODAC wasn’t moved. With consistent findings across multiple trials, the strength of the FDA’s data carried the day.

In a pooled analysis of the three companies’ unresectable or metastatic HER2–negative, microsatellite-stable gastric/gastroesophageal adenocarcinoma approval trials across almost 4000 patients, those with PD-L1 levels below 1% did not demonstrate a significant overall survival benefit (hazard ratio [HR], 0.91; 95% CI, 0.75-1.09). The median overall survival with immunotherapy plus chemotherapy was only 1 month more — 13.4 months vs 12.4 months with chemotherapy alone.

FDA’s pooled analysis for unresectable or metastatic esophageal squamous cell carcinoma also showed no overall survival benefit (HR, 1.1; 95% CI, 0.76-1.58), with a trend suggesting harm. Median overall survival with immunotherapy plus chemotherapy was 14.6 months vs 9.8 months with chemotherapy alone.

Despite the vote on esophageal squamous cell carcinoma, panelists had reservations about making decisions based on just over 160 patients with PD-L1 levels below 1% in the three esophageal squamous cell carcinoma trials.

Still, one panelist said, it’s likely “the best dataset we will get.”

The companies all used different methods to test PD-L1 levels, and attendees called for a single standardized PD-L1 test. Richard Pazdur, MD, head of the FDA’s Oncology Center of Excellence, said the agency has been working with companies for years to get them to agree to such a test, with no luck.

If the FDA ultimately decides to restrict immunotherapy use in this patient population based on PD-L1 levels, insurance company coverage may become more limited. Pazdur asked the companies if they would be willing to expand their patient assistance programs to provide free coverage of immune checkpoint blockers to patients with low or no PD-L1 expression.

BeiGene and BMS seemed open to the idea. Merck said, “We’ll have to ... think about it.”
 

A version of this article first appeared on Medscape.com.

Late last week, a US Food and Drug Administration (FDA) panel met to discuss whether to limit the use of nivolumab (Opdivo) and pembrolizumab (Keytruda) in patients with unresectable or metastatic esophageal squamous cell carcinoma and human epidermal growth factor receptor 2 (HER2)–negative, microsatellite stable gastric/gastroesophageal adenocarcinoma.

During the meeting, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted in favor of restricting the use of these immunotherapy agents to patients with programmed death-ligand 1 (PD-L1) expression of 1% or higher. 

The agency usually follows the ODAC’s advice.

The FDA had originally approved the two immune checkpoint inhibitors for both indications in combination with chemotherapy, regardless of patients’ PD-L1 status. The FDA had also approved nivolumab in combination with ipilimumab for esophageal cancer, regardless of PD-L1 expression. The approvals were based on overall benefit in intent-to-treat populations, not on specific PD-L1 expression subgroups.

Since then, additional studies — including the agency’s own pooled analyses of the approval trials — have found that overall survival benefits are limited to patients with PD-L1 expression of 1% or higher.

These findings have raised concerns that patients with no or low PD-L1 expression face the risks associated with immunotherapy, which include death, but minimal to no benefits.

In response, the FDA has considered changing the labeling for these indications to require a PD-L1 cutoff point of 1% or higher. The move would mirror guidelines from the American Society of Clinical Oncology and the National Comprehensive Cancer Network that already recommend use with chemotherapy only at certain PD-L1 cutoffs.

Before the agency acts, however, the FDA wanted the advice of the ODAC. It asked the 14-member panel whether the risk-benefit assessment is “favorable for the use of PD-1 inhibitors in first line” for the two indications among patients with PD-L1 expression below 1%.

In two nearly unanimous decisions for each indication, the panel voted that risk-benefit assessment was not favorable. In other words, the risks do outweigh the benefits for this patient population with no or low PD-L1 expression.

The determination also applies to tislelizumab (Tevimbra), an immune checkpoint inhibitor under review by the FDA for the same indications.

Voting came after hours of testimony from FDA scientists and the three drug companies involved in the decisions.

Merck, maker of pembrolizumab, was against any labeling change. Nivolumab’s maker, Bristol Myers Squibb (BMS), also wanted to stick with the current PD-L1 agnostic indications but said that any indication change should be class-wide to avoid confusion. BeiGene USA, maker of tislelizumab, had no problem with a PD-L1 cutoff of 1% because its approval trial showed benefit only in patients at that level or higher.

In general, Merck and BMS said the drug benefits correspond with higher PD-L1 expression but noted that patients with low or no PD-L1 expression can sometimes benefit from treatment. The companies had several patients testify to the benefits of the agents and noted patients like this would likely lose access. But an ODAC panelist noted that patients who died from immunotherapy complications weren’t there to respond.

The companies also expressed concern about taking treatment decisions out of the hands of oncologists as well as the need for additional biopsies to determine if tumors cross the proposed PD-L1 threshold at some point during treatment. With this potential new restriction, the companies were worried that insurance companies would be even less likely to pay for their checkpoint inhibitors in low or no PD-L1 expressors.

ODAC wasn’t moved. With consistent findings across multiple trials, the strength of the FDA’s data carried the day.

In a pooled analysis of the three companies’ unresectable or metastatic HER2–negative, microsatellite-stable gastric/gastroesophageal adenocarcinoma approval trials across almost 4000 patients, those with PD-L1 levels below 1% did not demonstrate a significant overall survival benefit (hazard ratio [HR], 0.91; 95% CI, 0.75-1.09). The median overall survival with immunotherapy plus chemotherapy was only 1 month more — 13.4 months vs 12.4 months with chemotherapy alone.

FDA’s pooled analysis for unresectable or metastatic esophageal squamous cell carcinoma also showed no overall survival benefit (HR, 1.1; 95% CI, 0.76-1.58), with a trend suggesting harm. Median overall survival with immunotherapy plus chemotherapy was 14.6 months vs 9.8 months with chemotherapy alone.

Despite the vote on esophageal squamous cell carcinoma, panelists had reservations about making decisions based on just over 160 patients with PD-L1 levels below 1% in the three esophageal squamous cell carcinoma trials.

Still, one panelist said, it’s likely “the best dataset we will get.”

The companies all used different methods to test PD-L1 levels, and attendees called for a single standardized PD-L1 test. Richard Pazdur, MD, head of the FDA’s Oncology Center of Excellence, said the agency has been working with companies for years to get them to agree to such a test, with no luck.

If the FDA ultimately decides to restrict immunotherapy use in this patient population based on PD-L1 levels, insurance company coverage may become more limited. Pazdur asked the companies if they would be willing to expand their patient assistance programs to provide free coverage of immune checkpoint blockers to patients with low or no PD-L1 expression.

BeiGene and BMS seemed open to the idea. Merck said, “We’ll have to ... think about it.”
 

A version of this article first appeared on Medscape.com.

Late last week, a US Food and Drug Administration (FDA) panel met to discuss whether to limit the use of nivolumab (Opdivo) and pembrolizumab (Keytruda) in patients with unresectable or metastatic esophageal squamous cell carcinoma and human epidermal growth factor receptor 2 (HER2)–negative, microsatellite stable gastric/gastroesophageal adenocarcinoma.

During the meeting, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted in favor of restricting the use of these immunotherapy agents to patients with programmed death-ligand 1 (PD-L1) expression of 1% or higher. 

The agency usually follows the ODAC’s advice.

The FDA had originally approved the two immune checkpoint inhibitors for both indications in combination with chemotherapy, regardless of patients’ PD-L1 status. The FDA had also approved nivolumab in combination with ipilimumab for esophageal cancer, regardless of PD-L1 expression. The approvals were based on overall benefit in intent-to-treat populations, not on specific PD-L1 expression subgroups.

Since then, additional studies — including the agency’s own pooled analyses of the approval trials — have found that overall survival benefits are limited to patients with PD-L1 expression of 1% or higher.

These findings have raised concerns that patients with no or low PD-L1 expression face the risks associated with immunotherapy, which include death, but minimal to no benefits.

In response, the FDA has considered changing the labeling for these indications to require a PD-L1 cutoff point of 1% or higher. The move would mirror guidelines from the American Society of Clinical Oncology and the National Comprehensive Cancer Network that already recommend use with chemotherapy only at certain PD-L1 cutoffs.

Before the agency acts, however, the FDA wanted the advice of the ODAC. It asked the 14-member panel whether the risk-benefit assessment is “favorable for the use of PD-1 inhibitors in first line” for the two indications among patients with PD-L1 expression below 1%.

In two nearly unanimous decisions for each indication, the panel voted that risk-benefit assessment was not favorable. In other words, the risks do outweigh the benefits for this patient population with no or low PD-L1 expression.

The determination also applies to tislelizumab (Tevimbra), an immune checkpoint inhibitor under review by the FDA for the same indications.

Voting came after hours of testimony from FDA scientists and the three drug companies involved in the decisions.

Merck, maker of pembrolizumab, was against any labeling change. Nivolumab’s maker, Bristol Myers Squibb (BMS), also wanted to stick with the current PD-L1 agnostic indications but said that any indication change should be class-wide to avoid confusion. BeiGene USA, maker of tislelizumab, had no problem with a PD-L1 cutoff of 1% because its approval trial showed benefit only in patients at that level or higher.

In general, Merck and BMS said the drug benefits correspond with higher PD-L1 expression but noted that patients with low or no PD-L1 expression can sometimes benefit from treatment. The companies had several patients testify to the benefits of the agents and noted patients like this would likely lose access. But an ODAC panelist noted that patients who died from immunotherapy complications weren’t there to respond.

The companies also expressed concern about taking treatment decisions out of the hands of oncologists as well as the need for additional biopsies to determine if tumors cross the proposed PD-L1 threshold at some point during treatment. With this potential new restriction, the companies were worried that insurance companies would be even less likely to pay for their checkpoint inhibitors in low or no PD-L1 expressors.

ODAC wasn’t moved. With consistent findings across multiple trials, the strength of the FDA’s data carried the day.

In a pooled analysis of the three companies’ unresectable or metastatic HER2–negative, microsatellite-stable gastric/gastroesophageal adenocarcinoma approval trials across almost 4000 patients, those with PD-L1 levels below 1% did not demonstrate a significant overall survival benefit (hazard ratio [HR], 0.91; 95% CI, 0.75-1.09). The median overall survival with immunotherapy plus chemotherapy was only 1 month more — 13.4 months vs 12.4 months with chemotherapy alone.

FDA’s pooled analysis for unresectable or metastatic esophageal squamous cell carcinoma also showed no overall survival benefit (HR, 1.1; 95% CI, 0.76-1.58), with a trend suggesting harm. Median overall survival with immunotherapy plus chemotherapy was 14.6 months vs 9.8 months with chemotherapy alone.

Despite the vote on esophageal squamous cell carcinoma, panelists had reservations about making decisions based on just over 160 patients with PD-L1 levels below 1% in the three esophageal squamous cell carcinoma trials.

Still, one panelist said, it’s likely “the best dataset we will get.”

The companies all used different methods to test PD-L1 levels, and attendees called for a single standardized PD-L1 test. Richard Pazdur, MD, head of the FDA’s Oncology Center of Excellence, said the agency has been working with companies for years to get them to agree to such a test, with no luck.

If the FDA ultimately decides to restrict immunotherapy use in this patient population based on PD-L1 levels, insurance company coverage may become more limited. Pazdur asked the companies if they would be willing to expand their patient assistance programs to provide free coverage of immune checkpoint blockers to patients with low or no PD-L1 expression.

BeiGene and BMS seemed open to the idea. Merck said, “We’ll have to ... think about it.”
 

A version of this article first appeared on Medscape.com.

When Jennifer Welsh, a 40-year-old from New Britain, Connecticut, visited her doctor about pain in her joints and neck, her doctor sent her to the emergency department (ED) to rule out meningitis. The ED did rule that out, as well as strep, so Ms. Welsh went to her follow-up appointment a few days later, hoping for answers or at least more tests to get those answers.

Instead, the doctor — a different one from the same practice as her primary care physician (PCP) — wouldn’t even talk to Ms. Welsh about her symptoms because she couldn’t see the ED’s results and refused to view the results that Ms. Welsh could pull up online.

“She just completely shut me down,” Ms. Welsh recalled. “It was a really awful appointment, and I left in tears. I was in physical pain, I had just been to the ER, nothing is really resolved, I’m stressed out about it, and this woman is completely dismissing me.”

She had been able to schedule an appointment with her regular PCP later that week, but after the harrowing experience with this doctor, she wondered if her PCP would order the rheumatoid arthritis (RA) test that Ms. Welsh suspected she needed. So, she took matters into her own hands.

“I was searching for what test to ask for from my doctor,” she said, and she found that she could order it on her own from a major lab company she was already familiar with. For around $100, “I could get it done and see what it says on my own,” she said.

But that’s not how it worked out. Her regular PCP apologized for the other doctor’s behavior and ordered the RA test as well as additional tests — and got results while Ms. Welsh still waited for the one she ordered to arrive over a week later.

At first, Ms. Welsh was grateful she could order the RA test without her doctor’s referral. “I felt it gave me a sense of control over the situation that I felt really not in control of, until the system failed me, and I didn’t get the results,” she said. But then, “not having someone I could call and get an answer about why my tests were delayed, why I wasn’t able to access them, why it was taking so long — it was definitely anxiety-inducing.”
 

A Growing Market

Ms. Welsh is one of a growing number of patients who are ordering direct-to-consumer (DTC) lab tests without the recommendation or guidance of a doctor. They’re offered online by labs ranging from well-established giants like Quest and Labcorp to smaller, potentially less vetted companies, although some smaller companies contract with larger companies like Quest. Combined, the DTC market is projected to be worth $2 billion by 2025.

Yet the burgeoning industry has also drawn critiques from both bioethicists and privacy experts. A research letter in JAMA in 2023, for example, found that less than half of the 21 companies identified in an online search declared Health Insurance Portability and Accountability Act compliance, while more than half “indicated the potential use of consumer data for research purposes either internally or through third-party sharing.” That study found the most commonly offered tests were related to diabetes, the thyroid, and vitamin levels, and hormone tests for men and women, such as testosterone or estradiol.

But a number of companies also offer tests related to rheumatologic conditions. A handful of tests offered by Labcorp, for example, could be used in rheumatology, such as tests for celiac antibodies or high-sensitivity C-reactive protein. Quest similarly offers a handful of autoimmune-related tests. But other companies offer a long slate of autoimmune or antibody tests.

The antinuclear antibody (ANA) test and RA panel offered by Quest are the same tests, run and analyzed in the same labs, as those ordered by physicians and hospitals, according to James Faix, MD, the medical director of immunology at Quest Diagnostics. Their RA panel includes rheumatoid factor and anti-cyclic citrullinated peptide as well as antibody to mutated citrullinated vimentin, “which may detect approximately 10%-15%” of patients who test negative to the first two.

Quest’s ANA test with reflex costs $112, and its RA panel costs $110, price points that are similar across other companies’ offerings. Labcorp declined to respond to questions about its DTC tests, and several smaller companies did not respond to queries about their offerings. It can therefore be hard to assess what’s included or what the quality is of many DTC tests, particularly from smaller, less established companies.
 

Oversight and Quality Control

Anthony Killeen, MD, PhD, president of the Association for Diagnostics & Laboratory Medicine (ADLM) and director of Clinical Laboratories at the University of Minnesota Medical Center in Minneapolis, said via email that the ADLM supports “expanding consumer access to direct-to-consumer laboratory testing services that have demonstrated analytical and clinical validity and clinical utility,” given the importance of individuals learning about their health status and becoming more involved in health decisions. But the ADLM also recommends “that only CLIA-certified laboratories perform direct-to-consumer testing,” he said.

Association for Diagnostics & Laboratory Medicine

“There are direct-to-consumer tests on the market that are not medical-grade laboratory tests and that may be performed in nonaccredited laboratories,” Dr. Killeen said. “We advise consumers to steer clear of such tests.” The ADLM also encourages consumers to “work with qualified healthcare providers when making decisions based off the results they receive from any direct-to-consumer tests” and recommends that DTC test companies “provide consumers with sufficient information and/or access to expert help to assist them in ordering tests and interpreting the results.”

Yet it’s unclear how much support, if any, consumers can receive in terms of understanding what their tests mean. Most of the companies in the 2023 study offered optional follow-up with a healthcare professional, but these professionals ranged from physicians to “health coaches,” and all the companies had disclaimers that “test results did not constitute medical advice.”

At Quest, the only company to respond to this news organization’s request for comment, consumer-initiated tests ordered online are first reviewed by a physician at PWNHealth, an independent, third-party physician network, to determine that it’s appropriate before the lab order is actually placed.

“Once results are available, individuals have the option to discuss their results with an independent physician at no extra cost,” Dr. Faix said. ANA or RA results outside the normal ranges may trigger a “call from a PWNHealth healthcare coordinator, who can help provide information, suggestions on next steps, and set up time for the individual to speak with an independent physician to discuss questions or concerns regarding the results,” he said.

“Our goal is not to replace the role of a healthcare provider,” Dr. Faix said. “We are providing an alternate way for people to engage with the healthcare system that offers convenience, gives people more control over their own healthcare journeys, and meets them where they are, supporting both consumers and their care teams.” The company has expanded its offerings from an initial 30 tests made available in 2018 to over 130 today, deciding which to offer “based on consumer research and expertise of clinical experts.” The company has also “seen steady interest in our two consumer rheumatology offerings,” Dr. Faix said.
 

The DTC Landscape in Rheumatology

Within rheumatology, among the most popular tests is for ANA, based on the experience of Alfred Kim, MD, PhD, associate professor of medicine at Washington University School of Medicine in St Louis, Missouri.

Dr. Kim

“For a lot of people, losing control over their health is maybe the most frightening experience they can have, so I think a lot of patients use this as a way to kind of have ownership over their health,” Dr. Kim said. “Let’s say they’ve been to four doctors. No one can explain what’s going on. They’re getting frustrated, and so they just turn to solutions where they feel like they have ownership over the situation.”

Though the market is undoubtedly growing, the growth appears uneven across geography and institution types. Kim has seen a “fair number of referrals,” with patients coming in with results from a DTC test. Michael Putman, MD, MSci, assistant professor of medicine at the Medical College of Wisconsin in Milwaukee, hasn’t seen it much. “I know that patients can get testing done themselves independently, but I don’t have people routinely coming in with tests they’ve ordered in advance of our appointment,” Dr. Putman said, but, like Dr. Kim, he recognizes why patients might seek them out.

Dr. Putman

Ron Hester Photography

Risks vs Benefits

DTC testing is not an answer to the national shortage of rheumatologists, however, especially given the risks that Dr. Niewold, Dr. Putman, and Dr. Kim worry outweigh potential benefits. On the one hand, getting online test results may help expedite a referral to a specialist, Dr. Niewold said. But a long wait for that appointment could then easily become a bigger source of anxiety than comfort, Dr. Putman said.

“It’s a trade-off where you are accepting a lot more people getting false-positive diagnoses and spending months thinking they have some disease where they might not, in exchange for a couple people who would have had a delayed diagnosis,” Dr. Putman said. “There’s an enormous amount of existential suffering,” that’s familiar to rheumatologists because some patients may dread the diagnosis of a rheumatic disease the way they might fear a cancer diagnosis, especially if they have lost a family member to a condition that they suspect they share, he said. “To put yourself into an existential catastrophe — that’s not a small harm.”

Dr. Niewold agreed, pointing out that patients with a positive ANA test may “get unnecessarily worried and stay up all night reading about lupus, getting scared for weeks on end before seeing a specialist.” And there are financial harms as well for patients who may order the same test multiple times, or a whole slate of tests, that they don’t need for hundreds or thousands of dollars. There’s also the lost time and effort of researching a condition or even seeking out support groups that patients may pursue, Dr. Niewold said.

The likely biggest risk to individuals, however, is the potential for overdiagnosis or misdiagnosis.

“If someone comes in and they’ve read the textbook on lupus and they have a positive ANA, it’s really hard as a rheumatologist to walk that back,” Dr. Putman said. “The human mind is a powerful thing,” he added, and people who get a positive test will likely start to notice things like joint pain or a rash on their cheeks and begin attributing it to a diagnosis they risk convincing themselves they have. “When people come into your clinic not knowing what a disease would look like and they just tell you how they’re feeling, it’s a much cleaner and more honest way to approach diagnosis.”

Most patients likely don’t realize, for example, that none of the tests rheumatologists usually order are diagnostic in and of themselves, Dr. Niewold said. “They’re all kind of like stars in the constellation of a diagnosis,” he said. “They’re helpful, but none of them is sufficient by itself.”

Dr. Killeen agreed, noting that “consumers might not understand the nuances of these tests well enough to know whether it is appropriate to order them or how to interpret the results correctly.” Given the long-term implications of a diagnosis for a rheumatologic disease, “I would have concerns about consumers ordering and interpreting rheumatologic tests without working closely with their physicians,” Dr. Killeen said. “The main concern that lab experts have about direct-to-consumer tests is the potential for people to get misleading results and/or to misinterpret their results, which in turn could lead to people not getting the treatment they need or getting treatment when they don’t need any at all.”

It’s one thing for patients to come in asking for a particular treatment they may not need but which a doctor may be able to dissuade them from seeking. But Dr. Kim also pointed out the risk that patients may decide to treat themselves with therapies that haven’t undergone rigorous testing or haven’t been recommended by a physician.

“We tend to have people who come in with a pretty clear idea of what they want done, but the problem is, we don’t know if their reasoning is correct from a clinical perspective,” Dr. Kim said. Companies offer these tests with the belief that they’re “providing patients a choice, an option to take ownership,” he said, “but the potential harm can be realized very quickly because there are going to be people who are misdiagnosing themselves and, worse yet, may then pursue their own treatment plan that’s going in the opposite direction of where we think it needs to go.”

Or, on the flip side, if a patient erroneously believes they have the answer to what ails them, it may delay diagnosis of a more serious condition that’s rarer or harder to detect. Kim pointed to, for example, intravascular lymphoma, which is notoriously as difficult to identify as it is rare and aggressive. If a patient’s confirmation bias has led them to believe they have an autoimmune condition, they may not receive the more serious diagnosis until it’s advanced too far to treat.
 

Patient-Provider Relationship Friction

Another concern is how these tests may lead to confusion and frustration that can erode the patient-provider relationship, particularly because most patients don’t know how to interpret the results or understand the bigger context in which the results have to be interpreted. Many patients may think a test can come back with a binary answer, a positive or negative, and that means they do or don’t have a condition. That’s generally true for pregnancy tests, COVID tests, and sexually transmitted infection tests — the kinds of tests that have long been available to consumers and which have fairly straightforward answers.

But physicians know that’s not the case for many conditions, particularly those in rheumatology.

“In rheumatic diseases, because the tests have such marginal value in terms of diagnosis, almost always we develop a suspicion before we even think about ordering the tests, and then that dictates whether or not we cross that threshold,” Dr. Kim said. “A negative test doesn’t exclude the fact that you may have disease X, but a positive test also doesn’t mean you have disease X. All they provide is an idea of the risk.”

But some patients who come in with DTC test results have “already made the decision in their mind that they have a certain condition,” Dr. Kim said. “This is obviously dangerous because the majority of these patients do not have the condition they think they have, and it leaves a very uncomfortable feeling after the visit because they feel like they’ve been either betrayed by me or by the test, and they leave more confused.”

Patients may also come in with tests that a doctor isn’t familiar with or isn’t sure how to interpret on its own, at least for that particular patient.

“For ANA testing, we have a pretty good idea of its positive and negative predictive value because it’s ordered so much, but for many of these tests being offered, there are specific autoantibodies, and we tend to only get them in people where there’s a clinical suspicion,” Dr. Kim said. “Within that very specific context, we kind of understand what that value means, but if you give it to the general public, then those numbers aren’t as applicable and most likely overestimate the risk of disease.”

Even if providers consider the results of a DTC test in their differential, they may want to be sure it’s from a trustworthy source. “If a provider is uncertain about whether a direct-to-consumer testing company is reputable or about whether a direct-to-consumer test result is reliable, I would encourage them to consult with their laboratory medicine colleagues,” Dr. Killeen said.
 

Responding to Patients

Like any other patient coming to a clinical visit, the most common reason patients are likely ordering these tests is that they’re seeking answers. Kim doesn’t typically see patients doing their own monitoring for diagnosed conditions between visits — the expense would add up too quickly — or testing for genetic markers, which likely wouldn’t be very helpful either.

“Even though most of our diseases probably have a genetic underpinning, how much it contributes is always unclear,” Dr. Kim said. Even conditions with clear genetic variants, such as familial Mediterranean fever, spondyloarthritis, and Behçet disease, can only support a diagnosis, not diagnose it on its own, Dr. Killeen said. And these are not among the tests currently available on most DTC company sites.

While there are also tests that can offer information about genetic risks for certain medications, such as a thiopurine methyltransferase test to find out if a patient lacks the enzyme needed to break down the immunosuppressant drug azathioprine, Kim hasn’t seen patients seeking these out either.

“The more global and more compassionate way to think about this is that we have a lot of people who are struggling to understand what’s going on with their bodies, and most physicians really don’t know what the next steps are for these people,” Dr. Kim said. “They’re desperate, and their quality of life is so poor that they’re going to take extreme steps to try to manage their own frustration with this condition.”

That means clinicians’ most powerful tools when patients come in with DTC test results are their listening skills.

“Empathy is the most important thing, just being able to share the patient’s frustration to the point where they had to take matters into their own hands,” Dr. Kim said. “I think a lot of rheumatologists are actually pretty comfortable being in this position.”

Additionally, doctors should know that some patients may be engaging in attempts to self-diagnose, self-treat, or otherwise self-manage their symptoms or perceived condition. “They just need to be aware and try to make sure there’s no harm being done,” Dr. Kim said.

Ms. Welsh didn’t seek treatment or diagnosis on her own, but getting her test also did not give her the control she was seeking. “Looking back, it was kind of a waste of money, but it felt good in the moment,” Ms. Welsh said. “I was so upset, and I wanted that control, and in the end, it didn’t get me results any sooner, and it didn’t give me peace of mind.”

It was Ms. Welsh’s primary care doctor listening to her concerns, ordering the same test she had ordered with several others, and working with her to seek answers that reassured her that her provider cared about her well-being.

“A lot of what I do in my business is reassure people that you know what they have is treatable or not going to end their life as they know it,” Dr. Putman said. “And you certainly can’t reassure them if they’re not in your clinic yet.”

Dr. Putman has participated in clinical trials with AbbVie, consulting with Novartis and GSK, and clinical trials and consulting with Amgen and AstraZeneca. Dr. Niewold reported receiving research grants from EMD Serono and Zenas BioPharma and consulting for Thermo Fisher Scientific, Progentec Diagnostics, Roivant Sciences, AstraZeneca, S3 Connected Health, Flagship Pioneering, and Guidepoint. Dr. Kim reported sponsored research agreements with AstraZeneca, Bristol-Myers Squibb, Novartis, and CRISPR Therapeutics; royalties from Kypha; and consulting/speaking for Amgen, ANI Pharmaceuticals, Atara Biotherapeutics, Aurinia Pharmaceuticals, CARGO Therapeutics, Exagen Diagnostics, GSK, Hinge Bio, Kypha, Progentec Diagnostics, Synthekine, and UpToDate. Dr. Killeen had no relevant financial relationships to disclose.
 

A version of this article first appeared on Medscape.com.

When Jennifer Welsh, a 40-year-old from New Britain, Connecticut, visited her doctor about pain in her joints and neck, her doctor sent her to the emergency department (ED) to rule out meningitis. The ED did rule that out, as well as strep, so Ms. Welsh went to her follow-up appointment a few days later, hoping for answers or at least more tests to get those answers.

Instead, the doctor — a different one from the same practice as her primary care physician (PCP) — wouldn’t even talk to Ms. Welsh about her symptoms because she couldn’t see the ED’s results and refused to view the results that Ms. Welsh could pull up online.

“She just completely shut me down,” Ms. Welsh recalled. “It was a really awful appointment, and I left in tears. I was in physical pain, I had just been to the ER, nothing is really resolved, I’m stressed out about it, and this woman is completely dismissing me.”

She had been able to schedule an appointment with her regular PCP later that week, but after the harrowing experience with this doctor, she wondered if her PCP would order the rheumatoid arthritis (RA) test that Ms. Welsh suspected she needed. So, she took matters into her own hands.

“I was searching for what test to ask for from my doctor,” she said, and she found that she could order it on her own from a major lab company she was already familiar with. For around $100, “I could get it done and see what it says on my own,” she said.

But that’s not how it worked out. Her regular PCP apologized for the other doctor’s behavior and ordered the RA test as well as additional tests — and got results while Ms. Welsh still waited for the one she ordered to arrive over a week later.

At first, Ms. Welsh was grateful she could order the RA test without her doctor’s referral. “I felt it gave me a sense of control over the situation that I felt really not in control of, until the system failed me, and I didn’t get the results,” she said. But then, “not having someone I could call and get an answer about why my tests were delayed, why I wasn’t able to access them, why it was taking so long — it was definitely anxiety-inducing.”
 

A Growing Market

Ms. Welsh is one of a growing number of patients who are ordering direct-to-consumer (DTC) lab tests without the recommendation or guidance of a doctor. They’re offered online by labs ranging from well-established giants like Quest and Labcorp to smaller, potentially less vetted companies, although some smaller companies contract with larger companies like Quest. Combined, the DTC market is projected to be worth $2 billion by 2025.

Yet the burgeoning industry has also drawn critiques from both bioethicists and privacy experts. A research letter in JAMA in 2023, for example, found that less than half of the 21 companies identified in an online search declared Health Insurance Portability and Accountability Act compliance, while more than half “indicated the potential use of consumer data for research purposes either internally or through third-party sharing.” That study found the most commonly offered tests were related to diabetes, the thyroid, and vitamin levels, and hormone tests for men and women, such as testosterone or estradiol.

But a number of companies also offer tests related to rheumatologic conditions. A handful of tests offered by Labcorp, for example, could be used in rheumatology, such as tests for celiac antibodies or high-sensitivity C-reactive protein. Quest similarly offers a handful of autoimmune-related tests. But other companies offer a long slate of autoimmune or antibody tests.

The antinuclear antibody (ANA) test and RA panel offered by Quest are the same tests, run and analyzed in the same labs, as those ordered by physicians and hospitals, according to James Faix, MD, the medical director of immunology at Quest Diagnostics. Their RA panel includes rheumatoid factor and anti-cyclic citrullinated peptide as well as antibody to mutated citrullinated vimentin, “which may detect approximately 10%-15%” of patients who test negative to the first two.

Quest’s ANA test with reflex costs $112, and its RA panel costs $110, price points that are similar across other companies’ offerings. Labcorp declined to respond to questions about its DTC tests, and several smaller companies did not respond to queries about their offerings. It can therefore be hard to assess what’s included or what the quality is of many DTC tests, particularly from smaller, less established companies.
 

Oversight and Quality Control

Anthony Killeen, MD, PhD, president of the Association for Diagnostics & Laboratory Medicine (ADLM) and director of Clinical Laboratories at the University of Minnesota Medical Center in Minneapolis, said via email that the ADLM supports “expanding consumer access to direct-to-consumer laboratory testing services that have demonstrated analytical and clinical validity and clinical utility,” given the importance of individuals learning about their health status and becoming more involved in health decisions. But the ADLM also recommends “that only CLIA-certified laboratories perform direct-to-consumer testing,” he said.

Association for Diagnostics & Laboratory Medicine

“There are direct-to-consumer tests on the market that are not medical-grade laboratory tests and that may be performed in nonaccredited laboratories,” Dr. Killeen said. “We advise consumers to steer clear of such tests.” The ADLM also encourages consumers to “work with qualified healthcare providers when making decisions based off the results they receive from any direct-to-consumer tests” and recommends that DTC test companies “provide consumers with sufficient information and/or access to expert help to assist them in ordering tests and interpreting the results.”

Yet it’s unclear how much support, if any, consumers can receive in terms of understanding what their tests mean. Most of the companies in the 2023 study offered optional follow-up with a healthcare professional, but these professionals ranged from physicians to “health coaches,” and all the companies had disclaimers that “test results did not constitute medical advice.”

At Quest, the only company to respond to this news organization’s request for comment, consumer-initiated tests ordered online are first reviewed by a physician at PWNHealth, an independent, third-party physician network, to determine that it’s appropriate before the lab order is actually placed.

“Once results are available, individuals have the option to discuss their results with an independent physician at no extra cost,” Dr. Faix said. ANA or RA results outside the normal ranges may trigger a “call from a PWNHealth healthcare coordinator, who can help provide information, suggestions on next steps, and set up time for the individual to speak with an independent physician to discuss questions or concerns regarding the results,” he said.

“Our goal is not to replace the role of a healthcare provider,” Dr. Faix said. “We are providing an alternate way for people to engage with the healthcare system that offers convenience, gives people more control over their own healthcare journeys, and meets them where they are, supporting both consumers and their care teams.” The company has expanded its offerings from an initial 30 tests made available in 2018 to over 130 today, deciding which to offer “based on consumer research and expertise of clinical experts.” The company has also “seen steady interest in our two consumer rheumatology offerings,” Dr. Faix said.
 

The DTC Landscape in Rheumatology

Within rheumatology, among the most popular tests is for ANA, based on the experience of Alfred Kim, MD, PhD, associate professor of medicine at Washington University School of Medicine in St Louis, Missouri.

Dr. Kim

“For a lot of people, losing control over their health is maybe the most frightening experience they can have, so I think a lot of patients use this as a way to kind of have ownership over their health,” Dr. Kim said. “Let’s say they’ve been to four doctors. No one can explain what’s going on. They’re getting frustrated, and so they just turn to solutions where they feel like they have ownership over the situation.”

Though the market is undoubtedly growing, the growth appears uneven across geography and institution types. Kim has seen a “fair number of referrals,” with patients coming in with results from a DTC test. Michael Putman, MD, MSci, assistant professor of medicine at the Medical College of Wisconsin in Milwaukee, hasn’t seen it much. “I know that patients can get testing done themselves independently, but I don’t have people routinely coming in with tests they’ve ordered in advance of our appointment,” Dr. Putman said, but, like Dr. Kim, he recognizes why patients might seek them out.

Dr. Putman

Ron Hester Photography

Risks vs Benefits

DTC testing is not an answer to the national shortage of rheumatologists, however, especially given the risks that Dr. Niewold, Dr. Putman, and Dr. Kim worry outweigh potential benefits. On the one hand, getting online test results may help expedite a referral to a specialist, Dr. Niewold said. But a long wait for that appointment could then easily become a bigger source of anxiety than comfort, Dr. Putman said.

“It’s a trade-off where you are accepting a lot more people getting false-positive diagnoses and spending months thinking they have some disease where they might not, in exchange for a couple people who would have had a delayed diagnosis,” Dr. Putman said. “There’s an enormous amount of existential suffering,” that’s familiar to rheumatologists because some patients may dread the diagnosis of a rheumatic disease the way they might fear a cancer diagnosis, especially if they have lost a family member to a condition that they suspect they share, he said. “To put yourself into an existential catastrophe — that’s not a small harm.”

Dr. Niewold agreed, pointing out that patients with a positive ANA test may “get unnecessarily worried and stay up all night reading about lupus, getting scared for weeks on end before seeing a specialist.” And there are financial harms as well for patients who may order the same test multiple times, or a whole slate of tests, that they don’t need for hundreds or thousands of dollars. There’s also the lost time and effort of researching a condition or even seeking out support groups that patients may pursue, Dr. Niewold said.

The likely biggest risk to individuals, however, is the potential for overdiagnosis or misdiagnosis.

“If someone comes in and they’ve read the textbook on lupus and they have a positive ANA, it’s really hard as a rheumatologist to walk that back,” Dr. Putman said. “The human mind is a powerful thing,” he added, and people who get a positive test will likely start to notice things like joint pain or a rash on their cheeks and begin attributing it to a diagnosis they risk convincing themselves they have. “When people come into your clinic not knowing what a disease would look like and they just tell you how they’re feeling, it’s a much cleaner and more honest way to approach diagnosis.”

Most patients likely don’t realize, for example, that none of the tests rheumatologists usually order are diagnostic in and of themselves, Dr. Niewold said. “They’re all kind of like stars in the constellation of a diagnosis,” he said. “They’re helpful, but none of them is sufficient by itself.”

Dr. Killeen agreed, noting that “consumers might not understand the nuances of these tests well enough to know whether it is appropriate to order them or how to interpret the results correctly.” Given the long-term implications of a diagnosis for a rheumatologic disease, “I would have concerns about consumers ordering and interpreting rheumatologic tests without working closely with their physicians,” Dr. Killeen said. “The main concern that lab experts have about direct-to-consumer tests is the potential for people to get misleading results and/or to misinterpret their results, which in turn could lead to people not getting the treatment they need or getting treatment when they don’t need any at all.”

It’s one thing for patients to come in asking for a particular treatment they may not need but which a doctor may be able to dissuade them from seeking. But Dr. Kim also pointed out the risk that patients may decide to treat themselves with therapies that haven’t undergone rigorous testing or haven’t been recommended by a physician.

“We tend to have people who come in with a pretty clear idea of what they want done, but the problem is, we don’t know if their reasoning is correct from a clinical perspective,” Dr. Kim said. Companies offer these tests with the belief that they’re “providing patients a choice, an option to take ownership,” he said, “but the potential harm can be realized very quickly because there are going to be people who are misdiagnosing themselves and, worse yet, may then pursue their own treatment plan that’s going in the opposite direction of where we think it needs to go.”

Or, on the flip side, if a patient erroneously believes they have the answer to what ails them, it may delay diagnosis of a more serious condition that’s rarer or harder to detect. Kim pointed to, for example, intravascular lymphoma, which is notoriously as difficult to identify as it is rare and aggressive. If a patient’s confirmation bias has led them to believe they have an autoimmune condition, they may not receive the more serious diagnosis until it’s advanced too far to treat.
 

Patient-Provider Relationship Friction

Another concern is how these tests may lead to confusion and frustration that can erode the patient-provider relationship, particularly because most patients don’t know how to interpret the results or understand the bigger context in which the results have to be interpreted. Many patients may think a test can come back with a binary answer, a positive or negative, and that means they do or don’t have a condition. That’s generally true for pregnancy tests, COVID tests, and sexually transmitted infection tests — the kinds of tests that have long been available to consumers and which have fairly straightforward answers.

But physicians know that’s not the case for many conditions, particularly those in rheumatology.

“In rheumatic diseases, because the tests have such marginal value in terms of diagnosis, almost always we develop a suspicion before we even think about ordering the tests, and then that dictates whether or not we cross that threshold,” Dr. Kim said. “A negative test doesn’t exclude the fact that you may have disease X, but a positive test also doesn’t mean you have disease X. All they provide is an idea of the risk.”

But some patients who come in with DTC test results have “already made the decision in their mind that they have a certain condition,” Dr. Kim said. “This is obviously dangerous because the majority of these patients do not have the condition they think they have, and it leaves a very uncomfortable feeling after the visit because they feel like they’ve been either betrayed by me or by the test, and they leave more confused.”

Patients may also come in with tests that a doctor isn’t familiar with or isn’t sure how to interpret on its own, at least for that particular patient.

“For ANA testing, we have a pretty good idea of its positive and negative predictive value because it’s ordered so much, but for many of these tests being offered, there are specific autoantibodies, and we tend to only get them in people where there’s a clinical suspicion,” Dr. Kim said. “Within that very specific context, we kind of understand what that value means, but if you give it to the general public, then those numbers aren’t as applicable and most likely overestimate the risk of disease.”

Even if providers consider the results of a DTC test in their differential, they may want to be sure it’s from a trustworthy source. “If a provider is uncertain about whether a direct-to-consumer testing company is reputable or about whether a direct-to-consumer test result is reliable, I would encourage them to consult with their laboratory medicine colleagues,” Dr. Killeen said.
 

Responding to Patients

Like any other patient coming to a clinical visit, the most common reason patients are likely ordering these tests is that they’re seeking answers. Kim doesn’t typically see patients doing their own monitoring for diagnosed conditions between visits — the expense would add up too quickly — or testing for genetic markers, which likely wouldn’t be very helpful either.

“Even though most of our diseases probably have a genetic underpinning, how much it contributes is always unclear,” Dr. Kim said. Even conditions with clear genetic variants, such as familial Mediterranean fever, spondyloarthritis, and Behçet disease, can only support a diagnosis, not diagnose it on its own, Dr. Killeen said. And these are not among the tests currently available on most DTC company sites.

While there are also tests that can offer information about genetic risks for certain medications, such as a thiopurine methyltransferase test to find out if a patient lacks the enzyme needed to break down the immunosuppressant drug azathioprine, Kim hasn’t seen patients seeking these out either.

“The more global and more compassionate way to think about this is that we have a lot of people who are struggling to understand what’s going on with their bodies, and most physicians really don’t know what the next steps are for these people,” Dr. Kim said. “They’re desperate, and their quality of life is so poor that they’re going to take extreme steps to try to manage their own frustration with this condition.”

That means clinicians’ most powerful tools when patients come in with DTC test results are their listening skills.

“Empathy is the most important thing, just being able to share the patient’s frustration to the point where they had to take matters into their own hands,” Dr. Kim said. “I think a lot of rheumatologists are actually pretty comfortable being in this position.”

Additionally, doctors should know that some patients may be engaging in attempts to self-diagnose, self-treat, or otherwise self-manage their symptoms or perceived condition. “They just need to be aware and try to make sure there’s no harm being done,” Dr. Kim said.

Ms. Welsh didn’t seek treatment or diagnosis on her own, but getting her test also did not give her the control she was seeking. “Looking back, it was kind of a waste of money, but it felt good in the moment,” Ms. Welsh said. “I was so upset, and I wanted that control, and in the end, it didn’t get me results any sooner, and it didn’t give me peace of mind.”

It was Ms. Welsh’s primary care doctor listening to her concerns, ordering the same test she had ordered with several others, and working with her to seek answers that reassured her that her provider cared about her well-being.

“A lot of what I do in my business is reassure people that you know what they have is treatable or not going to end their life as they know it,” Dr. Putman said. “And you certainly can’t reassure them if they’re not in your clinic yet.”

Dr. Putman has participated in clinical trials with AbbVie, consulting with Novartis and GSK, and clinical trials and consulting with Amgen and AstraZeneca. Dr. Niewold reported receiving research grants from EMD Serono and Zenas BioPharma and consulting for Thermo Fisher Scientific, Progentec Diagnostics, Roivant Sciences, AstraZeneca, S3 Connected Health, Flagship Pioneering, and Guidepoint. Dr. Kim reported sponsored research agreements with AstraZeneca, Bristol-Myers Squibb, Novartis, and CRISPR Therapeutics; royalties from Kypha; and consulting/speaking for Amgen, ANI Pharmaceuticals, Atara Biotherapeutics, Aurinia Pharmaceuticals, CARGO Therapeutics, Exagen Diagnostics, GSK, Hinge Bio, Kypha, Progentec Diagnostics, Synthekine, and UpToDate. Dr. Killeen had no relevant financial relationships to disclose.
 

A version of this article first appeared on Medscape.com.

When Jennifer Welsh, a 40-year-old from New Britain, Connecticut, visited her doctor about pain in her joints and neck, her doctor sent her to the emergency department (ED) to rule out meningitis. The ED did rule that out, as well as strep, so Ms. Welsh went to her follow-up appointment a few days later, hoping for answers or at least more tests to get those answers.

Instead, the doctor — a different one from the same practice as her primary care physician (PCP) — wouldn’t even talk to Ms. Welsh about her symptoms because she couldn’t see the ED’s results and refused to view the results that Ms. Welsh could pull up online.

“She just completely shut me down,” Ms. Welsh recalled. “It was a really awful appointment, and I left in tears. I was in physical pain, I had just been to the ER, nothing is really resolved, I’m stressed out about it, and this woman is completely dismissing me.”

She had been able to schedule an appointment with her regular PCP later that week, but after the harrowing experience with this doctor, she wondered if her PCP would order the rheumatoid arthritis (RA) test that Ms. Welsh suspected she needed. So, she took matters into her own hands.

“I was searching for what test to ask for from my doctor,” she said, and she found that she could order it on her own from a major lab company she was already familiar with. For around $100, “I could get it done and see what it says on my own,” she said.

But that’s not how it worked out. Her regular PCP apologized for the other doctor’s behavior and ordered the RA test as well as additional tests — and got results while Ms. Welsh still waited for the one she ordered to arrive over a week later.

At first, Ms. Welsh was grateful she could order the RA test without her doctor’s referral. “I felt it gave me a sense of control over the situation that I felt really not in control of, until the system failed me, and I didn’t get the results,” she said. But then, “not having someone I could call and get an answer about why my tests were delayed, why I wasn’t able to access them, why it was taking so long — it was definitely anxiety-inducing.”
 

A Growing Market

Ms. Welsh is one of a growing number of patients who are ordering direct-to-consumer (DTC) lab tests without the recommendation or guidance of a doctor. They’re offered online by labs ranging from well-established giants like Quest and Labcorp to smaller, potentially less vetted companies, although some smaller companies contract with larger companies like Quest. Combined, the DTC market is projected to be worth $2 billion by 2025.

Yet the burgeoning industry has also drawn critiques from both bioethicists and privacy experts. A research letter in JAMA in 2023, for example, found that less than half of the 21 companies identified in an online search declared Health Insurance Portability and Accountability Act compliance, while more than half “indicated the potential use of consumer data for research purposes either internally or through third-party sharing.” That study found the most commonly offered tests were related to diabetes, the thyroid, and vitamin levels, and hormone tests for men and women, such as testosterone or estradiol.

But a number of companies also offer tests related to rheumatologic conditions. A handful of tests offered by Labcorp, for example, could be used in rheumatology, such as tests for celiac antibodies or high-sensitivity C-reactive protein. Quest similarly offers a handful of autoimmune-related tests. But other companies offer a long slate of autoimmune or antibody tests.

The antinuclear antibody (ANA) test and RA panel offered by Quest are the same tests, run and analyzed in the same labs, as those ordered by physicians and hospitals, according to James Faix, MD, the medical director of immunology at Quest Diagnostics. Their RA panel includes rheumatoid factor and anti-cyclic citrullinated peptide as well as antibody to mutated citrullinated vimentin, “which may detect approximately 10%-15%” of patients who test negative to the first two.

Quest’s ANA test with reflex costs $112, and its RA panel costs $110, price points that are similar across other companies’ offerings. Labcorp declined to respond to questions about its DTC tests, and several smaller companies did not respond to queries about their offerings. It can therefore be hard to assess what’s included or what the quality is of many DTC tests, particularly from smaller, less established companies.
 

Oversight and Quality Control

Anthony Killeen, MD, PhD, president of the Association for Diagnostics & Laboratory Medicine (ADLM) and director of Clinical Laboratories at the University of Minnesota Medical Center in Minneapolis, said via email that the ADLM supports “expanding consumer access to direct-to-consumer laboratory testing services that have demonstrated analytical and clinical validity and clinical utility,” given the importance of individuals learning about their health status and becoming more involved in health decisions. But the ADLM also recommends “that only CLIA-certified laboratories perform direct-to-consumer testing,” he said.

Association for Diagnostics & Laboratory Medicine

“There are direct-to-consumer tests on the market that are not medical-grade laboratory tests and that may be performed in nonaccredited laboratories,” Dr. Killeen said. “We advise consumers to steer clear of such tests.” The ADLM also encourages consumers to “work with qualified healthcare providers when making decisions based off the results they receive from any direct-to-consumer tests” and recommends that DTC test companies “provide consumers with sufficient information and/or access to expert help to assist them in ordering tests and interpreting the results.”

Yet it’s unclear how much support, if any, consumers can receive in terms of understanding what their tests mean. Most of the companies in the 2023 study offered optional follow-up with a healthcare professional, but these professionals ranged from physicians to “health coaches,” and all the companies had disclaimers that “test results did not constitute medical advice.”

At Quest, the only company to respond to this news organization’s request for comment, consumer-initiated tests ordered online are first reviewed by a physician at PWNHealth, an independent, third-party physician network, to determine that it’s appropriate before the lab order is actually placed.

“Once results are available, individuals have the option to discuss their results with an independent physician at no extra cost,” Dr. Faix said. ANA or RA results outside the normal ranges may trigger a “call from a PWNHealth healthcare coordinator, who can help provide information, suggestions on next steps, and set up time for the individual to speak with an independent physician to discuss questions or concerns regarding the results,” he said.

“Our goal is not to replace the role of a healthcare provider,” Dr. Faix said. “We are providing an alternate way for people to engage with the healthcare system that offers convenience, gives people more control over their own healthcare journeys, and meets them where they are, supporting both consumers and their care teams.” The company has expanded its offerings from an initial 30 tests made available in 2018 to over 130 today, deciding which to offer “based on consumer research and expertise of clinical experts.” The company has also “seen steady interest in our two consumer rheumatology offerings,” Dr. Faix said.
 

The DTC Landscape in Rheumatology

Within rheumatology, among the most popular tests is for ANA, based on the experience of Alfred Kim, MD, PhD, associate professor of medicine at Washington University School of Medicine in St Louis, Missouri.

Dr. Kim

“For a lot of people, losing control over their health is maybe the most frightening experience they can have, so I think a lot of patients use this as a way to kind of have ownership over their health,” Dr. Kim said. “Let’s say they’ve been to four doctors. No one can explain what’s going on. They’re getting frustrated, and so they just turn to solutions where they feel like they have ownership over the situation.”

Though the market is undoubtedly growing, the growth appears uneven across geography and institution types. Kim has seen a “fair number of referrals,” with patients coming in with results from a DTC test. Michael Putman, MD, MSci, assistant professor of medicine at the Medical College of Wisconsin in Milwaukee, hasn’t seen it much. “I know that patients can get testing done themselves independently, but I don’t have people routinely coming in with tests they’ve ordered in advance of our appointment,” Dr. Putman said, but, like Dr. Kim, he recognizes why patients might seek them out.

Dr. Putman

Ron Hester Photography

Risks vs Benefits

DTC testing is not an answer to the national shortage of rheumatologists, however, especially given the risks that Dr. Niewold, Dr. Putman, and Dr. Kim worry outweigh potential benefits. On the one hand, getting online test results may help expedite a referral to a specialist, Dr. Niewold said. But a long wait for that appointment could then easily become a bigger source of anxiety than comfort, Dr. Putman said.

“It’s a trade-off where you are accepting a lot more people getting false-positive diagnoses and spending months thinking they have some disease where they might not, in exchange for a couple people who would have had a delayed diagnosis,” Dr. Putman said. “There’s an enormous amount of existential suffering,” that’s familiar to rheumatologists because some patients may dread the diagnosis of a rheumatic disease the way they might fear a cancer diagnosis, especially if they have lost a family member to a condition that they suspect they share, he said. “To put yourself into an existential catastrophe — that’s not a small harm.”

Dr. Niewold agreed, pointing out that patients with a positive ANA test may “get unnecessarily worried and stay up all night reading about lupus, getting scared for weeks on end before seeing a specialist.” And there are financial harms as well for patients who may order the same test multiple times, or a whole slate of tests, that they don’t need for hundreds or thousands of dollars. There’s also the lost time and effort of researching a condition or even seeking out support groups that patients may pursue, Dr. Niewold said.

The likely biggest risk to individuals, however, is the potential for overdiagnosis or misdiagnosis.

“If someone comes in and they’ve read the textbook on lupus and they have a positive ANA, it’s really hard as a rheumatologist to walk that back,” Dr. Putman said. “The human mind is a powerful thing,” he added, and people who get a positive test will likely start to notice things like joint pain or a rash on their cheeks and begin attributing it to a diagnosis they risk convincing themselves they have. “When people come into your clinic not knowing what a disease would look like and they just tell you how they’re feeling, it’s a much cleaner and more honest way to approach diagnosis.”

Most patients likely don’t realize, for example, that none of the tests rheumatologists usually order are diagnostic in and of themselves, Dr. Niewold said. “They’re all kind of like stars in the constellation of a diagnosis,” he said. “They’re helpful, but none of them is sufficient by itself.”

Dr. Killeen agreed, noting that “consumers might not understand the nuances of these tests well enough to know whether it is appropriate to order them or how to interpret the results correctly.” Given the long-term implications of a diagnosis for a rheumatologic disease, “I would have concerns about consumers ordering and interpreting rheumatologic tests without working closely with their physicians,” Dr. Killeen said. “The main concern that lab experts have about direct-to-consumer tests is the potential for people to get misleading results and/or to misinterpret their results, which in turn could lead to people not getting the treatment they need or getting treatment when they don’t need any at all.”

It’s one thing for patients to come in asking for a particular treatment they may not need but which a doctor may be able to dissuade them from seeking. But Dr. Kim also pointed out the risk that patients may decide to treat themselves with therapies that haven’t undergone rigorous testing or haven’t been recommended by a physician.

“We tend to have people who come in with a pretty clear idea of what they want done, but the problem is, we don’t know if their reasoning is correct from a clinical perspective,” Dr. Kim said. Companies offer these tests with the belief that they’re “providing patients a choice, an option to take ownership,” he said, “but the potential harm can be realized very quickly because there are going to be people who are misdiagnosing themselves and, worse yet, may then pursue their own treatment plan that’s going in the opposite direction of where we think it needs to go.”

Or, on the flip side, if a patient erroneously believes they have the answer to what ails them, it may delay diagnosis of a more serious condition that’s rarer or harder to detect. Kim pointed to, for example, intravascular lymphoma, which is notoriously as difficult to identify as it is rare and aggressive. If a patient’s confirmation bias has led them to believe they have an autoimmune condition, they may not receive the more serious diagnosis until it’s advanced too far to treat.
 

Patient-Provider Relationship Friction

Another concern is how these tests may lead to confusion and frustration that can erode the patient-provider relationship, particularly because most patients don’t know how to interpret the results or understand the bigger context in which the results have to be interpreted. Many patients may think a test can come back with a binary answer, a positive or negative, and that means they do or don’t have a condition. That’s generally true for pregnancy tests, COVID tests, and sexually transmitted infection tests — the kinds of tests that have long been available to consumers and which have fairly straightforward answers.

But physicians know that’s not the case for many conditions, particularly those in rheumatology.

“In rheumatic diseases, because the tests have such marginal value in terms of diagnosis, almost always we develop a suspicion before we even think about ordering the tests, and then that dictates whether or not we cross that threshold,” Dr. Kim said. “A negative test doesn’t exclude the fact that you may have disease X, but a positive test also doesn’t mean you have disease X. All they provide is an idea of the risk.”

But some patients who come in with DTC test results have “already made the decision in their mind that they have a certain condition,” Dr. Kim said. “This is obviously dangerous because the majority of these patients do not have the condition they think they have, and it leaves a very uncomfortable feeling after the visit because they feel like they’ve been either betrayed by me or by the test, and they leave more confused.”

Patients may also come in with tests that a doctor isn’t familiar with or isn’t sure how to interpret on its own, at least for that particular patient.

“For ANA testing, we have a pretty good idea of its positive and negative predictive value because it’s ordered so much, but for many of these tests being offered, there are specific autoantibodies, and we tend to only get them in people where there’s a clinical suspicion,” Dr. Kim said. “Within that very specific context, we kind of understand what that value means, but if you give it to the general public, then those numbers aren’t as applicable and most likely overestimate the risk of disease.”

Even if providers consider the results of a DTC test in their differential, they may want to be sure it’s from a trustworthy source. “If a provider is uncertain about whether a direct-to-consumer testing company is reputable or about whether a direct-to-consumer test result is reliable, I would encourage them to consult with their laboratory medicine colleagues,” Dr. Killeen said.
 

Responding to Patients

Like any other patient coming to a clinical visit, the most common reason patients are likely ordering these tests is that they’re seeking answers. Kim doesn’t typically see patients doing their own monitoring for diagnosed conditions between visits — the expense would add up too quickly — or testing for genetic markers, which likely wouldn’t be very helpful either.

“Even though most of our diseases probably have a genetic underpinning, how much it contributes is always unclear,” Dr. Kim said. Even conditions with clear genetic variants, such as familial Mediterranean fever, spondyloarthritis, and Behçet disease, can only support a diagnosis, not diagnose it on its own, Dr. Killeen said. And these are not among the tests currently available on most DTC company sites.

While there are also tests that can offer information about genetic risks for certain medications, such as a thiopurine methyltransferase test to find out if a patient lacks the enzyme needed to break down the immunosuppressant drug azathioprine, Kim hasn’t seen patients seeking these out either.

“The more global and more compassionate way to think about this is that we have a lot of people who are struggling to understand what’s going on with their bodies, and most physicians really don’t know what the next steps are for these people,” Dr. Kim said. “They’re desperate, and their quality of life is so poor that they’re going to take extreme steps to try to manage their own frustration with this condition.”

That means clinicians’ most powerful tools when patients come in with DTC test results are their listening skills.

“Empathy is the most important thing, just being able to share the patient’s frustration to the point where they had to take matters into their own hands,” Dr. Kim said. “I think a lot of rheumatologists are actually pretty comfortable being in this position.”

Additionally, doctors should know that some patients may be engaging in attempts to self-diagnose, self-treat, or otherwise self-manage their symptoms or perceived condition. “They just need to be aware and try to make sure there’s no harm being done,” Dr. Kim said.

Ms. Welsh didn’t seek treatment or diagnosis on her own, but getting her test also did not give her the control she was seeking. “Looking back, it was kind of a waste of money, but it felt good in the moment,” Ms. Welsh said. “I was so upset, and I wanted that control, and in the end, it didn’t get me results any sooner, and it didn’t give me peace of mind.”

It was Ms. Welsh’s primary care doctor listening to her concerns, ordering the same test she had ordered with several others, and working with her to seek answers that reassured her that her provider cared about her well-being.

“A lot of what I do in my business is reassure people that you know what they have is treatable or not going to end their life as they know it,” Dr. Putman said. “And you certainly can’t reassure them if they’re not in your clinic yet.”

Dr. Putman has participated in clinical trials with AbbVie, consulting with Novartis and GSK, and clinical trials and consulting with Amgen and AstraZeneca. Dr. Niewold reported receiving research grants from EMD Serono and Zenas BioPharma and consulting for Thermo Fisher Scientific, Progentec Diagnostics, Roivant Sciences, AstraZeneca, S3 Connected Health, Flagship Pioneering, and Guidepoint. Dr. Kim reported sponsored research agreements with AstraZeneca, Bristol-Myers Squibb, Novartis, and CRISPR Therapeutics; royalties from Kypha; and consulting/speaking for Amgen, ANI Pharmaceuticals, Atara Biotherapeutics, Aurinia Pharmaceuticals, CARGO Therapeutics, Exagen Diagnostics, GSK, Hinge Bio, Kypha, Progentec Diagnostics, Synthekine, and UpToDate. Dr. Killeen had no relevant financial relationships to disclose.
 

A version of this article first appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved osimertinib (Tagrisso, AstraZeneca) for the treatment of locally advanced, unresectable non–small cell lung cancer (NSCLC) in certain adult patients.

Specifically, the third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) was approved for patients whose disease has not progressed during or after concurrent or sequential platinum-based chemoradiation therapy and whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations. Such EGFR mutations can be detected by an FDA-approved test.

The FDA approved osimertinib in combination with platinum-based chemotherapy as first-line treatment for patients with locally advanced or metastatic NSCLC with the same mutations in February. The EGFR-TKI also carries other indications, including as first-line monotherapy for locally advanced or metastatic EGFR-mutated NSCLC.
 

Trial Findings Supporting Latest Approval

AstraZeneca announced in June that osimertinib had been granted Priority Review and Breakthrough Therapy Designation for its newest indication.

The September 25 approval was based on findings from the randomized, placebo-controlled LAURA trial of 216 patients, which demonstrated improved median progression-free survival with osimertinib vs placebo (39.1 vs 5.6 months; hazard ratio, 0.16). Overall survival results were immature at the most recent analysis, but “no trend towards a detriment was observed,” with 36% of prespecified deaths for the final analysis reported, according to an FDA press release.
 

Adverse Events

Study participants were randomized 2:1 to receive the osimertinib recommended dose of 80 mg given orally once daily or placebo until disease progression or unacceptable toxicity. The most common adverse reactions, occurring in at least 20% of patients, were lymphopenia, leukopenia, interstitial lung disease/pneumonitis, thrombocytopenia, neutropenia, rash, diarrhea, nail toxicity, musculoskeletal pain, cough, and COVID-19 infection.

A version of this article first appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved osimertinib (Tagrisso, AstraZeneca) for the treatment of locally advanced, unresectable non–small cell lung cancer (NSCLC) in certain adult patients.

Specifically, the third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) was approved for patients whose disease has not progressed during or after concurrent or sequential platinum-based chemoradiation therapy and whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations. Such EGFR mutations can be detected by an FDA-approved test.

The FDA approved osimertinib in combination with platinum-based chemotherapy as first-line treatment for patients with locally advanced or metastatic NSCLC with the same mutations in February. The EGFR-TKI also carries other indications, including as first-line monotherapy for locally advanced or metastatic EGFR-mutated NSCLC.
 

Trial Findings Supporting Latest Approval

AstraZeneca announced in June that osimertinib had been granted Priority Review and Breakthrough Therapy Designation for its newest indication.

The September 25 approval was based on findings from the randomized, placebo-controlled LAURA trial of 216 patients, which demonstrated improved median progression-free survival with osimertinib vs placebo (39.1 vs 5.6 months; hazard ratio, 0.16). Overall survival results were immature at the most recent analysis, but “no trend towards a detriment was observed,” with 36% of prespecified deaths for the final analysis reported, according to an FDA press release.
 

Adverse Events

Study participants were randomized 2:1 to receive the osimertinib recommended dose of 80 mg given orally once daily or placebo until disease progression or unacceptable toxicity. The most common adverse reactions, occurring in at least 20% of patients, were lymphopenia, leukopenia, interstitial lung disease/pneumonitis, thrombocytopenia, neutropenia, rash, diarrhea, nail toxicity, musculoskeletal pain, cough, and COVID-19 infection.

A version of this article first appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved osimertinib (Tagrisso, AstraZeneca) for the treatment of locally advanced, unresectable non–small cell lung cancer (NSCLC) in certain adult patients.

Specifically, the third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) was approved for patients whose disease has not progressed during or after concurrent or sequential platinum-based chemoradiation therapy and whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations. Such EGFR mutations can be detected by an FDA-approved test.

The FDA approved osimertinib in combination with platinum-based chemotherapy as first-line treatment for patients with locally advanced or metastatic NSCLC with the same mutations in February. The EGFR-TKI also carries other indications, including as first-line monotherapy for locally advanced or metastatic EGFR-mutated NSCLC.
 

Trial Findings Supporting Latest Approval

AstraZeneca announced in June that osimertinib had been granted Priority Review and Breakthrough Therapy Designation for its newest indication.

The September 25 approval was based on findings from the randomized, placebo-controlled LAURA trial of 216 patients, which demonstrated improved median progression-free survival with osimertinib vs placebo (39.1 vs 5.6 months; hazard ratio, 0.16). Overall survival results were immature at the most recent analysis, but “no trend towards a detriment was observed,” with 36% of prespecified deaths for the final analysis reported, according to an FDA press release.
 

Adverse Events

Study participants were randomized 2:1 to receive the osimertinib recommended dose of 80 mg given orally once daily or placebo until disease progression or unacceptable toxicity. The most common adverse reactions, occurring in at least 20% of patients, were lymphopenia, leukopenia, interstitial lung disease/pneumonitis, thrombocytopenia, neutropenia, rash, diarrhea, nail toxicity, musculoskeletal pain, cough, and COVID-19 infection.

A version of this article first appeared on Medscape.com.

Guinea pigs are important to Ohio State University leukemia specialist Kerry A. Rogers, MD, but not because they’re in her research laboratory, where the focus is on studying treatments for chronic lymphocytic leukemia (CLL), hairy cell leukemia, and autoimmune hemolytic anemia. Instead, two of these little creatures — Pancake and Maple — are pets who live with her at home. 

Sometimes, however, Dr. Rogers’ guinea pigs pay visits to her workplace. Every Halloween, she dresses them up and shows off their photos to just about everyone. Patients, coworkers, and even random people in the elevator get glimpses of the furry pair, who pose as dinosaurs, bats, aquarium shrimp, sharks, spiders, and bumblebees.

courtesy Ohio State University

“Being in the hospital is not funny, but guinea pigs dressed up for Halloween is incredibly funny,” Dr. Rogers said. “They make a lot of people smile.”

For physician-scientist Dr. Rogers, a native of suburban Chicago, quality of life for patients is a priority, even when she’s not trying to entertain them and lift their spirits.

The field of hematology “is trying to figure out not only what’s biologically effective for disease, but also what might be best for people living with the condition,” she said. “This is especially true in terms of patient preference for a treatment you complete vs an ongoing or continuous treatment. I really like this idea of having more attention paid to what matters to patients, which the field of medicine sometimes forgets.”

In an interview, Dr. Rogers spoke about the appeal of storytelling in medicine, advances in treatment for CLL and hairy cell leukemia, and the challenges of college football loyalty. 

How did you get drawn to medicine?

Ever since I was a kid, I thought, “Oh, I really want to be a doctor. That sounds fun.” At its core, medicine blends things I really like: science, helping people with a problem, and storytelling. 

The practice of medicine is a lot about stories — talking to people, figuring out what they’re telling you about what’s going on, then explaining what you know in a way that makes sense to them. 

What changes have you seen in leukemia care during your career?

The biggest change is the move from chemotherapies to targeted agents. When I first did a fellowship here at Ohio State, we had studies of ibrutinib, the first-in-class covalent Bruton tyrosine kinase (BTK) inhibitor. We’d see patients who’d been on chemotherapy for the past 10 or 15 years, and then they got in a clinical trial. Ibrutinib worked better than any of the chemotherapy, and they felt better and had fewer side effects. 

Now, chemoimmunotherapy is not recommended for CLL, and you can see the impact of targeted agents on the lives of patients. Instead of telling people they need to put aside work and personal plans to take intensive chemotherapy for 6 months, you say: “You’ve got to come to some more appointments to make sure the treatment is going well, and you don’t have too many side effects. But you can expect to continue to work full-time and go to your niece’s wedding out of state or whatever else you want to do.”

What are you most excited about working on? 

I’m the principal investigator of a study combining obinutuzumab, ibrutinib, and venetoclax for a fixed duration, a defined treatment course of a little over a year. I just reported the median progression-free survival for phase 2 at the European Hematology Association meeting this spring. It was over 7 years for both patients who had prior treatment and those who hadn’t. 

The idea that people could take a year of treatment and get a huge benefit after completing it is quite important. The regimen has gone into phase 3 testing, and we’re now trying to understand the impact in terms of which patients got longer responses or which didn’t. 

What are some challenges that remain in CLL?

There are still patients whose CLL becomes resistant to our two most commonly used classes of agents: BTK inhibitors and the BLC2 inhibitor, venetoclax. There are some more BCL2 inhibitors in development, but venetoclax is the only one currently approved.

I am also principal investigator on another study that added venetoclax to ibrutinib when resistance mutations developed that would predict ultimate resistance to ibrutinib. The median progression-free survival in that study was 40.7 months, whereas the expectation with venetoclax alone is 24 months. So, it really improved the amount of time people were in remission. This study is ongoing. 

We’ve just started a phase 2 study to have patients take pirtobrutinib, a noncovalent BTK inhibitor, in combination with venetoclax in cases where CLL has become resistant to a covalent BTK inhibitor. Patients will take this combination for about a year and a half. 

It’s been really exciting to see the impact of some of these combinations both as first-line CLL treatment and in CLL that’s become resistant. But trying to understand what predicts response is a harder thing. I wish I knew the answers for what causes this synergy between those two classes of drugs. 

You also specialize in hairy cell leukemia. Could you talk about what it is? 

CLL is the most prevalent adult leukemia in the Western world, whereas hairy cell leukemia is very rare. It’s a slow-growing B-cell cancer that got its name because under the microscope, the cells have hairy projections on them.

It had a survival of only about 2-4 years before the development of purine analogs. After a course of pentostatin or cladribine (2-chlordeoxyadenosine), some people never need treatment again in our natural lifespan. 

But some patients don’t benefit from purine analogs, either because they have a devastating infection that makes them unsuitable or for another reason. Or they end up needing treatment every 2-3 years, which isn’t something you want to do for 30 years of someone’s lifespan. 

What are some challenges in hairy cell leukemia?

It’s a rare condition, so it can be hard to do a really large clinical trial. A lot of physicians think that the prognosis is good and miss out on an opportunity for better treatment and to generate new knowledge.

Also, some people perceive that it’s not actually a problem for people living with it. Cladribine and pentostatin are not terrible chemotherapies, and most people tolerate them very well. But we can make treatment for hairy cell more effective and more tolerable for our patients if we put more effort into researching it. 

I’ve heard that you’re a big college football fan. How do you balance your history as a University of Michigan medical school alum with your work for archrival Ohio State? 

I went to Northwestern for undergrad, as did several people in my family. So, I usually just claim Northwestern is my football affiliation. It doesn’t inspire much vitriol if you cheer for Northwestern! 

And I understand that you live with a pair of guinea pigs. Do tell.

I adopted guinea pigs as pets in medical school. They’re cute and friendly, and they make all this noise for you when you come home. 

Courtesy Dr. Rogers

Once, one of my clinic patients said, “I don’t want to be your guinea pig.” I thought, “you should be so lucky.” They have quite a lifestyle: They’ve trained me to bring them vegetables, they nap most of the time, and they have a play space, a guinea pig playground. 

I thought I liked the guinea pigs more than they liked me. But last fall, I was gone for about 10 days. When I got back, I’d never heard them make so much noise. They were extremely happy to see me. Then for the next 48 hours, one would randomly start whistling for me to come over. I thought, “oh, these things do like me a little bit.”

Dr. Rogers disclosed receiving research funding from Genentech, AbbVie, Novartis, and AstraZeneca and consulting/advisory relationships with AstraZeneca, AbbVie, Genentech, Janssen, Pharmacyclics, BeiGene, Loxo@Lilly, and Alpine Immune Sciences. 

A version of this article first appeared on Medscape.com.

Guinea pigs are important to Ohio State University leukemia specialist Kerry A. Rogers, MD, but not because they’re in her research laboratory, where the focus is on studying treatments for chronic lymphocytic leukemia (CLL), hairy cell leukemia, and autoimmune hemolytic anemia. Instead, two of these little creatures — Pancake and Maple — are pets who live with her at home. 

Sometimes, however, Dr. Rogers’ guinea pigs pay visits to her workplace. Every Halloween, she dresses them up and shows off their photos to just about everyone. Patients, coworkers, and even random people in the elevator get glimpses of the furry pair, who pose as dinosaurs, bats, aquarium shrimp, sharks, spiders, and bumblebees.

courtesy Ohio State University

“Being in the hospital is not funny, but guinea pigs dressed up for Halloween is incredibly funny,” Dr. Rogers said. “They make a lot of people smile.”

For physician-scientist Dr. Rogers, a native of suburban Chicago, quality of life for patients is a priority, even when she’s not trying to entertain them and lift their spirits.

The field of hematology “is trying to figure out not only what’s biologically effective for disease, but also what might be best for people living with the condition,” she said. “This is especially true in terms of patient preference for a treatment you complete vs an ongoing or continuous treatment. I really like this idea of having more attention paid to what matters to patients, which the field of medicine sometimes forgets.”

In an interview, Dr. Rogers spoke about the appeal of storytelling in medicine, advances in treatment for CLL and hairy cell leukemia, and the challenges of college football loyalty. 

How did you get drawn to medicine?

Ever since I was a kid, I thought, “Oh, I really want to be a doctor. That sounds fun.” At its core, medicine blends things I really like: science, helping people with a problem, and storytelling. 

The practice of medicine is a lot about stories — talking to people, figuring out what they’re telling you about what’s going on, then explaining what you know in a way that makes sense to them. 

What changes have you seen in leukemia care during your career?

The biggest change is the move from chemotherapies to targeted agents. When I first did a fellowship here at Ohio State, we had studies of ibrutinib, the first-in-class covalent Bruton tyrosine kinase (BTK) inhibitor. We’d see patients who’d been on chemotherapy for the past 10 or 15 years, and then they got in a clinical trial. Ibrutinib worked better than any of the chemotherapy, and they felt better and had fewer side effects. 

Now, chemoimmunotherapy is not recommended for CLL, and you can see the impact of targeted agents on the lives of patients. Instead of telling people they need to put aside work and personal plans to take intensive chemotherapy for 6 months, you say: “You’ve got to come to some more appointments to make sure the treatment is going well, and you don’t have too many side effects. But you can expect to continue to work full-time and go to your niece’s wedding out of state or whatever else you want to do.”

What are you most excited about working on? 

I’m the principal investigator of a study combining obinutuzumab, ibrutinib, and venetoclax for a fixed duration, a defined treatment course of a little over a year. I just reported the median progression-free survival for phase 2 at the European Hematology Association meeting this spring. It was over 7 years for both patients who had prior treatment and those who hadn’t. 

The idea that people could take a year of treatment and get a huge benefit after completing it is quite important. The regimen has gone into phase 3 testing, and we’re now trying to understand the impact in terms of which patients got longer responses or which didn’t. 

What are some challenges that remain in CLL?

There are still patients whose CLL becomes resistant to our two most commonly used classes of agents: BTK inhibitors and the BLC2 inhibitor, venetoclax. There are some more BCL2 inhibitors in development, but venetoclax is the only one currently approved.

I am also principal investigator on another study that added venetoclax to ibrutinib when resistance mutations developed that would predict ultimate resistance to ibrutinib. The median progression-free survival in that study was 40.7 months, whereas the expectation with venetoclax alone is 24 months. So, it really improved the amount of time people were in remission. This study is ongoing. 

We’ve just started a phase 2 study to have patients take pirtobrutinib, a noncovalent BTK inhibitor, in combination with venetoclax in cases where CLL has become resistant to a covalent BTK inhibitor. Patients will take this combination for about a year and a half. 

It’s been really exciting to see the impact of some of these combinations both as first-line CLL treatment and in CLL that’s become resistant. But trying to understand what predicts response is a harder thing. I wish I knew the answers for what causes this synergy between those two classes of drugs. 

You also specialize in hairy cell leukemia. Could you talk about what it is? 

CLL is the most prevalent adult leukemia in the Western world, whereas hairy cell leukemia is very rare. It’s a slow-growing B-cell cancer that got its name because under the microscope, the cells have hairy projections on them.

It had a survival of only about 2-4 years before the development of purine analogs. After a course of pentostatin or cladribine (2-chlordeoxyadenosine), some people never need treatment again in our natural lifespan. 

But some patients don’t benefit from purine analogs, either because they have a devastating infection that makes them unsuitable or for another reason. Or they end up needing treatment every 2-3 years, which isn’t something you want to do for 30 years of someone’s lifespan. 

What are some challenges in hairy cell leukemia?

It’s a rare condition, so it can be hard to do a really large clinical trial. A lot of physicians think that the prognosis is good and miss out on an opportunity for better treatment and to generate new knowledge.

Also, some people perceive that it’s not actually a problem for people living with it. Cladribine and pentostatin are not terrible chemotherapies, and most people tolerate them very well. But we can make treatment for hairy cell more effective and more tolerable for our patients if we put more effort into researching it. 

I’ve heard that you’re a big college football fan. How do you balance your history as a University of Michigan medical school alum with your work for archrival Ohio State? 

I went to Northwestern for undergrad, as did several people in my family. So, I usually just claim Northwestern is my football affiliation. It doesn’t inspire much vitriol if you cheer for Northwestern! 

And I understand that you live with a pair of guinea pigs. Do tell.

I adopted guinea pigs as pets in medical school. They’re cute and friendly, and they make all this noise for you when you come home. 

Courtesy Dr. Rogers

Once, one of my clinic patients said, “I don’t want to be your guinea pig.” I thought, “you should be so lucky.” They have quite a lifestyle: They’ve trained me to bring them vegetables, they nap most of the time, and they have a play space, a guinea pig playground. 

I thought I liked the guinea pigs more than they liked me. But last fall, I was gone for about 10 days. When I got back, I’d never heard them make so much noise. They were extremely happy to see me. Then for the next 48 hours, one would randomly start whistling for me to come over. I thought, “oh, these things do like me a little bit.”

Dr. Rogers disclosed receiving research funding from Genentech, AbbVie, Novartis, and AstraZeneca and consulting/advisory relationships with AstraZeneca, AbbVie, Genentech, Janssen, Pharmacyclics, BeiGene, Loxo@Lilly, and Alpine Immune Sciences. 

A version of this article first appeared on Medscape.com.

Guinea pigs are important to Ohio State University leukemia specialist Kerry A. Rogers, MD, but not because they’re in her research laboratory, where the focus is on studying treatments for chronic lymphocytic leukemia (CLL), hairy cell leukemia, and autoimmune hemolytic anemia. Instead, two of these little creatures — Pancake and Maple — are pets who live with her at home. 

Sometimes, however, Dr. Rogers’ guinea pigs pay visits to her workplace. Every Halloween, she dresses them up and shows off their photos to just about everyone. Patients, coworkers, and even random people in the elevator get glimpses of the furry pair, who pose as dinosaurs, bats, aquarium shrimp, sharks, spiders, and bumblebees.

courtesy Ohio State University

“Being in the hospital is not funny, but guinea pigs dressed up for Halloween is incredibly funny,” Dr. Rogers said. “They make a lot of people smile.”

For physician-scientist Dr. Rogers, a native of suburban Chicago, quality of life for patients is a priority, even when she’s not trying to entertain them and lift their spirits.

The field of hematology “is trying to figure out not only what’s biologically effective for disease, but also what might be best for people living with the condition,” she said. “This is especially true in terms of patient preference for a treatment you complete vs an ongoing or continuous treatment. I really like this idea of having more attention paid to what matters to patients, which the field of medicine sometimes forgets.”

In an interview, Dr. Rogers spoke about the appeal of storytelling in medicine, advances in treatment for CLL and hairy cell leukemia, and the challenges of college football loyalty. 

How did you get drawn to medicine?

Ever since I was a kid, I thought, “Oh, I really want to be a doctor. That sounds fun.” At its core, medicine blends things I really like: science, helping people with a problem, and storytelling. 

The practice of medicine is a lot about stories — talking to people, figuring out what they’re telling you about what’s going on, then explaining what you know in a way that makes sense to them. 

What changes have you seen in leukemia care during your career?

The biggest change is the move from chemotherapies to targeted agents. When I first did a fellowship here at Ohio State, we had studies of ibrutinib, the first-in-class covalent Bruton tyrosine kinase (BTK) inhibitor. We’d see patients who’d been on chemotherapy for the past 10 or 15 years, and then they got in a clinical trial. Ibrutinib worked better than any of the chemotherapy, and they felt better and had fewer side effects. 

Now, chemoimmunotherapy is not recommended for CLL, and you can see the impact of targeted agents on the lives of patients. Instead of telling people they need to put aside work and personal plans to take intensive chemotherapy for 6 months, you say: “You’ve got to come to some more appointments to make sure the treatment is going well, and you don’t have too many side effects. But you can expect to continue to work full-time and go to your niece’s wedding out of state or whatever else you want to do.”

What are you most excited about working on? 

I’m the principal investigator of a study combining obinutuzumab, ibrutinib, and venetoclax for a fixed duration, a defined treatment course of a little over a year. I just reported the median progression-free survival for phase 2 at the European Hematology Association meeting this spring. It was over 7 years for both patients who had prior treatment and those who hadn’t. 

The idea that people could take a year of treatment and get a huge benefit after completing it is quite important. The regimen has gone into phase 3 testing, and we’re now trying to understand the impact in terms of which patients got longer responses or which didn’t. 

What are some challenges that remain in CLL?

There are still patients whose CLL becomes resistant to our two most commonly used classes of agents: BTK inhibitors and the BLC2 inhibitor, venetoclax. There are some more BCL2 inhibitors in development, but venetoclax is the only one currently approved.

I am also principal investigator on another study that added venetoclax to ibrutinib when resistance mutations developed that would predict ultimate resistance to ibrutinib. The median progression-free survival in that study was 40.7 months, whereas the expectation with venetoclax alone is 24 months. So, it really improved the amount of time people were in remission. This study is ongoing. 

We’ve just started a phase 2 study to have patients take pirtobrutinib, a noncovalent BTK inhibitor, in combination with venetoclax in cases where CLL has become resistant to a covalent BTK inhibitor. Patients will take this combination for about a year and a half. 

It’s been really exciting to see the impact of some of these combinations both as first-line CLL treatment and in CLL that’s become resistant. But trying to understand what predicts response is a harder thing. I wish I knew the answers for what causes this synergy between those two classes of drugs. 

You also specialize in hairy cell leukemia. Could you talk about what it is? 

CLL is the most prevalent adult leukemia in the Western world, whereas hairy cell leukemia is very rare. It’s a slow-growing B-cell cancer that got its name because under the microscope, the cells have hairy projections on them.

It had a survival of only about 2-4 years before the development of purine analogs. After a course of pentostatin or cladribine (2-chlordeoxyadenosine), some people never need treatment again in our natural lifespan. 

But some patients don’t benefit from purine analogs, either because they have a devastating infection that makes them unsuitable or for another reason. Or they end up needing treatment every 2-3 years, which isn’t something you want to do for 30 years of someone’s lifespan. 

What are some challenges in hairy cell leukemia?

It’s a rare condition, so it can be hard to do a really large clinical trial. A lot of physicians think that the prognosis is good and miss out on an opportunity for better treatment and to generate new knowledge.

Also, some people perceive that it’s not actually a problem for people living with it. Cladribine and pentostatin are not terrible chemotherapies, and most people tolerate them very well. But we can make treatment for hairy cell more effective and more tolerable for our patients if we put more effort into researching it. 

I’ve heard that you’re a big college football fan. How do you balance your history as a University of Michigan medical school alum with your work for archrival Ohio State? 

I went to Northwestern for undergrad, as did several people in my family. So, I usually just claim Northwestern is my football affiliation. It doesn’t inspire much vitriol if you cheer for Northwestern! 

And I understand that you live with a pair of guinea pigs. Do tell.

I adopted guinea pigs as pets in medical school. They’re cute and friendly, and they make all this noise for you when you come home. 

Courtesy Dr. Rogers

Once, one of my clinic patients said, “I don’t want to be your guinea pig.” I thought, “you should be so lucky.” They have quite a lifestyle: They’ve trained me to bring them vegetables, they nap most of the time, and they have a play space, a guinea pig playground. 

I thought I liked the guinea pigs more than they liked me. But last fall, I was gone for about 10 days. When I got back, I’d never heard them make so much noise. They were extremely happy to see me. Then for the next 48 hours, one would randomly start whistling for me to come over. I thought, “oh, these things do like me a little bit.”

Dr. Rogers disclosed receiving research funding from Genentech, AbbVie, Novartis, and AstraZeneca and consulting/advisory relationships with AstraZeneca, AbbVie, Genentech, Janssen, Pharmacyclics, BeiGene, Loxo@Lilly, and Alpine Immune Sciences. 

A version of this article first appeared on Medscape.com.