Hematology News

For some medical students and trainees who go on to become hematologists, attraction to the field happens the first time they’re engrossed in figuring out what a blood smear is telling them. Others get drawn to hematology during a rotation in residency, when they encounter patients with hemophilia or sickle cell disease.

But when it comes to turning people on to the idea of a career in classical hematology (CH), there may be no more powerful influence than a mentor who loves their job. That’s why the field is focusing so much on supporting mentors and mentees amid a stark shortage of classical hematologists.

“Mentorship is key for maintaining trainee interest in the field and for providing role models for career growth,” said Rakhi P. Naik, MD, MHS, associate professor of medicine and director of the Hematology Fellowship Track at Johns Hopkins University, Baltimore, Maryland, in an interview. “This collaboration is especially critical because there are so few trainees and so few mentors currently in the field.”

Now there’s new research backing up the power of mentorship, even when it’s only provided virtually, and a brand-new program aims to unite more mentors and mentees.

Here’s a closer look at mentor-focused efforts to attract medical students to CH.

 

How Severe Is the Shortage in CH?

Patients with conditions treated by classical hematologists are waiting months for appointments at many outpatient centers, with some being forced to wait 6 months or more, said Srikanth Nagalla, MD, chief of benign hematology at Miami Cancer Institute, Florida, in an interview.

The shortage is creating dire problems in the inpatient setting too, Nagalla said. “Serious blood disorders like heparin-induced thrombocytopenia, acute chest syndrome [a complication of sickle cell disease], and thrombotic thrombocytopenic purpura have to be diagnosed and treated in a timely manner. If not, the morbidity and mortality are really high.”

If classical hematologists aren’t available, he said, oncologists and others not trained in hematology will need to cover these patients. 

Hematologist Ariela Marshall, MD, associate professor of medicine at the University of Minnesota in Minneapolis, noted in an interview that the CH shortage comes at a time when medical advances and an aging population are boosting the number of patients with noncancerous blood disorders. Older people are at greater risk for blood clots, she said. And lifespans for patients with bleeding and clotting disorders are rising thanks to effective new treatments.

“Because of our larger patient population in CH, we are going to need more classical hematologists to follow them for longer and longer periods of time,” she said. 

There’s no sign yet that newly minted physicians will take up the slack in CH. A 2019 study found that just 4.6% of 626 of hematology/oncology fellows said they planned to go into CH, also known as benign hematology, vs 67.1% who expected to treat patients with solid tumors, blood cancer, or both. The rest, 24.6%, planned to work in CH plus the two oncology fields.

 

Why Does a Shortage Exist?

“The reasons are complex, but one of the most important factors was the combining of the adult hematology and medical oncology training programs by the Accreditation Council for Graduate Medical Education in 1995,” Naik said. “After that time, the majority of fellowship training programs went from having separate programs for hematology and medical oncology to combining the training for the two specialties into one. Because most of these combined training programs resided within Cancer Centers, classical hematology training slowly became de-emphasized.”

As a result, fewer fellows ended up specializing in CH, she said. 

The field of CH also appears to suffer from a less than enticing reputation. According to a 2019 study coauthored by Marshall, surveys of thousands of hematology/oncology fellows found that “hematology, particularly benign hematology, was viewed as having poorer income potential, research funding, job availability, and job security than oncology.”

Regarding pay, Marshall said the good news is that many classical hematologists work in academia, where it’s common for pay to be “equitable across hematology/oncology divisions and based more on academic rank and other factors rather than subspecialty within hematology oncology.”

However, she noted, “this may differ at institutions where hematology and oncology are different departments. For example, centers where oncology is its own department, and hematology is part of the department of medicine.” 

As for job availability, Naik said that there’s plenty of demand. “In academics, it is clear that there are jobs available everywhere, but trainees are often worried about job prospects in private practice. While classical hematology jobs in private practice are not widely advertised, I can attest that there is no shortage of need,” she said. “Many private practices do not specifically advertise for classical hematologists because they assume that classical hematology experts are not available. But I assure you that every private practice my trainees have ever approached is always ecstatic to hire a classical hematologist.”

 

Why Are Mentors Important?

Mentorship is crucial to promoting the value of CH as a great career choice in a competitive environment, classical hematologists say. “We can motivate trainees by showing how the disease states themselves are so fascinating and how the treatments are showing great outcomes,” Nagalla said. “We can show positive results, how patient lives can be changed, and how well-respected across the system [we] are.”

As a selling point, classical hematologists like to emphasize that their field requires intensive detective work. “Let’s say a patient comes with anemia, which might have 15 different causes. You get some labs, and then you systemically rule in or rule out most of these on the differential diagnosis,” Nagalla said. “Then once you narrow it down, you get more labs. You keep going to the next step and next step, and so finally you come to a conclusion.”

As for therapy, Marshall said that “while for many cancers there are specific treatment recommendations for patients with a specific cancer type at a specific stage, there is not always a specific treatment recommendation (or a ‘right answer’) for our CH patients. Treatment planning depends strongly on a patient’s preferences, other medical conditions, and a discussion about risks [and] benefits of different treatment options such that two patients with the same condition may choose two different treatment options.”

Marshall also emphasizes to trainees that “CH is a broad field. Physicians and trainees are able to interact and collaborate with physicians in other specialties such as gastroenterology, cardiology, ob/gyn, and surgical specialties.” 

 

Does Research Support Mentorship in CH?

The 2019 study that revealed just 4.6% of fellows planned to go into CH found that “fellows who planned to enter hematology-only careers were significantly more likely to report having clinical training and mentorship experiences in hematology throughout their training relative to fellows with oncology-only or combined hematology/oncology career plans.”

Now there are more data to support mentorships. For a study published in Blood Advances in September 2024, Zoya Qureshy, MD, an internal medicine chief resident at the University of California at San Diego, and colleagues evaluated a year-long external membership program implemented by the American Society of Hematology (ASH) Medical Educators Institute. 

The program linked 35 US hematology/oncology fellows (80% female, 46% White, 35% Asian) who were interested in CH to 34 North American faculty members. The pairs were told to meet virtually once a month. 

Of 30 mentees and 23 mentors surveyed, 94% and 85%, respectively, said their pairings were good matches. Two thirds of the mentees accepted faculty positions in CH after their mentorships.

“Our study showed that external mentorship in a virtual format is feasible,” Qureshy said in an interview. “Additionally, external mentorship provided benefits such as different perspectives and the opportunity for mentorship for those who may not have it in their field of interest at their home institution.”

Qureshy added that “one strength of our mentorship program was that mentoring pairs were meticulously assigned based on shared interests and background. Many participants cited this common ground as a reason why they thought their mentoring pair was a good match.” 

There’s an important caveat: Most of the mentees weren’t new to CH. About 70% had previously worked with a mentor in the CH field, and 86% had previously conducted research in the field. 

 

What’s Next for Mentorship in CH?

The ASH Hematology-Focused Fellowship Training Program Consortium aims to mint 50 new academic hematologists by 2030 through programs at 12 institutions. “Mentorship is an exciting aspect of the program since it allows classical hematology trainees to form a network of peers nationally and also provides access to mentors across institutions,” Naik said. “And as the workforce grows, there will be more and more role models for future trainees to look up to.”

Moving forward, she said, “we hope to inspire even more institutions to adopt hematology training tracks throughout the country.”

Meanwhile, ASH’s new Classical Hematology Advancement Mentorship is taking applications for its debut 2025 program through January 9, 2025. Trainees will meet monthly with mentors both virtually and in person. Applicants must have been in their first or second year of hematology/oncology fellowship training at accredited programs in the United States as of July 15, 2024.

Naik, Marshall, Nagalla, and Qureshy have no relevant disclosures.

A version of this article appeared on Medscape.com.

For some medical students and trainees who go on to become hematologists, attraction to the field happens the first time they’re engrossed in figuring out what a blood smear is telling them. Others get drawn to hematology during a rotation in residency, when they encounter patients with hemophilia or sickle cell disease.

But when it comes to turning people on to the idea of a career in classical hematology (CH), there may be no more powerful influence than a mentor who loves their job. That’s why the field is focusing so much on supporting mentors and mentees amid a stark shortage of classical hematologists.

“Mentorship is key for maintaining trainee interest in the field and for providing role models for career growth,” said Rakhi P. Naik, MD, MHS, associate professor of medicine and director of the Hematology Fellowship Track at Johns Hopkins University, Baltimore, Maryland, in an interview. “This collaboration is especially critical because there are so few trainees and so few mentors currently in the field.”

Now there’s new research backing up the power of mentorship, even when it’s only provided virtually, and a brand-new program aims to unite more mentors and mentees.

Here’s a closer look at mentor-focused efforts to attract medical students to CH.

 

How Severe Is the Shortage in CH?

Patients with conditions treated by classical hematologists are waiting months for appointments at many outpatient centers, with some being forced to wait 6 months or more, said Srikanth Nagalla, MD, chief of benign hematology at Miami Cancer Institute, Florida, in an interview.

The shortage is creating dire problems in the inpatient setting too, Nagalla said. “Serious blood disorders like heparin-induced thrombocytopenia, acute chest syndrome [a complication of sickle cell disease], and thrombotic thrombocytopenic purpura have to be diagnosed and treated in a timely manner. If not, the morbidity and mortality are really high.”

If classical hematologists aren’t available, he said, oncologists and others not trained in hematology will need to cover these patients. 

Hematologist Ariela Marshall, MD, associate professor of medicine at the University of Minnesota in Minneapolis, noted in an interview that the CH shortage comes at a time when medical advances and an aging population are boosting the number of patients with noncancerous blood disorders. Older people are at greater risk for blood clots, she said. And lifespans for patients with bleeding and clotting disorders are rising thanks to effective new treatments.

“Because of our larger patient population in CH, we are going to need more classical hematologists to follow them for longer and longer periods of time,” she said. 

There’s no sign yet that newly minted physicians will take up the slack in CH. A 2019 study found that just 4.6% of 626 of hematology/oncology fellows said they planned to go into CH, also known as benign hematology, vs 67.1% who expected to treat patients with solid tumors, blood cancer, or both. The rest, 24.6%, planned to work in CH plus the two oncology fields.

 

Why Does a Shortage Exist?

“The reasons are complex, but one of the most important factors was the combining of the adult hematology and medical oncology training programs by the Accreditation Council for Graduate Medical Education in 1995,” Naik said. “After that time, the majority of fellowship training programs went from having separate programs for hematology and medical oncology to combining the training for the two specialties into one. Because most of these combined training programs resided within Cancer Centers, classical hematology training slowly became de-emphasized.”

As a result, fewer fellows ended up specializing in CH, she said. 

The field of CH also appears to suffer from a less than enticing reputation. According to a 2019 study coauthored by Marshall, surveys of thousands of hematology/oncology fellows found that “hematology, particularly benign hematology, was viewed as having poorer income potential, research funding, job availability, and job security than oncology.”

Regarding pay, Marshall said the good news is that many classical hematologists work in academia, where it’s common for pay to be “equitable across hematology/oncology divisions and based more on academic rank and other factors rather than subspecialty within hematology oncology.”

However, she noted, “this may differ at institutions where hematology and oncology are different departments. For example, centers where oncology is its own department, and hematology is part of the department of medicine.” 

As for job availability, Naik said that there’s plenty of demand. “In academics, it is clear that there are jobs available everywhere, but trainees are often worried about job prospects in private practice. While classical hematology jobs in private practice are not widely advertised, I can attest that there is no shortage of need,” she said. “Many private practices do not specifically advertise for classical hematologists because they assume that classical hematology experts are not available. But I assure you that every private practice my trainees have ever approached is always ecstatic to hire a classical hematologist.”

 

Why Are Mentors Important?

Mentorship is crucial to promoting the value of CH as a great career choice in a competitive environment, classical hematologists say. “We can motivate trainees by showing how the disease states themselves are so fascinating and how the treatments are showing great outcomes,” Nagalla said. “We can show positive results, how patient lives can be changed, and how well-respected across the system [we] are.”

As a selling point, classical hematologists like to emphasize that their field requires intensive detective work. “Let’s say a patient comes with anemia, which might have 15 different causes. You get some labs, and then you systemically rule in or rule out most of these on the differential diagnosis,” Nagalla said. “Then once you narrow it down, you get more labs. You keep going to the next step and next step, and so finally you come to a conclusion.”

As for therapy, Marshall said that “while for many cancers there are specific treatment recommendations for patients with a specific cancer type at a specific stage, there is not always a specific treatment recommendation (or a ‘right answer’) for our CH patients. Treatment planning depends strongly on a patient’s preferences, other medical conditions, and a discussion about risks [and] benefits of different treatment options such that two patients with the same condition may choose two different treatment options.”

Marshall also emphasizes to trainees that “CH is a broad field. Physicians and trainees are able to interact and collaborate with physicians in other specialties such as gastroenterology, cardiology, ob/gyn, and surgical specialties.” 

 

Does Research Support Mentorship in CH?

The 2019 study that revealed just 4.6% of fellows planned to go into CH found that “fellows who planned to enter hematology-only careers were significantly more likely to report having clinical training and mentorship experiences in hematology throughout their training relative to fellows with oncology-only or combined hematology/oncology career plans.”

Now there are more data to support mentorships. For a study published in Blood Advances in September 2024, Zoya Qureshy, MD, an internal medicine chief resident at the University of California at San Diego, and colleagues evaluated a year-long external membership program implemented by the American Society of Hematology (ASH) Medical Educators Institute. 

The program linked 35 US hematology/oncology fellows (80% female, 46% White, 35% Asian) who were interested in CH to 34 North American faculty members. The pairs were told to meet virtually once a month. 

Of 30 mentees and 23 mentors surveyed, 94% and 85%, respectively, said their pairings were good matches. Two thirds of the mentees accepted faculty positions in CH after their mentorships.

“Our study showed that external mentorship in a virtual format is feasible,” Qureshy said in an interview. “Additionally, external mentorship provided benefits such as different perspectives and the opportunity for mentorship for those who may not have it in their field of interest at their home institution.”

Qureshy added that “one strength of our mentorship program was that mentoring pairs were meticulously assigned based on shared interests and background. Many participants cited this common ground as a reason why they thought their mentoring pair was a good match.” 

There’s an important caveat: Most of the mentees weren’t new to CH. About 70% had previously worked with a mentor in the CH field, and 86% had previously conducted research in the field. 

 

What’s Next for Mentorship in CH?

The ASH Hematology-Focused Fellowship Training Program Consortium aims to mint 50 new academic hematologists by 2030 through programs at 12 institutions. “Mentorship is an exciting aspect of the program since it allows classical hematology trainees to form a network of peers nationally and also provides access to mentors across institutions,” Naik said. “And as the workforce grows, there will be more and more role models for future trainees to look up to.”

Moving forward, she said, “we hope to inspire even more institutions to adopt hematology training tracks throughout the country.”

Meanwhile, ASH’s new Classical Hematology Advancement Mentorship is taking applications for its debut 2025 program through January 9, 2025. Trainees will meet monthly with mentors both virtually and in person. Applicants must have been in their first or second year of hematology/oncology fellowship training at accredited programs in the United States as of July 15, 2024.

Naik, Marshall, Nagalla, and Qureshy have no relevant disclosures.

A version of this article appeared on Medscape.com.

For some medical students and trainees who go on to become hematologists, attraction to the field happens the first time they’re engrossed in figuring out what a blood smear is telling them. Others get drawn to hematology during a rotation in residency, when they encounter patients with hemophilia or sickle cell disease.

But when it comes to turning people on to the idea of a career in classical hematology (CH), there may be no more powerful influence than a mentor who loves their job. That’s why the field is focusing so much on supporting mentors and mentees amid a stark shortage of classical hematologists.

“Mentorship is key for maintaining trainee interest in the field and for providing role models for career growth,” said Rakhi P. Naik, MD, MHS, associate professor of medicine and director of the Hematology Fellowship Track at Johns Hopkins University, Baltimore, Maryland, in an interview. “This collaboration is especially critical because there are so few trainees and so few mentors currently in the field.”

Now there’s new research backing up the power of mentorship, even when it’s only provided virtually, and a brand-new program aims to unite more mentors and mentees.

Here’s a closer look at mentor-focused efforts to attract medical students to CH.

 

How Severe Is the Shortage in CH?

Patients with conditions treated by classical hematologists are waiting months for appointments at many outpatient centers, with some being forced to wait 6 months or more, said Srikanth Nagalla, MD, chief of benign hematology at Miami Cancer Institute, Florida, in an interview.

The shortage is creating dire problems in the inpatient setting too, Nagalla said. “Serious blood disorders like heparin-induced thrombocytopenia, acute chest syndrome [a complication of sickle cell disease], and thrombotic thrombocytopenic purpura have to be diagnosed and treated in a timely manner. If not, the morbidity and mortality are really high.”

If classical hematologists aren’t available, he said, oncologists and others not trained in hematology will need to cover these patients. 

Hematologist Ariela Marshall, MD, associate professor of medicine at the University of Minnesota in Minneapolis, noted in an interview that the CH shortage comes at a time when medical advances and an aging population are boosting the number of patients with noncancerous blood disorders. Older people are at greater risk for blood clots, she said. And lifespans for patients with bleeding and clotting disorders are rising thanks to effective new treatments.

“Because of our larger patient population in CH, we are going to need more classical hematologists to follow them for longer and longer periods of time,” she said. 

There’s no sign yet that newly minted physicians will take up the slack in CH. A 2019 study found that just 4.6% of 626 of hematology/oncology fellows said they planned to go into CH, also known as benign hematology, vs 67.1% who expected to treat patients with solid tumors, blood cancer, or both. The rest, 24.6%, planned to work in CH plus the two oncology fields.

 

Why Does a Shortage Exist?

“The reasons are complex, but one of the most important factors was the combining of the adult hematology and medical oncology training programs by the Accreditation Council for Graduate Medical Education in 1995,” Naik said. “After that time, the majority of fellowship training programs went from having separate programs for hematology and medical oncology to combining the training for the two specialties into one. Because most of these combined training programs resided within Cancer Centers, classical hematology training slowly became de-emphasized.”

As a result, fewer fellows ended up specializing in CH, she said. 

The field of CH also appears to suffer from a less than enticing reputation. According to a 2019 study coauthored by Marshall, surveys of thousands of hematology/oncology fellows found that “hematology, particularly benign hematology, was viewed as having poorer income potential, research funding, job availability, and job security than oncology.”

Regarding pay, Marshall said the good news is that many classical hematologists work in academia, where it’s common for pay to be “equitable across hematology/oncology divisions and based more on academic rank and other factors rather than subspecialty within hematology oncology.”

However, she noted, “this may differ at institutions where hematology and oncology are different departments. For example, centers where oncology is its own department, and hematology is part of the department of medicine.” 

As for job availability, Naik said that there’s plenty of demand. “In academics, it is clear that there are jobs available everywhere, but trainees are often worried about job prospects in private practice. While classical hematology jobs in private practice are not widely advertised, I can attest that there is no shortage of need,” she said. “Many private practices do not specifically advertise for classical hematologists because they assume that classical hematology experts are not available. But I assure you that every private practice my trainees have ever approached is always ecstatic to hire a classical hematologist.”

 

Why Are Mentors Important?

Mentorship is crucial to promoting the value of CH as a great career choice in a competitive environment, classical hematologists say. “We can motivate trainees by showing how the disease states themselves are so fascinating and how the treatments are showing great outcomes,” Nagalla said. “We can show positive results, how patient lives can be changed, and how well-respected across the system [we] are.”

As a selling point, classical hematologists like to emphasize that their field requires intensive detective work. “Let’s say a patient comes with anemia, which might have 15 different causes. You get some labs, and then you systemically rule in or rule out most of these on the differential diagnosis,” Nagalla said. “Then once you narrow it down, you get more labs. You keep going to the next step and next step, and so finally you come to a conclusion.”

As for therapy, Marshall said that “while for many cancers there are specific treatment recommendations for patients with a specific cancer type at a specific stage, there is not always a specific treatment recommendation (or a ‘right answer’) for our CH patients. Treatment planning depends strongly on a patient’s preferences, other medical conditions, and a discussion about risks [and] benefits of different treatment options such that two patients with the same condition may choose two different treatment options.”

Marshall also emphasizes to trainees that “CH is a broad field. Physicians and trainees are able to interact and collaborate with physicians in other specialties such as gastroenterology, cardiology, ob/gyn, and surgical specialties.” 

 

Does Research Support Mentorship in CH?

The 2019 study that revealed just 4.6% of fellows planned to go into CH found that “fellows who planned to enter hematology-only careers were significantly more likely to report having clinical training and mentorship experiences in hematology throughout their training relative to fellows with oncology-only or combined hematology/oncology career plans.”

Now there are more data to support mentorships. For a study published in Blood Advances in September 2024, Zoya Qureshy, MD, an internal medicine chief resident at the University of California at San Diego, and colleagues evaluated a year-long external membership program implemented by the American Society of Hematology (ASH) Medical Educators Institute. 

The program linked 35 US hematology/oncology fellows (80% female, 46% White, 35% Asian) who were interested in CH to 34 North American faculty members. The pairs were told to meet virtually once a month. 

Of 30 mentees and 23 mentors surveyed, 94% and 85%, respectively, said their pairings were good matches. Two thirds of the mentees accepted faculty positions in CH after their mentorships.

“Our study showed that external mentorship in a virtual format is feasible,” Qureshy said in an interview. “Additionally, external mentorship provided benefits such as different perspectives and the opportunity for mentorship for those who may not have it in their field of interest at their home institution.”

Qureshy added that “one strength of our mentorship program was that mentoring pairs were meticulously assigned based on shared interests and background. Many participants cited this common ground as a reason why they thought their mentoring pair was a good match.” 

There’s an important caveat: Most of the mentees weren’t new to CH. About 70% had previously worked with a mentor in the CH field, and 86% had previously conducted research in the field. 

 

What’s Next for Mentorship in CH?

The ASH Hematology-Focused Fellowship Training Program Consortium aims to mint 50 new academic hematologists by 2030 through programs at 12 institutions. “Mentorship is an exciting aspect of the program since it allows classical hematology trainees to form a network of peers nationally and also provides access to mentors across institutions,” Naik said. “And as the workforce grows, there will be more and more role models for future trainees to look up to.”

Moving forward, she said, “we hope to inspire even more institutions to adopt hematology training tracks throughout the country.”

Meanwhile, ASH’s new Classical Hematology Advancement Mentorship is taking applications for its debut 2025 program through January 9, 2025. Trainees will meet monthly with mentors both virtually and in person. Applicants must have been in their first or second year of hematology/oncology fellowship training at accredited programs in the United States as of July 15, 2024.

Naik, Marshall, Nagalla, and Qureshy have no relevant disclosures.

A version of this article appeared on Medscape.com.

President-elect Donald Trump’s vision for the nation’s top health agencies is coming into focus with three nominations announced Nov. 22 that drew both criticism and praise:

• Surgeon and health researcher Martin A. Makary, MD, MPH, to lead the US Food and Drug Administration (FDA).
• Former Republican congressman and physician David J. Weldon, MD, for director of the US Centers for Disease Control and Prevention (CDC).
• Fox News contributor Janette Nesheiwat, MD, for surgeon general.

Earlier in November, Trump nominated vaccine skeptic and former presidential candidate Robert F. Kennedy Jr. to lead the US Department of Health and Human Services (HHS). 

Here’s what to know about the latest nominees, who, like Kennedy, must be confirmed by the US Senate.

 

Martin A. Makary

Currently a professor at the Johns Hopkins School of Medicine and chief of islet transplant surgery at Johns Hopkins Hospital, Makary co-invented in 2006 a surgery checklist that became a widely-used patient safety tool. 

As a US FDA commissioner, Makary would preside over a $6.5 billion agency with more than 18,000 employees. The agency, part of HHS, oversees human and animal drugs and vaccines, medical devices, food, tobacco and other products. Some of Makary’s views align closely with those of HHS nominee Kennedy. 

Makary is also chief medical officer of telehealth platform Sesame.

Makary was primarily known as a health researcher and author of books about price transparency and the cost of health care until the COVID-19 pandemic, when he became an outspoken critic of the federal response, lambasting restrictions and mandates advocated by the CDC and other public health officials. 

In 2023, Makary told the House Select Subcommittee on the COVID Pandemic that federal officials had ignored what he called “natural immunity.” Studies have shown that natural immunity is “at least as effective as vaccinated immunity, and probably better,” testified Makary.

Makary called for an overhaul of the US FDA in a 2021 Fox News opinion, saying that its culture was “defined by counterproductive rigidity and a refusal to adapt.”

Blind Spots, his most recent book, takes on what he calls “medical dogma” and challenges conventional views on subjects ranging from the microbiome to marijuana to cancer prevention, hormone replacement therapy, antibiotics and peanut allergies.

In an interview he posted to X, Makary blames inappropriate use of antibiotics for a variety of childhood illnesses. He cites increases in obesity, learning disabilities, attention deficit disorder, asthma, celiac disease, ulcerative colitis and Crohn’s disease as all potentially causally related to antibiotics given in childhood.

Makary is an advisor to two conservative think tanks, the Foundation for Research on Equal Opportunity, and to Paragon Health Institute, begun in 2021 by two former top officials in the previous Trump administration.

Makary would “cut the bureaucratic red tape at the agency to make sure Americans get the medical cures and treatments they deserve,” Trump said on his social media platform, Truth Social, and in a press release.

While Los Angeles Times owner and physician-entrepreneur Patrick Soon-Shiong, MBBCh, MSc, praised the nomination of Makary (and the two other nominees) as “inspired,” other physicians criticized Makary for his anti-COVID mandate views and “fear-mongering” over COVID vaccine side effects.

 

Janette Nesheiwat

As surgeon general, Nesheiwat would serve as the top “health communicator in chief” and oversee the 6000 member US Public Health Service Commissioned Corps.

She is a frequent medical contributor to Fox News and serves as a medical director for a group of urgent care clinics in New York. She received her medical degree from the American University of the Caribbean School of Medicine and completed a family medicine residency at the University of Arkansas for Medical Sciences. She is board-certified in family medicine.

Nesheiwat sells vitamin supplements on her website and in December will publish a book on “miracles in medicine” and her Christian faith. 

Trump said in a statement that Nesheiwat “is a fierce advocate and strong communicator for preventive medicine and public health. She is committed to ensuring that Americans have access to affordable, quality healthcare, and believes in empowering individuals to take charge of their health to live longer, healthier lives.”

While Nesheiwat was critical of COVID mandates, she voiced more support for COVID vaccines and mask-wearing during the pandemic than her fellow nominees, leading some Trump supporters to criticize her nomination. 

“A good appointment, happy about this: I got to know @DoctorJanette during the pandemic, exchanging information. She is very smart, thoughtful, interested in learning, and a compassionate doctor, and…a truly nice person,” noted vaccine researcher Peter Hotez, MD, PhD, said on X.

 

David J. Weldon

If confirmed, former congressman Weldon would oversee the sprawling CDC, an agency with a roughly $17 billion budget, 15,000 employees or contractors, and numerous centers covering everything from health statistics to vaccines to epidemiology.

After earning his medical degree from the University at Buffalo School of Medicine, Weldon served in the US Army and US Army reserve. The Republican later served for 14 years in Congress representing Florida’s 15th district, which covers the Tampa region.

He now practices as an internist in Brevard County, Florida.

In Congress, Weldon raised concerns about the safety of some vaccines and promoted the false narrative that a former vaccine ingredient, thimerosal, caused autism, the Washington Post reported. Thimerosal has not been used in child vaccines for more than two decades. He also introduced a bill to move vaccine safety oversight from the CDC to an independent agency within HHS.

Trump said in a statement that Weldon “will proudly restore the CDC to its true purpose, and will work to end the Chronic Disease Epidemic.” 

But some physicians criticized Weldon for what they called his anti-vaccine views.

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

President-elect Donald Trump’s vision for the nation’s top health agencies is coming into focus with three nominations announced Nov. 22 that drew both criticism and praise:

• Surgeon and health researcher Martin A. Makary, MD, MPH, to lead the US Food and Drug Administration (FDA).
• Former Republican congressman and physician David J. Weldon, MD, for director of the US Centers for Disease Control and Prevention (CDC).
• Fox News contributor Janette Nesheiwat, MD, for surgeon general.

Earlier in November, Trump nominated vaccine skeptic and former presidential candidate Robert F. Kennedy Jr. to lead the US Department of Health and Human Services (HHS). 

Here’s what to know about the latest nominees, who, like Kennedy, must be confirmed by the US Senate.

 

Martin A. Makary

Currently a professor at the Johns Hopkins School of Medicine and chief of islet transplant surgery at Johns Hopkins Hospital, Makary co-invented in 2006 a surgery checklist that became a widely-used patient safety tool. 

As a US FDA commissioner, Makary would preside over a $6.5 billion agency with more than 18,000 employees. The agency, part of HHS, oversees human and animal drugs and vaccines, medical devices, food, tobacco and other products. Some of Makary’s views align closely with those of HHS nominee Kennedy. 

Makary is also chief medical officer of telehealth platform Sesame.

Makary was primarily known as a health researcher and author of books about price transparency and the cost of health care until the COVID-19 pandemic, when he became an outspoken critic of the federal response, lambasting restrictions and mandates advocated by the CDC and other public health officials. 

In 2023, Makary told the House Select Subcommittee on the COVID Pandemic that federal officials had ignored what he called “natural immunity.” Studies have shown that natural immunity is “at least as effective as vaccinated immunity, and probably better,” testified Makary.

Makary called for an overhaul of the US FDA in a 2021 Fox News opinion, saying that its culture was “defined by counterproductive rigidity and a refusal to adapt.”

Blind Spots, his most recent book, takes on what he calls “medical dogma” and challenges conventional views on subjects ranging from the microbiome to marijuana to cancer prevention, hormone replacement therapy, antibiotics and peanut allergies.

In an interview he posted to X, Makary blames inappropriate use of antibiotics for a variety of childhood illnesses. He cites increases in obesity, learning disabilities, attention deficit disorder, asthma, celiac disease, ulcerative colitis and Crohn’s disease as all potentially causally related to antibiotics given in childhood.

Makary is an advisor to two conservative think tanks, the Foundation for Research on Equal Opportunity, and to Paragon Health Institute, begun in 2021 by two former top officials in the previous Trump administration.

Makary would “cut the bureaucratic red tape at the agency to make sure Americans get the medical cures and treatments they deserve,” Trump said on his social media platform, Truth Social, and in a press release.

While Los Angeles Times owner and physician-entrepreneur Patrick Soon-Shiong, MBBCh, MSc, praised the nomination of Makary (and the two other nominees) as “inspired,” other physicians criticized Makary for his anti-COVID mandate views and “fear-mongering” over COVID vaccine side effects.

 

Janette Nesheiwat

As surgeon general, Nesheiwat would serve as the top “health communicator in chief” and oversee the 6000 member US Public Health Service Commissioned Corps.

She is a frequent medical contributor to Fox News and serves as a medical director for a group of urgent care clinics in New York. She received her medical degree from the American University of the Caribbean School of Medicine and completed a family medicine residency at the University of Arkansas for Medical Sciences. She is board-certified in family medicine.

Nesheiwat sells vitamin supplements on her website and in December will publish a book on “miracles in medicine” and her Christian faith. 

Trump said in a statement that Nesheiwat “is a fierce advocate and strong communicator for preventive medicine and public health. She is committed to ensuring that Americans have access to affordable, quality healthcare, and believes in empowering individuals to take charge of their health to live longer, healthier lives.”

While Nesheiwat was critical of COVID mandates, she voiced more support for COVID vaccines and mask-wearing during the pandemic than her fellow nominees, leading some Trump supporters to criticize her nomination. 

“A good appointment, happy about this: I got to know @DoctorJanette during the pandemic, exchanging information. She is very smart, thoughtful, interested in learning, and a compassionate doctor, and…a truly nice person,” noted vaccine researcher Peter Hotez, MD, PhD, said on X.

 

David J. Weldon

If confirmed, former congressman Weldon would oversee the sprawling CDC, an agency with a roughly $17 billion budget, 15,000 employees or contractors, and numerous centers covering everything from health statistics to vaccines to epidemiology.

After earning his medical degree from the University at Buffalo School of Medicine, Weldon served in the US Army and US Army reserve. The Republican later served for 14 years in Congress representing Florida’s 15th district, which covers the Tampa region.

He now practices as an internist in Brevard County, Florida.

In Congress, Weldon raised concerns about the safety of some vaccines and promoted the false narrative that a former vaccine ingredient, thimerosal, caused autism, the Washington Post reported. Thimerosal has not been used in child vaccines for more than two decades. He also introduced a bill to move vaccine safety oversight from the CDC to an independent agency within HHS.

Trump said in a statement that Weldon “will proudly restore the CDC to its true purpose, and will work to end the Chronic Disease Epidemic.” 

But some physicians criticized Weldon for what they called his anti-vaccine views.

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

President-elect Donald Trump’s vision for the nation’s top health agencies is coming into focus with three nominations announced Nov. 22 that drew both criticism and praise:

• Surgeon and health researcher Martin A. Makary, MD, MPH, to lead the US Food and Drug Administration (FDA).
• Former Republican congressman and physician David J. Weldon, MD, for director of the US Centers for Disease Control and Prevention (CDC).
• Fox News contributor Janette Nesheiwat, MD, for surgeon general.

Earlier in November, Trump nominated vaccine skeptic and former presidential candidate Robert F. Kennedy Jr. to lead the US Department of Health and Human Services (HHS). 

Here’s what to know about the latest nominees, who, like Kennedy, must be confirmed by the US Senate.

 

Martin A. Makary

Currently a professor at the Johns Hopkins School of Medicine and chief of islet transplant surgery at Johns Hopkins Hospital, Makary co-invented in 2006 a surgery checklist that became a widely-used patient safety tool. 

As a US FDA commissioner, Makary would preside over a $6.5 billion agency with more than 18,000 employees. The agency, part of HHS, oversees human and animal drugs and vaccines, medical devices, food, tobacco and other products. Some of Makary’s views align closely with those of HHS nominee Kennedy. 

Makary is also chief medical officer of telehealth platform Sesame.

Makary was primarily known as a health researcher and author of books about price transparency and the cost of health care until the COVID-19 pandemic, when he became an outspoken critic of the federal response, lambasting restrictions and mandates advocated by the CDC and other public health officials. 

In 2023, Makary told the House Select Subcommittee on the COVID Pandemic that federal officials had ignored what he called “natural immunity.” Studies have shown that natural immunity is “at least as effective as vaccinated immunity, and probably better,” testified Makary.

Makary called for an overhaul of the US FDA in a 2021 Fox News opinion, saying that its culture was “defined by counterproductive rigidity and a refusal to adapt.”

Blind Spots, his most recent book, takes on what he calls “medical dogma” and challenges conventional views on subjects ranging from the microbiome to marijuana to cancer prevention, hormone replacement therapy, antibiotics and peanut allergies.

In an interview he posted to X, Makary blames inappropriate use of antibiotics for a variety of childhood illnesses. He cites increases in obesity, learning disabilities, attention deficit disorder, asthma, celiac disease, ulcerative colitis and Crohn’s disease as all potentially causally related to antibiotics given in childhood.

Makary is an advisor to two conservative think tanks, the Foundation for Research on Equal Opportunity, and to Paragon Health Institute, begun in 2021 by two former top officials in the previous Trump administration.

Makary would “cut the bureaucratic red tape at the agency to make sure Americans get the medical cures and treatments they deserve,” Trump said on his social media platform, Truth Social, and in a press release.

While Los Angeles Times owner and physician-entrepreneur Patrick Soon-Shiong, MBBCh, MSc, praised the nomination of Makary (and the two other nominees) as “inspired,” other physicians criticized Makary for his anti-COVID mandate views and “fear-mongering” over COVID vaccine side effects.

 

Janette Nesheiwat

As surgeon general, Nesheiwat would serve as the top “health communicator in chief” and oversee the 6000 member US Public Health Service Commissioned Corps.

She is a frequent medical contributor to Fox News and serves as a medical director for a group of urgent care clinics in New York. She received her medical degree from the American University of the Caribbean School of Medicine and completed a family medicine residency at the University of Arkansas for Medical Sciences. She is board-certified in family medicine.

Nesheiwat sells vitamin supplements on her website and in December will publish a book on “miracles in medicine” and her Christian faith. 

Trump said in a statement that Nesheiwat “is a fierce advocate and strong communicator for preventive medicine and public health. She is committed to ensuring that Americans have access to affordable, quality healthcare, and believes in empowering individuals to take charge of their health to live longer, healthier lives.”

While Nesheiwat was critical of COVID mandates, she voiced more support for COVID vaccines and mask-wearing during the pandemic than her fellow nominees, leading some Trump supporters to criticize her nomination. 

“A good appointment, happy about this: I got to know @DoctorJanette during the pandemic, exchanging information. She is very smart, thoughtful, interested in learning, and a compassionate doctor, and…a truly nice person,” noted vaccine researcher Peter Hotez, MD, PhD, said on X.

 

David J. Weldon

If confirmed, former congressman Weldon would oversee the sprawling CDC, an agency with a roughly $17 billion budget, 15,000 employees or contractors, and numerous centers covering everything from health statistics to vaccines to epidemiology.

After earning his medical degree from the University at Buffalo School of Medicine, Weldon served in the US Army and US Army reserve. The Republican later served for 14 years in Congress representing Florida’s 15th district, which covers the Tampa region.

He now practices as an internist in Brevard County, Florida.

In Congress, Weldon raised concerns about the safety of some vaccines and promoted the false narrative that a former vaccine ingredient, thimerosal, caused autism, the Washington Post reported. Thimerosal has not been used in child vaccines for more than two decades. He also introduced a bill to move vaccine safety oversight from the CDC to an independent agency within HHS.

Trump said in a statement that Weldon “will proudly restore the CDC to its true purpose, and will work to end the Chronic Disease Epidemic.” 

But some physicians criticized Weldon for what they called his anti-vaccine views.

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

As a doctor with a keen interest in myeloma who is looking ahead to the 2024 American Society of Hematology (ASH) annual meeting December 7-10 in San Diego, I’ve chosen 10 presentations that seem most likely to make the greatest impact on my thinking and practice.

First, let me place my selected studies in context by acknowledging my biases. As a clinician, I’m prone to choosing clinical rather than basic science or translational work — even if translational work might well end up exerting a pivotal impact on practice in the future. And now — in no particular order — here are my picks: 

 

IFM 2017-03 Phase 3 Study 

Frail patients are underrepresented in most myeloma studies, yet in this randomized trial for newly diagnosed myeloma, exclusively frail patients were enrolled. The trial compared daratumumab/lenalidomide to lenalidomide/dexamethasone, and the most recent follow/up shows a progression-free survival (PFS) (48.5 vs 21 months) and overall survival (OS) (not reached vs 36 months) benefit to daratumumab/lenalidomide. What I see in practice is that anti-CD38 monoclonal antibodies are the best-tolerated drugs in this space and should be the backbone of any regimen for frail patients. Steroids should be omitted as early as possible. Future trials may optimize what to give in addition to the anti-CD38 therapy, and how to adapt/escalate therapy to frailty and clinical status, as lenalidomide remains difficult for such patients to tolerate.

AQUILA Study 

This is a randomized, phase 3 comparison of daratumumab to observation for patients with smoldering myeloma. The endpoint was PFS. For context, similar studies done in asymptomatic CLL have shown improved PFS, but not OS, and the authors of such studies have concluded that improvement in PFS alone should not justify a change in approach.

This study shows that daratumumab can improve laboratory markers and reduce progression (60-month PFS rates of 63.1% for daratumumab vs 40.8% with observation). However, several important caveats remain. The protocol only mandated spine/pelvis MRI imaging, not whole-body MRI imaging, and such imaging was only performed once a year, which may not be frequent enough to catch lesions at an earlier stage. These details have important implications, as previous research shows that up to half of lesions can be missed by doing only a spine MRI, as opposed to a whole-body MRI. 

All of this means that had more comprehensive imaging been done, many more patients may have been diagnosed with myeloma. Such patients may have been undertreated, and single agent daratumumab, with a response rate of just 63%, may not have been enough. Conversely, some patients may also have been overtreated using this approach, as the protocol allowed patients who had been diagnosed with smoldering myeloma up to 5 years earlier to be enrolled. Many of these people could have had indolent disease for years prior to enrollment and may not have ever progressed. 

Further information is needed to help us understand this study better. What was the nature of the progression events: asymptomatic lab changes or morbid end organ damage? Was daratumumab given when patients in the control arm progressed to myeloma? My concern is that if patients in the control arm do not universally receive modern daratumumab-containing therapies when they develop myeloma, then an overall survival advantage may be shown simply because patients in the intervention arm are getting a good drug earlier in the disease, while those in the control arm are not getting a good drug at all. Nevertheless, despite these limitations, it is likely this trial will lead to regulatory approval of daratumumab in this space, and lots of discussions from patients and clinicians alike.

 

Extended Follow-Up of Anito-Cel in its First In-Human study 

Two chimeric antigen receptor therapy (CAR-T) products are currently approved for myeloma. Cilta-cel is clearly effective but is associated with problematic late-onset neurological toxicities. Ide-cel appears much less effective. There is clearly a need for a product that is both effective and safe.

Anito-cel has two relevant abstracts this meeting that show much promise. Extended follow-up of anito-cel from its first in human study shows a promising 27-month PFS of 52%, and with no cases of delayed neurotoxicity. I also eagerly await further information from the registrational single-arm study of anti-cel being presented at ASH 2024, which should (hopefully) lead to its accelerated approval.

 

Screening for Myeloma for all People With Vertebral Fractures Likely Unnecessary 

This elegant study of over 9,000 people with vertebral fractures shows that absolute risks for myeloma were 0.43% and 0.63% in women and men with grade 2-3 fractures, respectively, indicating that there is likely little benefit in evaluating asymptomatic individuals with incidentally discovered vertebral fractures for myeloma, unless other features are present. Spread the word.

Further Information on Cevostamab, Another Bispecific Option

We do need effective treatments for targets beyond just BCMA and GPRC5D. Cevostamab, a bispecific targeting FCRH5, represents another option, with updated data on 167 patients. With an overall response rate of 43% (duration of response, 10 months), and a response rate of about 30% in those with prior bispecific exposure, this data helps us contextualize expected benefits as we look forward to the eventual approval of this drug. The efficacy is relatively modest in those who have already progressed on bispecifics, but cevostamab would still be a welcome addition to our arsenal.

Is GPRC5D a Better Target for Car T Rather Than Bispecifics? 

Our currently available GPRC5D bispecific (talquetamab) leads to high rates of skin, oral, and nail toxicity. This drug can also bring significant weight loss. These side effects make me consider that continuous targeting of GPRC5D through a bispecific may not be ideal, and that GPRC5D may be better as a one-time CAR T target. At ASH 2024, we will have 15-month follow-up data from BMS-986393, a GPRC5D CAR T. Response rates for this heavily pretreated population (76% of whom had triple refractory disease) were at 87%, with a median PFS of 14.5 months. Only 6% of patients experienced treatment-related weight loss, and nail (19%), skin (30%), and oral (31%) toxicities were relatively low. I look forward to updated data, as well as data on the resolution of the toxicities seen.

Daratumumab, a Game-Changer for AL Amyloidosis 

A truly effective drug given early can change the natural history of disease, even if patients in the control arm only receive the drug later. A case in point is daratumumab. The 5-year survival rate was 76.1% for the daratumumab/cyclophosphamide/bortezomib/dexamethasone arm and 64.7% for cyclophosphamide/bortezomib/dexamethasone arm. This happened despite the fact that 67% of the control arm patients (among those who received therapy) went on to receive daratumumab later in the disease course.

Understanding how SMM Progresses to MM 

We often hear that we should treat SMM and not just watch carefully because fractures may suddenly happen, or a patient may end up on dialysis. What this retrospective study tells us that amongst 427 patients with SMM, 42 had progression to myeloma, and amongst those 42, only 1 developed renal dysfunction (unclear if this resolved), and only 1 had lytic lesions that were symptomatic. The remainder were all asymptomatic lab and imaging changes. This is a retrospective study, and one should assume that follow-up was thus highly variable. It does not appear that diffusion weighted whole-body MRI imaging (our most sensitive imaging test) was employed universally or very frequently. Nevertheless, these powerful findings reassure us that, with close observation, morbidity is unlikely. Our group has designed a prospective study incorporating frequent diffusion weighted whole body MRI imaging to formally test this hypothesis (SPOTLIGHT, NCT06212323). 

The Underperformance of Daratumumab/Lenalidomide/Dexamethasone in the Real World 

At every major meeting I am reminded of the disconnect between real-world efficacy and clinical trial efficacy. Case in point: In this Austrian experience, daratumumab/lenalidomide/dexamethasone led to a PFS of 22.7 months vs 61.9 months in the MAIA trial of daratumumab/lenalidomide/dexamethasone. Such a sobering difference! And if you think this is an isolated experience, even in a US real-world cohort, consider that in a recently published comparative study dara/len/dex underperformed, although the time to next treatment or death was longer (37.8 months).

Delayed Neurotoxicity may not be Just a Consequence of high tumor burden 

We currently think that some of the scariest side effects of cilta-cel, such as delayed neurotoxicity, may be a consequence of a high number of cancer cells and may be prevented by better disease control at the time of infusion. This study, a sobering analysis of 52 patients with delayed neurotoxicity occurring after CAR T, included 8 patients (15%) who were not heavily pretreated, and all had less than 5% plasma cells at the time of infusion. None of these patients had extramedullary disease. This result worries me, especially because cilta-cel is being studied and is poised for future approval in earlier line settings. It suggests that this toxicity may not always be a product of disease burden, contrary to our current belief.

I will be paying close attention to these 10 myeloma studies at ASH 2024, where I look forward to meeting you and learning more.Dr. Mohyuddin is assistant professor in the multiple myeloma program at the Huntsman Cancer Institute at the University of Utah, Salt Lake City.

As a doctor with a keen interest in myeloma who is looking ahead to the 2024 American Society of Hematology (ASH) annual meeting December 7-10 in San Diego, I’ve chosen 10 presentations that seem most likely to make the greatest impact on my thinking and practice.

First, let me place my selected studies in context by acknowledging my biases. As a clinician, I’m prone to choosing clinical rather than basic science or translational work — even if translational work might well end up exerting a pivotal impact on practice in the future. And now — in no particular order — here are my picks: 

 

IFM 2017-03 Phase 3 Study 

Frail patients are underrepresented in most myeloma studies, yet in this randomized trial for newly diagnosed myeloma, exclusively frail patients were enrolled. The trial compared daratumumab/lenalidomide to lenalidomide/dexamethasone, and the most recent follow/up shows a progression-free survival (PFS) (48.5 vs 21 months) and overall survival (OS) (not reached vs 36 months) benefit to daratumumab/lenalidomide. What I see in practice is that anti-CD38 monoclonal antibodies are the best-tolerated drugs in this space and should be the backbone of any regimen for frail patients. Steroids should be omitted as early as possible. Future trials may optimize what to give in addition to the anti-CD38 therapy, and how to adapt/escalate therapy to frailty and clinical status, as lenalidomide remains difficult for such patients to tolerate.

AQUILA Study 

This is a randomized, phase 3 comparison of daratumumab to observation for patients with smoldering myeloma. The endpoint was PFS. For context, similar studies done in asymptomatic CLL have shown improved PFS, but not OS, and the authors of such studies have concluded that improvement in PFS alone should not justify a change in approach.

This study shows that daratumumab can improve laboratory markers and reduce progression (60-month PFS rates of 63.1% for daratumumab vs 40.8% with observation). However, several important caveats remain. The protocol only mandated spine/pelvis MRI imaging, not whole-body MRI imaging, and such imaging was only performed once a year, which may not be frequent enough to catch lesions at an earlier stage. These details have important implications, as previous research shows that up to half of lesions can be missed by doing only a spine MRI, as opposed to a whole-body MRI. 

All of this means that had more comprehensive imaging been done, many more patients may have been diagnosed with myeloma. Such patients may have been undertreated, and single agent daratumumab, with a response rate of just 63%, may not have been enough. Conversely, some patients may also have been overtreated using this approach, as the protocol allowed patients who had been diagnosed with smoldering myeloma up to 5 years earlier to be enrolled. Many of these people could have had indolent disease for years prior to enrollment and may not have ever progressed. 

Further information is needed to help us understand this study better. What was the nature of the progression events: asymptomatic lab changes or morbid end organ damage? Was daratumumab given when patients in the control arm progressed to myeloma? My concern is that if patients in the control arm do not universally receive modern daratumumab-containing therapies when they develop myeloma, then an overall survival advantage may be shown simply because patients in the intervention arm are getting a good drug earlier in the disease, while those in the control arm are not getting a good drug at all. Nevertheless, despite these limitations, it is likely this trial will lead to regulatory approval of daratumumab in this space, and lots of discussions from patients and clinicians alike.

 

Extended Follow-Up of Anito-Cel in its First In-Human study 

Two chimeric antigen receptor therapy (CAR-T) products are currently approved for myeloma. Cilta-cel is clearly effective but is associated with problematic late-onset neurological toxicities. Ide-cel appears much less effective. There is clearly a need for a product that is both effective and safe.

Anito-cel has two relevant abstracts this meeting that show much promise. Extended follow-up of anito-cel from its first in human study shows a promising 27-month PFS of 52%, and with no cases of delayed neurotoxicity. I also eagerly await further information from the registrational single-arm study of anti-cel being presented at ASH 2024, which should (hopefully) lead to its accelerated approval.

 

Screening for Myeloma for all People With Vertebral Fractures Likely Unnecessary 

This elegant study of over 9,000 people with vertebral fractures shows that absolute risks for myeloma were 0.43% and 0.63% in women and men with grade 2-3 fractures, respectively, indicating that there is likely little benefit in evaluating asymptomatic individuals with incidentally discovered vertebral fractures for myeloma, unless other features are present. Spread the word.

Further Information on Cevostamab, Another Bispecific Option

We do need effective treatments for targets beyond just BCMA and GPRC5D. Cevostamab, a bispecific targeting FCRH5, represents another option, with updated data on 167 patients. With an overall response rate of 43% (duration of response, 10 months), and a response rate of about 30% in those with prior bispecific exposure, this data helps us contextualize expected benefits as we look forward to the eventual approval of this drug. The efficacy is relatively modest in those who have already progressed on bispecifics, but cevostamab would still be a welcome addition to our arsenal.

Is GPRC5D a Better Target for Car T Rather Than Bispecifics? 

Our currently available GPRC5D bispecific (talquetamab) leads to high rates of skin, oral, and nail toxicity. This drug can also bring significant weight loss. These side effects make me consider that continuous targeting of GPRC5D through a bispecific may not be ideal, and that GPRC5D may be better as a one-time CAR T target. At ASH 2024, we will have 15-month follow-up data from BMS-986393, a GPRC5D CAR T. Response rates for this heavily pretreated population (76% of whom had triple refractory disease) were at 87%, with a median PFS of 14.5 months. Only 6% of patients experienced treatment-related weight loss, and nail (19%), skin (30%), and oral (31%) toxicities were relatively low. I look forward to updated data, as well as data on the resolution of the toxicities seen.

Daratumumab, a Game-Changer for AL Amyloidosis 

A truly effective drug given early can change the natural history of disease, even if patients in the control arm only receive the drug later. A case in point is daratumumab. The 5-year survival rate was 76.1% for the daratumumab/cyclophosphamide/bortezomib/dexamethasone arm and 64.7% for cyclophosphamide/bortezomib/dexamethasone arm. This happened despite the fact that 67% of the control arm patients (among those who received therapy) went on to receive daratumumab later in the disease course.

Understanding how SMM Progresses to MM 

We often hear that we should treat SMM and not just watch carefully because fractures may suddenly happen, or a patient may end up on dialysis. What this retrospective study tells us that amongst 427 patients with SMM, 42 had progression to myeloma, and amongst those 42, only 1 developed renal dysfunction (unclear if this resolved), and only 1 had lytic lesions that were symptomatic. The remainder were all asymptomatic lab and imaging changes. This is a retrospective study, and one should assume that follow-up was thus highly variable. It does not appear that diffusion weighted whole-body MRI imaging (our most sensitive imaging test) was employed universally or very frequently. Nevertheless, these powerful findings reassure us that, with close observation, morbidity is unlikely. Our group has designed a prospective study incorporating frequent diffusion weighted whole body MRI imaging to formally test this hypothesis (SPOTLIGHT, NCT06212323). 

The Underperformance of Daratumumab/Lenalidomide/Dexamethasone in the Real World 

At every major meeting I am reminded of the disconnect between real-world efficacy and clinical trial efficacy. Case in point: In this Austrian experience, daratumumab/lenalidomide/dexamethasone led to a PFS of 22.7 months vs 61.9 months in the MAIA trial of daratumumab/lenalidomide/dexamethasone. Such a sobering difference! And if you think this is an isolated experience, even in a US real-world cohort, consider that in a recently published comparative study dara/len/dex underperformed, although the time to next treatment or death was longer (37.8 months).

Delayed Neurotoxicity may not be Just a Consequence of high tumor burden 

We currently think that some of the scariest side effects of cilta-cel, such as delayed neurotoxicity, may be a consequence of a high number of cancer cells and may be prevented by better disease control at the time of infusion. This study, a sobering analysis of 52 patients with delayed neurotoxicity occurring after CAR T, included 8 patients (15%) who were not heavily pretreated, and all had less than 5% plasma cells at the time of infusion. None of these patients had extramedullary disease. This result worries me, especially because cilta-cel is being studied and is poised for future approval in earlier line settings. It suggests that this toxicity may not always be a product of disease burden, contrary to our current belief.

I will be paying close attention to these 10 myeloma studies at ASH 2024, where I look forward to meeting you and learning more.Dr. Mohyuddin is assistant professor in the multiple myeloma program at the Huntsman Cancer Institute at the University of Utah, Salt Lake City.

As a doctor with a keen interest in myeloma who is looking ahead to the 2024 American Society of Hematology (ASH) annual meeting December 7-10 in San Diego, I’ve chosen 10 presentations that seem most likely to make the greatest impact on my thinking and practice.

First, let me place my selected studies in context by acknowledging my biases. As a clinician, I’m prone to choosing clinical rather than basic science or translational work — even if translational work might well end up exerting a pivotal impact on practice in the future. And now — in no particular order — here are my picks: 

 

IFM 2017-03 Phase 3 Study 

Frail patients are underrepresented in most myeloma studies, yet in this randomized trial for newly diagnosed myeloma, exclusively frail patients were enrolled. The trial compared daratumumab/lenalidomide to lenalidomide/dexamethasone, and the most recent follow/up shows a progression-free survival (PFS) (48.5 vs 21 months) and overall survival (OS) (not reached vs 36 months) benefit to daratumumab/lenalidomide. What I see in practice is that anti-CD38 monoclonal antibodies are the best-tolerated drugs in this space and should be the backbone of any regimen for frail patients. Steroids should be omitted as early as possible. Future trials may optimize what to give in addition to the anti-CD38 therapy, and how to adapt/escalate therapy to frailty and clinical status, as lenalidomide remains difficult for such patients to tolerate.

AQUILA Study 

This is a randomized, phase 3 comparison of daratumumab to observation for patients with smoldering myeloma. The endpoint was PFS. For context, similar studies done in asymptomatic CLL have shown improved PFS, but not OS, and the authors of such studies have concluded that improvement in PFS alone should not justify a change in approach.

This study shows that daratumumab can improve laboratory markers and reduce progression (60-month PFS rates of 63.1% for daratumumab vs 40.8% with observation). However, several important caveats remain. The protocol only mandated spine/pelvis MRI imaging, not whole-body MRI imaging, and such imaging was only performed once a year, which may not be frequent enough to catch lesions at an earlier stage. These details have important implications, as previous research shows that up to half of lesions can be missed by doing only a spine MRI, as opposed to a whole-body MRI. 

All of this means that had more comprehensive imaging been done, many more patients may have been diagnosed with myeloma. Such patients may have been undertreated, and single agent daratumumab, with a response rate of just 63%, may not have been enough. Conversely, some patients may also have been overtreated using this approach, as the protocol allowed patients who had been diagnosed with smoldering myeloma up to 5 years earlier to be enrolled. Many of these people could have had indolent disease for years prior to enrollment and may not have ever progressed. 

Further information is needed to help us understand this study better. What was the nature of the progression events: asymptomatic lab changes or morbid end organ damage? Was daratumumab given when patients in the control arm progressed to myeloma? My concern is that if patients in the control arm do not universally receive modern daratumumab-containing therapies when they develop myeloma, then an overall survival advantage may be shown simply because patients in the intervention arm are getting a good drug earlier in the disease, while those in the control arm are not getting a good drug at all. Nevertheless, despite these limitations, it is likely this trial will lead to regulatory approval of daratumumab in this space, and lots of discussions from patients and clinicians alike.

 

Extended Follow-Up of Anito-Cel in its First In-Human study 

Two chimeric antigen receptor therapy (CAR-T) products are currently approved for myeloma. Cilta-cel is clearly effective but is associated with problematic late-onset neurological toxicities. Ide-cel appears much less effective. There is clearly a need for a product that is both effective and safe.

Anito-cel has two relevant abstracts this meeting that show much promise. Extended follow-up of anito-cel from its first in human study shows a promising 27-month PFS of 52%, and with no cases of delayed neurotoxicity. I also eagerly await further information from the registrational single-arm study of anti-cel being presented at ASH 2024, which should (hopefully) lead to its accelerated approval.

 

Screening for Myeloma for all People With Vertebral Fractures Likely Unnecessary 

This elegant study of over 9,000 people with vertebral fractures shows that absolute risks for myeloma were 0.43% and 0.63% in women and men with grade 2-3 fractures, respectively, indicating that there is likely little benefit in evaluating asymptomatic individuals with incidentally discovered vertebral fractures for myeloma, unless other features are present. Spread the word.

Further Information on Cevostamab, Another Bispecific Option

We do need effective treatments for targets beyond just BCMA and GPRC5D. Cevostamab, a bispecific targeting FCRH5, represents another option, with updated data on 167 patients. With an overall response rate of 43% (duration of response, 10 months), and a response rate of about 30% in those with prior bispecific exposure, this data helps us contextualize expected benefits as we look forward to the eventual approval of this drug. The efficacy is relatively modest in those who have already progressed on bispecifics, but cevostamab would still be a welcome addition to our arsenal.

Is GPRC5D a Better Target for Car T Rather Than Bispecifics? 

Our currently available GPRC5D bispecific (talquetamab) leads to high rates of skin, oral, and nail toxicity. This drug can also bring significant weight loss. These side effects make me consider that continuous targeting of GPRC5D through a bispecific may not be ideal, and that GPRC5D may be better as a one-time CAR T target. At ASH 2024, we will have 15-month follow-up data from BMS-986393, a GPRC5D CAR T. Response rates for this heavily pretreated population (76% of whom had triple refractory disease) were at 87%, with a median PFS of 14.5 months. Only 6% of patients experienced treatment-related weight loss, and nail (19%), skin (30%), and oral (31%) toxicities were relatively low. I look forward to updated data, as well as data on the resolution of the toxicities seen.

Daratumumab, a Game-Changer for AL Amyloidosis 

A truly effective drug given early can change the natural history of disease, even if patients in the control arm only receive the drug later. A case in point is daratumumab. The 5-year survival rate was 76.1% for the daratumumab/cyclophosphamide/bortezomib/dexamethasone arm and 64.7% for cyclophosphamide/bortezomib/dexamethasone arm. This happened despite the fact that 67% of the control arm patients (among those who received therapy) went on to receive daratumumab later in the disease course.

Understanding how SMM Progresses to MM 

We often hear that we should treat SMM and not just watch carefully because fractures may suddenly happen, or a patient may end up on dialysis. What this retrospective study tells us that amongst 427 patients with SMM, 42 had progression to myeloma, and amongst those 42, only 1 developed renal dysfunction (unclear if this resolved), and only 1 had lytic lesions that were symptomatic. The remainder were all asymptomatic lab and imaging changes. This is a retrospective study, and one should assume that follow-up was thus highly variable. It does not appear that diffusion weighted whole-body MRI imaging (our most sensitive imaging test) was employed universally or very frequently. Nevertheless, these powerful findings reassure us that, with close observation, morbidity is unlikely. Our group has designed a prospective study incorporating frequent diffusion weighted whole body MRI imaging to formally test this hypothesis (SPOTLIGHT, NCT06212323). 

The Underperformance of Daratumumab/Lenalidomide/Dexamethasone in the Real World 

At every major meeting I am reminded of the disconnect between real-world efficacy and clinical trial efficacy. Case in point: In this Austrian experience, daratumumab/lenalidomide/dexamethasone led to a PFS of 22.7 months vs 61.9 months in the MAIA trial of daratumumab/lenalidomide/dexamethasone. Such a sobering difference! And if you think this is an isolated experience, even in a US real-world cohort, consider that in a recently published comparative study dara/len/dex underperformed, although the time to next treatment or death was longer (37.8 months).

Delayed Neurotoxicity may not be Just a Consequence of high tumor burden 

We currently think that some of the scariest side effects of cilta-cel, such as delayed neurotoxicity, may be a consequence of a high number of cancer cells and may be prevented by better disease control at the time of infusion. This study, a sobering analysis of 52 patients with delayed neurotoxicity occurring after CAR T, included 8 patients (15%) who were not heavily pretreated, and all had less than 5% plasma cells at the time of infusion. None of these patients had extramedullary disease. This result worries me, especially because cilta-cel is being studied and is poised for future approval in earlier line settings. It suggests that this toxicity may not always be a product of disease burden, contrary to our current belief.

I will be paying close attention to these 10 myeloma studies at ASH 2024, where I look forward to meeting you and learning more.Dr. Mohyuddin is assistant professor in the multiple myeloma program at the Huntsman Cancer Institute at the University of Utah, Salt Lake City.

Summary and Key Highlights

Summary: This section explores why physicians often struggle to recognize burnout within themselves, partly due to stigma and a tendency to focus on productivity over well-being. Dr. Tyra Fainstad shares personal experiences of burnout symptoms, emphasizing the importance of awareness and self-reflection. Recognizing and addressing burnout early can help physicians find healthier coping strategies, avoid productivity traps, and seek support.

Key Takeaways:

• Many physicians struggle to identify burnout due to stigma and self-blame.
• Awareness of burnout symptoms is essential for early intervention and healthy coping.
• Seeking support can prevent burnout from worsening and improve quality of life.

Our Editors Also Recommend: 

Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’

Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance 

Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance 

A Transformative Rx for Burnout, Grief, and Illness: Dance 

 

Next Medscape Masters Event:

Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations.

Lotte Dyrbye, has disclosed the following relevant financial relationships: Co-inventor of the Well-being Index and its derivatives, which Mayo Clinic has licensed. Dyrbye receives royalties.

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

Summary and Key Highlights

Summary: This section explores why physicians often struggle to recognize burnout within themselves, partly due to stigma and a tendency to focus on productivity over well-being. Dr. Tyra Fainstad shares personal experiences of burnout symptoms, emphasizing the importance of awareness and self-reflection. Recognizing and addressing burnout early can help physicians find healthier coping strategies, avoid productivity traps, and seek support.

Key Takeaways:

• Many physicians struggle to identify burnout due to stigma and self-blame.
• Awareness of burnout symptoms is essential for early intervention and healthy coping.
• Seeking support can prevent burnout from worsening and improve quality of life.

Our Editors Also Recommend: 

Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’

Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance 

Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance 

A Transformative Rx for Burnout, Grief, and Illness: Dance 

 

Next Medscape Masters Event:

Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations.

Lotte Dyrbye, has disclosed the following relevant financial relationships: Co-inventor of the Well-being Index and its derivatives, which Mayo Clinic has licensed. Dyrbye receives royalties.

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

Summary and Key Highlights

Summary: This section explores why physicians often struggle to recognize burnout within themselves, partly due to stigma and a tendency to focus on productivity over well-being. Dr. Tyra Fainstad shares personal experiences of burnout symptoms, emphasizing the importance of awareness and self-reflection. Recognizing and addressing burnout early can help physicians find healthier coping strategies, avoid productivity traps, and seek support.

Key Takeaways:

• Many physicians struggle to identify burnout due to stigma and self-blame.
• Awareness of burnout symptoms is essential for early intervention and healthy coping.
• Seeking support can prevent burnout from worsening and improve quality of life.

Our Editors Also Recommend: 

Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’

Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance 

Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance 

A Transformative Rx for Burnout, Grief, and Illness: Dance 

 

Next Medscape Masters Event:

Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations.

Lotte Dyrbye, has disclosed the following relevant financial relationships: Co-inventor of the Well-being Index and its derivatives, which Mayo Clinic has licensed. Dyrbye receives royalties.

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

Summary and Key Highlights

Summary: Dr Tyra Fainstad explores the ingrained culture in medicine that encourages self-criticism, with many physicians feeling that they must be hard on themselves to succeed. Dr Fainstad challenges this belief, advocating for self-compassion as a healthier alternative. The evolving medical field now includes physicians who prioritize well-being without sacrificing quality of care, underscoring the importance of self-kindness for sustainable practice.

Key Takeaways:

• Many physicians believe that self-criticism is necessary for success, a mindset rooted in medical culture.
• Practicing self-compassion can improve long-term resilience and prevent burnout.
• The changing landscape of healthcare supports a more balanced approach to physician well-being.

Our Editors Also Recommend: 

Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’

Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance 

Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance 

A Transformative Rx for Burnout, Grief, and Illness: Dance 

 

Next Medscape Masters Event:

Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations. 

Lotte Dyrbye, has disclosed the following relevant financial relationships: Co-inventor of the Well-being Index and its derivatives, which Mayo Clinic has licensed. Dyrbye receives royalties.

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

Summary and Key Highlights

Summary: Dr Tyra Fainstad explores the ingrained culture in medicine that encourages self-criticism, with many physicians feeling that they must be hard on themselves to succeed. Dr Fainstad challenges this belief, advocating for self-compassion as a healthier alternative. The evolving medical field now includes physicians who prioritize well-being without sacrificing quality of care, underscoring the importance of self-kindness for sustainable practice.

Key Takeaways:

• Many physicians believe that self-criticism is necessary for success, a mindset rooted in medical culture.
• Practicing self-compassion can improve long-term resilience and prevent burnout.
• The changing landscape of healthcare supports a more balanced approach to physician well-being.

Our Editors Also Recommend: 

Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’

Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance 

Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance 

A Transformative Rx for Burnout, Grief, and Illness: Dance 

 

Next Medscape Masters Event:

Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations. 

Lotte Dyrbye, has disclosed the following relevant financial relationships: Co-inventor of the Well-being Index and its derivatives, which Mayo Clinic has licensed. Dyrbye receives royalties.

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

Summary and Key Highlights

Summary: Dr Tyra Fainstad explores the ingrained culture in medicine that encourages self-criticism, with many physicians feeling that they must be hard on themselves to succeed. Dr Fainstad challenges this belief, advocating for self-compassion as a healthier alternative. The evolving medical field now includes physicians who prioritize well-being without sacrificing quality of care, underscoring the importance of self-kindness for sustainable practice.

Key Takeaways:

• Many physicians believe that self-criticism is necessary for success, a mindset rooted in medical culture.
• Practicing self-compassion can improve long-term resilience and prevent burnout.
• The changing landscape of healthcare supports a more balanced approach to physician well-being.

Our Editors Also Recommend: 

Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’

Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance 

Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance 

A Transformative Rx for Burnout, Grief, and Illness: Dance 

 

Next Medscape Masters Event:

Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations. 

Lotte Dyrbye, has disclosed the following relevant financial relationships: Co-inventor of the Well-being Index and its derivatives, which Mayo Clinic has licensed. Dyrbye receives royalties.

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

Summary and Key Highlights

Summary: Dr Tyra Fainstad shares her personal experience with burnout and the journey to recovery through coaching and self-compassion. She describes the pressures of seeking validation through external achievements, which ultimately led to a crisis in self-worth after medical training. Through coaching, she learned to cultivate a sense of internal fulfillment, reconnecting with her passion for medicine and achieving a healthier balance.

Key Takeaways:

• Relying solely on external validation can deepen burnout and affect well-being.
• Coaching empowers physicians to develop self-compassion and sustainable coping strategies.
• Shifting from external to internal validation strengthens long-term fulfillment and job satisfaction.

Our Editors Also Recommend: 

Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’

Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance 

Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance 

A Transformative Rx for Burnout, Grief, and Illness: Dance 

 

Next Medscape Masters Event:

Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations.

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

Summary and Key Highlights

Summary: Dr Tyra Fainstad shares her personal experience with burnout and the journey to recovery through coaching and self-compassion. She describes the pressures of seeking validation through external achievements, which ultimately led to a crisis in self-worth after medical training. Through coaching, she learned to cultivate a sense of internal fulfillment, reconnecting with her passion for medicine and achieving a healthier balance.

Key Takeaways:

• Relying solely on external validation can deepen burnout and affect well-being.
• Coaching empowers physicians to develop self-compassion and sustainable coping strategies.
• Shifting from external to internal validation strengthens long-term fulfillment and job satisfaction.

Our Editors Also Recommend: 

Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’

Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance 

Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance 

A Transformative Rx for Burnout, Grief, and Illness: Dance 

 

Next Medscape Masters Event:

Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations.

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

Summary and Key Highlights

Summary: Dr Tyra Fainstad shares her personal experience with burnout and the journey to recovery through coaching and self-compassion. She describes the pressures of seeking validation through external achievements, which ultimately led to a crisis in self-worth after medical training. Through coaching, she learned to cultivate a sense of internal fulfillment, reconnecting with her passion for medicine and achieving a healthier balance.

Key Takeaways:

• Relying solely on external validation can deepen burnout and affect well-being.
• Coaching empowers physicians to develop self-compassion and sustainable coping strategies.
• Shifting from external to internal validation strengthens long-term fulfillment and job satisfaction.

Our Editors Also Recommend: 

Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’

Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance 

Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance 

A Transformative Rx for Burnout, Grief, and Illness: Dance 

 

Next Medscape Masters Event:

Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations.

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

The Food and Drug Administration has approved revumenib (Revuforj — Syndax Pharmaceuticals) for relapsed or refractory acute leukemia with a KMT2A gene translocation in adult and pediatric patients 1 year or older.

The approval makes the oral small-molecule menin inhibitor the first pharmaceutical to carry the indication. It blocks the binding of menin to mutated KMT2A fusion proteins, tamping down the process that leads to the disease.

Although a relatively uncommon form of leukemia, KMT2A rearrangements are a major driver of acute leukemia in infants.

Approval was based on a single-arm of the open-label AUGMENT-101 trial with 104 adult and pediatric patients with the mutation. Pediatric patients were at least 30 days old.

The rate of complete remission (CR) plus CR with partial hematologic recovery was 21.2% (22 patients) with a median duration of 6.4 months. The median time to remission was 1.9 months.

Eighty-three patients required blood cell and/or platelet transfusions at baseline; 12 (14%) did not need transfusions for 56 days afterward. Of the 21 who were transfusion free at baseline, 10 (48%) remained so over the same period.

The most common adverse reactions in 20% or more of patients were hemorrhage, nausea, increased phosphate, musculoskeletal pain, infection, increased aspartate aminotransferase, febrile neutropenia, increased alanine aminotransferase, increased intact parathyroid hormone, bacterial infection, diarrhea, differentiation syndrome, electrocardiogram QT prolonged, decreased phosphate, increased triglycerides, decreased potassium, decreased appetite, constipation, edema, viral infection, fatigue, and increased alkaline phosphatase.

The recommended dose varies by weight and concomitant use of strong CYP3A4 inhibitors. Because of an anticipated delay in commercial availability, the lowest strength dose of revumenib will be available through an expanded access program for patients who weigh < 40 kg.

A version of this article appeared on Medscape.com.

The Food and Drug Administration has approved revumenib (Revuforj — Syndax Pharmaceuticals) for relapsed or refractory acute leukemia with a KMT2A gene translocation in adult and pediatric patients 1 year or older.

The approval makes the oral small-molecule menin inhibitor the first pharmaceutical to carry the indication. It blocks the binding of menin to mutated KMT2A fusion proteins, tamping down the process that leads to the disease.

Although a relatively uncommon form of leukemia, KMT2A rearrangements are a major driver of acute leukemia in infants.

Approval was based on a single-arm of the open-label AUGMENT-101 trial with 104 adult and pediatric patients with the mutation. Pediatric patients were at least 30 days old.

The rate of complete remission (CR) plus CR with partial hematologic recovery was 21.2% (22 patients) with a median duration of 6.4 months. The median time to remission was 1.9 months.

Eighty-three patients required blood cell and/or platelet transfusions at baseline; 12 (14%) did not need transfusions for 56 days afterward. Of the 21 who were transfusion free at baseline, 10 (48%) remained so over the same period.

The most common adverse reactions in 20% or more of patients were hemorrhage, nausea, increased phosphate, musculoskeletal pain, infection, increased aspartate aminotransferase, febrile neutropenia, increased alanine aminotransferase, increased intact parathyroid hormone, bacterial infection, diarrhea, differentiation syndrome, electrocardiogram QT prolonged, decreased phosphate, increased triglycerides, decreased potassium, decreased appetite, constipation, edema, viral infection, fatigue, and increased alkaline phosphatase.

The recommended dose varies by weight and concomitant use of strong CYP3A4 inhibitors. Because of an anticipated delay in commercial availability, the lowest strength dose of revumenib will be available through an expanded access program for patients who weigh < 40 kg.

A version of this article appeared on Medscape.com.

The Food and Drug Administration has approved revumenib (Revuforj — Syndax Pharmaceuticals) for relapsed or refractory acute leukemia with a KMT2A gene translocation in adult and pediatric patients 1 year or older.

The approval makes the oral small-molecule menin inhibitor the first pharmaceutical to carry the indication. It blocks the binding of menin to mutated KMT2A fusion proteins, tamping down the process that leads to the disease.

Although a relatively uncommon form of leukemia, KMT2A rearrangements are a major driver of acute leukemia in infants.

Approval was based on a single-arm of the open-label AUGMENT-101 trial with 104 adult and pediatric patients with the mutation. Pediatric patients were at least 30 days old.

The rate of complete remission (CR) plus CR with partial hematologic recovery was 21.2% (22 patients) with a median duration of 6.4 months. The median time to remission was 1.9 months.

Eighty-three patients required blood cell and/or platelet transfusions at baseline; 12 (14%) did not need transfusions for 56 days afterward. Of the 21 who were transfusion free at baseline, 10 (48%) remained so over the same period.

The most common adverse reactions in 20% or more of patients were hemorrhage, nausea, increased phosphate, musculoskeletal pain, infection, increased aspartate aminotransferase, febrile neutropenia, increased alanine aminotransferase, increased intact parathyroid hormone, bacterial infection, diarrhea, differentiation syndrome, electrocardiogram QT prolonged, decreased phosphate, increased triglycerides, decreased potassium, decreased appetite, constipation, edema, viral infection, fatigue, and increased alkaline phosphatase.

The recommended dose varies by weight and concomitant use of strong CYP3A4 inhibitors. Because of an anticipated delay in commercial availability, the lowest strength dose of revumenib will be available through an expanded access program for patients who weigh < 40 kg.

A version of this article appeared on Medscape.com.

The 340B Drug Pricing Program began as a noble endeavor, a lifeline designed to help safety-net providers deliver affordable care to America’s most vulnerable populations. However, over the years, this well-intentioned program has strayed from its original purpose, becoming a lucrative space where profits often outweigh patients. Loopholes, lax oversight, and unchecked expansion have allowed some powerful players, such as certain disproportionate share hospitals and their “child sites” as well as for-profit pharmacies, to exploit the system. What was once a program to uplift underserved communities now risks becoming a case study in how good intentions can go astray without accountability.

What exactly is this “340B program” that has captured headlines and the interest of legislatures around the country? What ensures that pharmaceutical manufacturers continue to participate in this program? How lucrative is it? How have underserved populations benefited and how is that measured? 
 

The 340B Drug Pricing Program was established in 1992 under the Public Health Service Act. Its primary goal is to enable covered entities (such as hospitals and clinics serving low-income and uninsured patients) to purchase outpatient drugs from pharmaceutical manufacturers at significantly reduced prices in order to support their care of the low-income and underserved populations. Drug makers are required to participate in this program as a condition of their participation in Medicaid and Medicare Part B and offer these steep discounts to covered entities if they want their medications to be available to 38% of patients nationwide. 

The hospitals that make up 78% of the program’s spending are known as disproportionate share hospitals (DSHs). These hospitals must be nonprofit and have at least an 11.75% “disproportionate” share of low-income Medicare or Medicaid inpatients. The other types of non-hospital entities qualifying for 340B pricing are known as initial “federal grantees.” Some examples include federally qualified health centers (FQHC), Ryan White HIV/AIDS program grantees, and other types of specialized clinics, such as hemophilia treatment centers. It needs to be noted up front that it is not these initial non-hospital federal grantees that need more oversight or reform, since according to the Health Resources and Services Administration (HRSA) 2023 report they make up only 22% of all program spending. It is the large, predominantly DSH health systems that are profiting immensely through exponential growth of their clinics and contract pharmacies. However, these health systems have not been able to show exactly who are their eligible patients and how they have been benefiting them.

When the 340B program was established to offer financial relief to hospitals and clinics taking care of the uninsured, it allowed them to save 20%-50% on drug purchases, which could be reinvested in patient care services. It was hoped that savings from the program could be used to provide free or low-cost medications, free vaccines, and other essential health services, essentially allowing safety-net providers to serve their communities despite financial constraints. The initial grantees are fulfilling that mission, but there are concerns regarding DSHs. (See the Coalition of State Rheumatology Organization’s 340B explanatory statement and policy position for more.)

 

Why Should Independent Practice Physicians Care About This?

Independent doctors should care about the lack of oversight in the 340B program because it affects healthcare costs, patient assistance, market competition, and access to affordable care for underserved and uninsured patients.

It also plays a strong hand in the healthcare consolidation that continues to threaten private physician practices. These acquisitions threaten the viability of independent practices in a variety of specialties across the United States, including rheumatology. HRSA allows 340B-covered entities to register their off-campus outpatient facilities, or child sites, under their 340B designation. Covered entities can acquire drugs at the 340B price, while imposing markups on the reimbursement they submit to private insurance. The additional revenue these covered entities can pocket provides them with a cash flow advantage that physician practices and outpatient clinics will never be able to actualize. This uneven playing field may make rheumatology practices more susceptible to hospital acquisitions. In fact, between 2016 and 2022, large 340B hospitals were responsible for approximately 80% of hospital acquisitions.

Perhaps the most important reason that we should all be concerned about the trajectory of this well-meaning program is that we have seen patients with hospital debt being sued by DSHs who receive 340B discounts so that they can take care of the low-income patients they are suing. We have seen Medicaid patients be turned away from a DSH clinic after being discharged from that hospital, because the hospital had reached its disproportionate share (11.75%) of inpatient Medicare and Medicaid patients. While not illegal, that type of behavior by covered entities is WRONG! Oversight and reform are needed if the 340B program is going to live up to its purpose and not be just another well-intentioned program not fulfilling its mission.

 

Areas of Concern

There has been controversy regarding the limited oversight of the 340B program by HRSA, leading to abuse of the program. There are deep concerns regarding a lack of transparency in how savings from the program are being used, and there are concerns about the challenges associated with accurate tracking and reporting of 340B discounts, possibly leading to the duplication of discounts for both Medicaid and 340B. For example, a “duplicate discount” occurs if a manufacturer sells medications to a DSH at the 340B price and later pays a Medicaid rebate on the same drug. The extent of duplicate discounts in the 340B program is unknown. However, an audit of 1,536 cases conducted by HRSA between 2012 and 2019 found 429 instances of noncompliance related to duplicate discounts, which is nearly 30% of cases.

DSHs and their contracted pharmacies have been accused of exploiting the program by increasing the number of contract pharmacies and expanding the number of offsite outpatient clinics to maximize profits. As of mid-2024, the number of 340B contract pharmacies, counted by Drug Channels Institute (DCI), numbered 32,883 unique locations. According to DCI, the top five pharmacies in the program happen also to be among the top pharmacy revenue generators and are “for-profit.” They are CVS, Walgreens, Walmart, Express Scripts, and Optum RX. Additionally, a study in JAMA Health Forum showed that, from 2011 to 2019, contract pharmacies in areas with the lowest income decreased by 5.6% while those in the most affluent neighborhoods grew by 5%. 

There also has been tremendous growth in the number of covered entities in the 340B program, which grew from just over 8,100 in 2000 to 50,000 in 2020. Before 2004, DSHs made up less than 10% of these entities, but by 2020, they accounted for over 60%. Another study shows that DSHs are expanding their offsite outpatient clinics (“child clinics”) into the affluent neighborhoods serving commercially insured patients who are not low income, to capture the high commercial reimbursements for medications they acquired at steeply discounted prices. This clearly is diverting care away from the intended beneficiaries of the 340B program. 

Furthermore, DSHs have been acquiring specialty practices that prescribe some of the most expensive drugs, in order to take advantage of commercial reimbursement for medications that were acquired at the 340B discount price. Independent oncology practices have complained specifically about this happening in their area, where in some cases the DSHs have “stolen” their patients to profit off of the 340B pricing margins. This has the unintended consequence of increasing government spending, according to a study in the New England Journal of Medicine that showed price markups at 340B eligible hospitals were 6.59 times as high as those in independent physician practices after accounting for drug, patient, and geographic factors.

 

Legal Challenges and Legislation

On May 21, 2024, the US Court of Appeals for the DC Circuit issued a unanimous decision in favor of drug manufacturers, finding that certain manufacturer restrictions on the use of contract pharmacies under the 340B drug pricing program are permissible. The court’s decision follows a lower court (3rd Circuit) ruling which concluded that the 340B statute does not require manufacturers to deliver 340B drugs to an “unlimited number of contract pharmacies.” We’re still awaiting a decision from the 7th Circuit Court on a similar issue. If the 7th Circuit agrees with the government, creating a split decision, there is an increase in the likelihood that the Supreme Court would take up the case.

Johnson & Johnson has also sued the federal government for blocking their proposed use of a rebate model for DSHs that purchase through 340B two of its medications, Stelara and Xarelto, whose maximum fair price was negotiated through the Inflation Reduction Act’s Medicare Drug Price Negotiation Program. J&J states this would ensure that the claims are actually acquired and dispensed by a covered 340B entity, as well as ensuring there are no duplicate discounts as statutorily required by the IRA. When initially proposed, HRSA threatened to remove J&J’s access to Medicare and Medicaid if it pursued this change. J&J’s suit challenges that decision.

However, seven states (Arkansas, Kansas, Louisiana, Minnesota, Missouri, Mississippi, and West Virginia) have been active on this issue, passing laws to prevent manufacturers from limiting contract pharmacies’ ability to acquire 340B-discounted drugs. The model legislation also bans restrictions on the “number, location, ownership, or type of 340B contract pharmacy.”

It should also be noted that there are states that are looking for ways to encourage certain independent private practice specialties (such as gastroenterology and rheumatology) to see Medicaid patients, as well as increase testing for sexually transmitted diseases, by offering the possibility of obtaining 340B pricing in their clinics. 

Shifting our focus to Congress, six bipartisan Senators, known as the Group of 6, are working to modernize the 340B program, which hasn’t been updated since the original law in 1992. In 2024, legislation was introduced (see here and here) to reform a number of the features of the 340B drug discount program, including transparency, contract pharmacy requirements, and federal agency oversight.

 

Who’s Guarding the Hen House?

The Government Accountability Office and the Office of Inspector General over the last 5-10 years have asked HRSA to better define an “eligible” patient, to have more specifics concerning hospital eligibility criteria, and to have better oversight of the program to avoid duplicate discounts. HRSA has said that it doesn’t have the ability or the funding to achieve some of these goals. Consequently, little has been done on any of these fronts, creating frustration among pharmaceutical manufacturers and those calling for more oversight of the program to ensure that eligible patients are receiving the benefit of 340B pricing. Again, these frustrations are not pointed at the initial federally qualified centers or “grantees.”

HRSA now audits 200 covered entities a year, which is less than 2% of entities participating in the 340B program. HRSA expects the 340B entities themselves to have an oversight committee in place to ensure compliance with program requirements. 

So essentially, the fox is guarding the hen house?

Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at rhnews@mdedge.com.

The 340B Drug Pricing Program began as a noble endeavor, a lifeline designed to help safety-net providers deliver affordable care to America’s most vulnerable populations. However, over the years, this well-intentioned program has strayed from its original purpose, becoming a lucrative space where profits often outweigh patients. Loopholes, lax oversight, and unchecked expansion have allowed some powerful players, such as certain disproportionate share hospitals and their “child sites” as well as for-profit pharmacies, to exploit the system. What was once a program to uplift underserved communities now risks becoming a case study in how good intentions can go astray without accountability.

What exactly is this “340B program” that has captured headlines and the interest of legislatures around the country? What ensures that pharmaceutical manufacturers continue to participate in this program? How lucrative is it? How have underserved populations benefited and how is that measured? 
 

The 340B Drug Pricing Program was established in 1992 under the Public Health Service Act. Its primary goal is to enable covered entities (such as hospitals and clinics serving low-income and uninsured patients) to purchase outpatient drugs from pharmaceutical manufacturers at significantly reduced prices in order to support their care of the low-income and underserved populations. Drug makers are required to participate in this program as a condition of their participation in Medicaid and Medicare Part B and offer these steep discounts to covered entities if they want their medications to be available to 38% of patients nationwide. 

The hospitals that make up 78% of the program’s spending are known as disproportionate share hospitals (DSHs). These hospitals must be nonprofit and have at least an 11.75% “disproportionate” share of low-income Medicare or Medicaid inpatients. The other types of non-hospital entities qualifying for 340B pricing are known as initial “federal grantees.” Some examples include federally qualified health centers (FQHC), Ryan White HIV/AIDS program grantees, and other types of specialized clinics, such as hemophilia treatment centers. It needs to be noted up front that it is not these initial non-hospital federal grantees that need more oversight or reform, since according to the Health Resources and Services Administration (HRSA) 2023 report they make up only 22% of all program spending. It is the large, predominantly DSH health systems that are profiting immensely through exponential growth of their clinics and contract pharmacies. However, these health systems have not been able to show exactly who are their eligible patients and how they have been benefiting them.

When the 340B program was established to offer financial relief to hospitals and clinics taking care of the uninsured, it allowed them to save 20%-50% on drug purchases, which could be reinvested in patient care services. It was hoped that savings from the program could be used to provide free or low-cost medications, free vaccines, and other essential health services, essentially allowing safety-net providers to serve their communities despite financial constraints. The initial grantees are fulfilling that mission, but there are concerns regarding DSHs. (See the Coalition of State Rheumatology Organization’s 340B explanatory statement and policy position for more.)

 

Why Should Independent Practice Physicians Care About This?

Independent doctors should care about the lack of oversight in the 340B program because it affects healthcare costs, patient assistance, market competition, and access to affordable care for underserved and uninsured patients.

It also plays a strong hand in the healthcare consolidation that continues to threaten private physician practices. These acquisitions threaten the viability of independent practices in a variety of specialties across the United States, including rheumatology. HRSA allows 340B-covered entities to register their off-campus outpatient facilities, or child sites, under their 340B designation. Covered entities can acquire drugs at the 340B price, while imposing markups on the reimbursement they submit to private insurance. The additional revenue these covered entities can pocket provides them with a cash flow advantage that physician practices and outpatient clinics will never be able to actualize. This uneven playing field may make rheumatology practices more susceptible to hospital acquisitions. In fact, between 2016 and 2022, large 340B hospitals were responsible for approximately 80% of hospital acquisitions.

Perhaps the most important reason that we should all be concerned about the trajectory of this well-meaning program is that we have seen patients with hospital debt being sued by DSHs who receive 340B discounts so that they can take care of the low-income patients they are suing. We have seen Medicaid patients be turned away from a DSH clinic after being discharged from that hospital, because the hospital had reached its disproportionate share (11.75%) of inpatient Medicare and Medicaid patients. While not illegal, that type of behavior by covered entities is WRONG! Oversight and reform are needed if the 340B program is going to live up to its purpose and not be just another well-intentioned program not fulfilling its mission.

 

Areas of Concern

There has been controversy regarding the limited oversight of the 340B program by HRSA, leading to abuse of the program. There are deep concerns regarding a lack of transparency in how savings from the program are being used, and there are concerns about the challenges associated with accurate tracking and reporting of 340B discounts, possibly leading to the duplication of discounts for both Medicaid and 340B. For example, a “duplicate discount” occurs if a manufacturer sells medications to a DSH at the 340B price and later pays a Medicaid rebate on the same drug. The extent of duplicate discounts in the 340B program is unknown. However, an audit of 1,536 cases conducted by HRSA between 2012 and 2019 found 429 instances of noncompliance related to duplicate discounts, which is nearly 30% of cases.

DSHs and their contracted pharmacies have been accused of exploiting the program by increasing the number of contract pharmacies and expanding the number of offsite outpatient clinics to maximize profits. As of mid-2024, the number of 340B contract pharmacies, counted by Drug Channels Institute (DCI), numbered 32,883 unique locations. According to DCI, the top five pharmacies in the program happen also to be among the top pharmacy revenue generators and are “for-profit.” They are CVS, Walgreens, Walmart, Express Scripts, and Optum RX. Additionally, a study in JAMA Health Forum showed that, from 2011 to 2019, contract pharmacies in areas with the lowest income decreased by 5.6% while those in the most affluent neighborhoods grew by 5%. 

There also has been tremendous growth in the number of covered entities in the 340B program, which grew from just over 8,100 in 2000 to 50,000 in 2020. Before 2004, DSHs made up less than 10% of these entities, but by 2020, they accounted for over 60%. Another study shows that DSHs are expanding their offsite outpatient clinics (“child clinics”) into the affluent neighborhoods serving commercially insured patients who are not low income, to capture the high commercial reimbursements for medications they acquired at steeply discounted prices. This clearly is diverting care away from the intended beneficiaries of the 340B program. 

Furthermore, DSHs have been acquiring specialty practices that prescribe some of the most expensive drugs, in order to take advantage of commercial reimbursement for medications that were acquired at the 340B discount price. Independent oncology practices have complained specifically about this happening in their area, where in some cases the DSHs have “stolen” their patients to profit off of the 340B pricing margins. This has the unintended consequence of increasing government spending, according to a study in the New England Journal of Medicine that showed price markups at 340B eligible hospitals were 6.59 times as high as those in independent physician practices after accounting for drug, patient, and geographic factors.

 

Legal Challenges and Legislation

On May 21, 2024, the US Court of Appeals for the DC Circuit issued a unanimous decision in favor of drug manufacturers, finding that certain manufacturer restrictions on the use of contract pharmacies under the 340B drug pricing program are permissible. The court’s decision follows a lower court (3rd Circuit) ruling which concluded that the 340B statute does not require manufacturers to deliver 340B drugs to an “unlimited number of contract pharmacies.” We’re still awaiting a decision from the 7th Circuit Court on a similar issue. If the 7th Circuit agrees with the government, creating a split decision, there is an increase in the likelihood that the Supreme Court would take up the case.

Johnson & Johnson has also sued the federal government for blocking their proposed use of a rebate model for DSHs that purchase through 340B two of its medications, Stelara and Xarelto, whose maximum fair price was negotiated through the Inflation Reduction Act’s Medicare Drug Price Negotiation Program. J&J states this would ensure that the claims are actually acquired and dispensed by a covered 340B entity, as well as ensuring there are no duplicate discounts as statutorily required by the IRA. When initially proposed, HRSA threatened to remove J&J’s access to Medicare and Medicaid if it pursued this change. J&J’s suit challenges that decision.

However, seven states (Arkansas, Kansas, Louisiana, Minnesota, Missouri, Mississippi, and West Virginia) have been active on this issue, passing laws to prevent manufacturers from limiting contract pharmacies’ ability to acquire 340B-discounted drugs. The model legislation also bans restrictions on the “number, location, ownership, or type of 340B contract pharmacy.”

It should also be noted that there are states that are looking for ways to encourage certain independent private practice specialties (such as gastroenterology and rheumatology) to see Medicaid patients, as well as increase testing for sexually transmitted diseases, by offering the possibility of obtaining 340B pricing in their clinics. 

Shifting our focus to Congress, six bipartisan Senators, known as the Group of 6, are working to modernize the 340B program, which hasn’t been updated since the original law in 1992. In 2024, legislation was introduced (see here and here) to reform a number of the features of the 340B drug discount program, including transparency, contract pharmacy requirements, and federal agency oversight.

 

Who’s Guarding the Hen House?

The Government Accountability Office and the Office of Inspector General over the last 5-10 years have asked HRSA to better define an “eligible” patient, to have more specifics concerning hospital eligibility criteria, and to have better oversight of the program to avoid duplicate discounts. HRSA has said that it doesn’t have the ability or the funding to achieve some of these goals. Consequently, little has been done on any of these fronts, creating frustration among pharmaceutical manufacturers and those calling for more oversight of the program to ensure that eligible patients are receiving the benefit of 340B pricing. Again, these frustrations are not pointed at the initial federally qualified centers or “grantees.”

HRSA now audits 200 covered entities a year, which is less than 2% of entities participating in the 340B program. HRSA expects the 340B entities themselves to have an oversight committee in place to ensure compliance with program requirements. 

So essentially, the fox is guarding the hen house?

Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at rhnews@mdedge.com.

The 340B Drug Pricing Program began as a noble endeavor, a lifeline designed to help safety-net providers deliver affordable care to America’s most vulnerable populations. However, over the years, this well-intentioned program has strayed from its original purpose, becoming a lucrative space where profits often outweigh patients. Loopholes, lax oversight, and unchecked expansion have allowed some powerful players, such as certain disproportionate share hospitals and their “child sites” as well as for-profit pharmacies, to exploit the system. What was once a program to uplift underserved communities now risks becoming a case study in how good intentions can go astray without accountability.

What exactly is this “340B program” that has captured headlines and the interest of legislatures around the country? What ensures that pharmaceutical manufacturers continue to participate in this program? How lucrative is it? How have underserved populations benefited and how is that measured? 
 

The 340B Drug Pricing Program was established in 1992 under the Public Health Service Act. Its primary goal is to enable covered entities (such as hospitals and clinics serving low-income and uninsured patients) to purchase outpatient drugs from pharmaceutical manufacturers at significantly reduced prices in order to support their care of the low-income and underserved populations. Drug makers are required to participate in this program as a condition of their participation in Medicaid and Medicare Part B and offer these steep discounts to covered entities if they want their medications to be available to 38% of patients nationwide. 

The hospitals that make up 78% of the program’s spending are known as disproportionate share hospitals (DSHs). These hospitals must be nonprofit and have at least an 11.75% “disproportionate” share of low-income Medicare or Medicaid inpatients. The other types of non-hospital entities qualifying for 340B pricing are known as initial “federal grantees.” Some examples include federally qualified health centers (FQHC), Ryan White HIV/AIDS program grantees, and other types of specialized clinics, such as hemophilia treatment centers. It needs to be noted up front that it is not these initial non-hospital federal grantees that need more oversight or reform, since according to the Health Resources and Services Administration (HRSA) 2023 report they make up only 22% of all program spending. It is the large, predominantly DSH health systems that are profiting immensely through exponential growth of their clinics and contract pharmacies. However, these health systems have not been able to show exactly who are their eligible patients and how they have been benefiting them.

When the 340B program was established to offer financial relief to hospitals and clinics taking care of the uninsured, it allowed them to save 20%-50% on drug purchases, which could be reinvested in patient care services. It was hoped that savings from the program could be used to provide free or low-cost medications, free vaccines, and other essential health services, essentially allowing safety-net providers to serve their communities despite financial constraints. The initial grantees are fulfilling that mission, but there are concerns regarding DSHs. (See the Coalition of State Rheumatology Organization’s 340B explanatory statement and policy position for more.)

 

Why Should Independent Practice Physicians Care About This?

Independent doctors should care about the lack of oversight in the 340B program because it affects healthcare costs, patient assistance, market competition, and access to affordable care for underserved and uninsured patients.

It also plays a strong hand in the healthcare consolidation that continues to threaten private physician practices. These acquisitions threaten the viability of independent practices in a variety of specialties across the United States, including rheumatology. HRSA allows 340B-covered entities to register their off-campus outpatient facilities, or child sites, under their 340B designation. Covered entities can acquire drugs at the 340B price, while imposing markups on the reimbursement they submit to private insurance. The additional revenue these covered entities can pocket provides them with a cash flow advantage that physician practices and outpatient clinics will never be able to actualize. This uneven playing field may make rheumatology practices more susceptible to hospital acquisitions. In fact, between 2016 and 2022, large 340B hospitals were responsible for approximately 80% of hospital acquisitions.

Perhaps the most important reason that we should all be concerned about the trajectory of this well-meaning program is that we have seen patients with hospital debt being sued by DSHs who receive 340B discounts so that they can take care of the low-income patients they are suing. We have seen Medicaid patients be turned away from a DSH clinic after being discharged from that hospital, because the hospital had reached its disproportionate share (11.75%) of inpatient Medicare and Medicaid patients. While not illegal, that type of behavior by covered entities is WRONG! Oversight and reform are needed if the 340B program is going to live up to its purpose and not be just another well-intentioned program not fulfilling its mission.

 

Areas of Concern

There has been controversy regarding the limited oversight of the 340B program by HRSA, leading to abuse of the program. There are deep concerns regarding a lack of transparency in how savings from the program are being used, and there are concerns about the challenges associated with accurate tracking and reporting of 340B discounts, possibly leading to the duplication of discounts for both Medicaid and 340B. For example, a “duplicate discount” occurs if a manufacturer sells medications to a DSH at the 340B price and later pays a Medicaid rebate on the same drug. The extent of duplicate discounts in the 340B program is unknown. However, an audit of 1,536 cases conducted by HRSA between 2012 and 2019 found 429 instances of noncompliance related to duplicate discounts, which is nearly 30% of cases.

DSHs and their contracted pharmacies have been accused of exploiting the program by increasing the number of contract pharmacies and expanding the number of offsite outpatient clinics to maximize profits. As of mid-2024, the number of 340B contract pharmacies, counted by Drug Channels Institute (DCI), numbered 32,883 unique locations. According to DCI, the top five pharmacies in the program happen also to be among the top pharmacy revenue generators and are “for-profit.” They are CVS, Walgreens, Walmart, Express Scripts, and Optum RX. Additionally, a study in JAMA Health Forum showed that, from 2011 to 2019, contract pharmacies in areas with the lowest income decreased by 5.6% while those in the most affluent neighborhoods grew by 5%. 

There also has been tremendous growth in the number of covered entities in the 340B program, which grew from just over 8,100 in 2000 to 50,000 in 2020. Before 2004, DSHs made up less than 10% of these entities, but by 2020, they accounted for over 60%. Another study shows that DSHs are expanding their offsite outpatient clinics (“child clinics”) into the affluent neighborhoods serving commercially insured patients who are not low income, to capture the high commercial reimbursements for medications they acquired at steeply discounted prices. This clearly is diverting care away from the intended beneficiaries of the 340B program. 

Furthermore, DSHs have been acquiring specialty practices that prescribe some of the most expensive drugs, in order to take advantage of commercial reimbursement for medications that were acquired at the 340B discount price. Independent oncology practices have complained specifically about this happening in their area, where in some cases the DSHs have “stolen” their patients to profit off of the 340B pricing margins. This has the unintended consequence of increasing government spending, according to a study in the New England Journal of Medicine that showed price markups at 340B eligible hospitals were 6.59 times as high as those in independent physician practices after accounting for drug, patient, and geographic factors.

 

Legal Challenges and Legislation

On May 21, 2024, the US Court of Appeals for the DC Circuit issued a unanimous decision in favor of drug manufacturers, finding that certain manufacturer restrictions on the use of contract pharmacies under the 340B drug pricing program are permissible. The court’s decision follows a lower court (3rd Circuit) ruling which concluded that the 340B statute does not require manufacturers to deliver 340B drugs to an “unlimited number of contract pharmacies.” We’re still awaiting a decision from the 7th Circuit Court on a similar issue. If the 7th Circuit agrees with the government, creating a split decision, there is an increase in the likelihood that the Supreme Court would take up the case.

Johnson & Johnson has also sued the federal government for blocking their proposed use of a rebate model for DSHs that purchase through 340B two of its medications, Stelara and Xarelto, whose maximum fair price was negotiated through the Inflation Reduction Act’s Medicare Drug Price Negotiation Program. J&J states this would ensure that the claims are actually acquired and dispensed by a covered 340B entity, as well as ensuring there are no duplicate discounts as statutorily required by the IRA. When initially proposed, HRSA threatened to remove J&J’s access to Medicare and Medicaid if it pursued this change. J&J’s suit challenges that decision.

However, seven states (Arkansas, Kansas, Louisiana, Minnesota, Missouri, Mississippi, and West Virginia) have been active on this issue, passing laws to prevent manufacturers from limiting contract pharmacies’ ability to acquire 340B-discounted drugs. The model legislation also bans restrictions on the “number, location, ownership, or type of 340B contract pharmacy.”

It should also be noted that there are states that are looking for ways to encourage certain independent private practice specialties (such as gastroenterology and rheumatology) to see Medicaid patients, as well as increase testing for sexually transmitted diseases, by offering the possibility of obtaining 340B pricing in their clinics. 

Shifting our focus to Congress, six bipartisan Senators, known as the Group of 6, are working to modernize the 340B program, which hasn’t been updated since the original law in 1992. In 2024, legislation was introduced (see here and here) to reform a number of the features of the 340B drug discount program, including transparency, contract pharmacy requirements, and federal agency oversight.

 

Who’s Guarding the Hen House?

The Government Accountability Office and the Office of Inspector General over the last 5-10 years have asked HRSA to better define an “eligible” patient, to have more specifics concerning hospital eligibility criteria, and to have better oversight of the program to avoid duplicate discounts. HRSA has said that it doesn’t have the ability or the funding to achieve some of these goals. Consequently, little has been done on any of these fronts, creating frustration among pharmaceutical manufacturers and those calling for more oversight of the program to ensure that eligible patients are receiving the benefit of 340B pricing. Again, these frustrations are not pointed at the initial federally qualified centers or “grantees.”

HRSA now audits 200 covered entities a year, which is less than 2% of entities participating in the 340B program. HRSA expects the 340B entities themselves to have an oversight committee in place to ensure compliance with program requirements. 

So essentially, the fox is guarding the hen house?

Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at rhnews@mdedge.com.

If the name of leukemia specialist Jorge Cortes, MD, appears any more often in PubMed, they’ll need to name a wing after him. 

Over 30 years, Cortes has led or coauthored hundreds of studies, including many trials of landmark drugs to treat chronic myeloid leukemia (CML). His work has helped transform CML into an often-survivable disease instead of one that took the lives of most patients within 5 years.

“It’s been remarkable to see the evolution in CML and to be part of that transition as a fellow, as faculty, and as leader of some of the trials,” said Cortes, who directs the Georgia Cancer Center at Augusta University. “I’m the luckiest person in the world.”

In an interview, Cortes talked about his youth in Mexico, his research path, and his close connections to cancer medicine in Latin America.

Q: You grew up in Mexico City. What was your family like?

A: “My father grew up very poor in a small town in Michoacán in the southwest part of Mexico. In Mexico City, he had a tiny grocery store in an old-fashioned market, and we were lower middle class.

One of the things I learned was to work hard. There’s nobody I know who worked as hard as my father. He opened his store every day of the year, [Mexican] Independence Day or New Year’s or Christmas. He worked hard so we could have a better life than he did.

We learned English from a very young age. My elementary school was called Westminster School because he wanted a school where we would learn English.

As for my mother, she stayed with us [at home] and made sure we did our homework and were taken care of. I learned about being honest and dedicating to what you were doing.”

Q: You trained at the Salvador Zubirán National Institute of Health Sciences and Nutrition in Mexico City. Then what happened? 

A: “Through encouragement by my dermatologist older brother and a mentor at the institution where I was training as a hematologist, I decided to come to the United States.

My initial focus was going to be on coagulation and thrombosis. I came to Houston (Texas) for a fellowship at the University of Texas Health Science Center.

Then I started doing my rotation for the malignant part of the fellowship at MD Anderson Cancer Center [Houston]. One of my first rotations was with Susan M. O’Brien, [MD,] who became my greatest mentor throughout my career. I really enjoyed my rotation. I thought she was great clinically, and she was doing research and teaching. That’s what I wanted for my career.”

Q: What drew you to leukemia specifically?

A: “Dr O’Brien worked in leukemia during my initial rotation, and I really loved it. It was hard work, but it was very inspiring to see the clinical research and the things you could for patients. She had a lot of joy doing that. 

I told my program director I’d change and transfer to MD Anderson, and I ended up staying at MD Anderson for 23 years.”

Q: What was leukemia research like in those days?

A: “We didn’t have the understanding of the biology and the new drugs that we have now. When I started in Mexico, we didn’t even have hydroxyurea. What we were doing was much more basic. But still, the field sounded like a great field to be involved with because they were doing so many trials and had an outstanding database. 

Because of the influence of Dr [Moshe] Talpaz, [MD,] I started getting very involved with CML. In my initial years as a young faculty, I started working with him on interferon. Then imatinib appeared. I saw even from the phase 1 study how impressive the outcomes were in patients who had no response to anything and were in bad shape.”

Q: What CML medications have you worked on?

A: “I’ve been involved with all of them. Imatinib early on, then I led trials with dasatinib and nilotinib. Then, I led the registration trials of bosutinib and ponatinib. More recently, I was part of the development of asciminib.”

Q: What were some of the biggest challenges in CML research?

A: “We had an opportunity to do a lot of analysis about TKIs [tyrosine kinase inhibitors] when these were new drugs. It was a very steep curve of learning, how to monitor and manage side effects.

Then patients were starting to have resistance to two to three TKIs. Ponatinib came along, and it was an incredibly effective drug. But after it was approved, we started to recognize the occurrence of heart attacks and strokes.

That was unexpected and not something that was known for any TKI. It was a big challenge. The drug was taken off the market for some time, and trials were put on hold by the FDA [US Food and Drug Administration].

We scrambled to understand the mechanism of action. For a year or two, it was a stressful time. But eventually we moved past it, and we learned a lot.”

Q: What sort of work have you done in Latin America?

A: “I’ve always been very close to Latin America. I have many good friends and colleagues there, and I’ve always been interested in working with them. 

We’ve done research and studies and created an organization called Latin American Leukemia Net to develop more trials in Latin America. The most rewarding thing has been the educational programs for patients that we’ve done, helping them understand the disease, the treatments, and the goals of treatment. 

We’ve conducted a number of programs, and they have been effective, well-attended, and well received. I still work with my colleagues to develop local guidelines and do collaborative research.”

Q: What convinced you to leave MD Anderson for Georgia?

A: “I never thought I’d leave MD Anderson. I had my well-oiled machine of clinical trials, my clinic, and my fellowship program. But the one thing that I wanted to see if I could try next was to develop an institution.

That was the goal here, to take the Georgia Cancer Center to NCI [National Cancer Institute] designation. So, I thought, ‘That’s a nice challenge.’ It may be a good opportunity to try a different aspect of what it means to be an oncologist.

There are days that you think, ‘What am I doing here?’ when you have to deal with budgets and personnel and all these things. But it’s part of the process. It’s still good to know that we have a goal, and that we’re going to make it. 

Also, I still see my patients, and I enjoy that I still do some research and mentoring.”

Q: What’s the current state of CML treatment?

A: “Many patients have a pretty much normal life expectancy while [on therapy]. Still, one of the goals of many patients is to stop therapy. But that’s a reality only for a small percentage of patients. How can we make that happen for more patients?”

Q: By stopping therapy, do you mean curing the cancer?

A: “Yes, pretty much. You have a good response, you stop the therapy, and it doesn’t come back.

There are also patients who really don’t do well. We hear about CML being with a disease with such a good outcome, but we have patients for whom nothing works. Is it a matter of [needing] another TKI, or do we need to look at something else?”

Q: What do you see on the horizon?

A: “We are developing new approaches like combination therapies. We’re scratching the surface on that. We need to understand which combinations work, and where and when.

And we can make more efficient uses of the drugs we have now in terms of which ones to use when, the doses, the safety profiles. I think we can do better.”

Cortes disclosed consulting for Amphivena, Astellas, Bio-Path, BioLineRx, Bristol Myers Squibb, Daiichi Sankyo, Jazz, Novartis, Pfizer, and Takeda and research funding from Astellas Pharma, Bristol Myers Squibb, Daiichi Sankyo, Immunogen, Jazz, Merus, Novartis, Pfizer, Sun Pharma, Takeda, Tolero and Trovagene.
 

A version of this article appeared on Medscape.com.

If the name of leukemia specialist Jorge Cortes, MD, appears any more often in PubMed, they’ll need to name a wing after him. 

Over 30 years, Cortes has led or coauthored hundreds of studies, including many trials of landmark drugs to treat chronic myeloid leukemia (CML). His work has helped transform CML into an often-survivable disease instead of one that took the lives of most patients within 5 years.

“It’s been remarkable to see the evolution in CML and to be part of that transition as a fellow, as faculty, and as leader of some of the trials,” said Cortes, who directs the Georgia Cancer Center at Augusta University. “I’m the luckiest person in the world.”

In an interview, Cortes talked about his youth in Mexico, his research path, and his close connections to cancer medicine in Latin America.

Q: You grew up in Mexico City. What was your family like?

A: “My father grew up very poor in a small town in Michoacán in the southwest part of Mexico. In Mexico City, he had a tiny grocery store in an old-fashioned market, and we were lower middle class.

One of the things I learned was to work hard. There’s nobody I know who worked as hard as my father. He opened his store every day of the year, [Mexican] Independence Day or New Year’s or Christmas. He worked hard so we could have a better life than he did.

We learned English from a very young age. My elementary school was called Westminster School because he wanted a school where we would learn English.

As for my mother, she stayed with us [at home] and made sure we did our homework and were taken care of. I learned about being honest and dedicating to what you were doing.”

Q: You trained at the Salvador Zubirán National Institute of Health Sciences and Nutrition in Mexico City. Then what happened? 

A: “Through encouragement by my dermatologist older brother and a mentor at the institution where I was training as a hematologist, I decided to come to the United States.

My initial focus was going to be on coagulation and thrombosis. I came to Houston (Texas) for a fellowship at the University of Texas Health Science Center.

Then I started doing my rotation for the malignant part of the fellowship at MD Anderson Cancer Center [Houston]. One of my first rotations was with Susan M. O’Brien, [MD,] who became my greatest mentor throughout my career. I really enjoyed my rotation. I thought she was great clinically, and she was doing research and teaching. That’s what I wanted for my career.”

Q: What drew you to leukemia specifically?

A: “Dr O’Brien worked in leukemia during my initial rotation, and I really loved it. It was hard work, but it was very inspiring to see the clinical research and the things you could for patients. She had a lot of joy doing that. 

I told my program director I’d change and transfer to MD Anderson, and I ended up staying at MD Anderson for 23 years.”

Q: What was leukemia research like in those days?

A: “We didn’t have the understanding of the biology and the new drugs that we have now. When I started in Mexico, we didn’t even have hydroxyurea. What we were doing was much more basic. But still, the field sounded like a great field to be involved with because they were doing so many trials and had an outstanding database. 

Because of the influence of Dr [Moshe] Talpaz, [MD,] I started getting very involved with CML. In my initial years as a young faculty, I started working with him on interferon. Then imatinib appeared. I saw even from the phase 1 study how impressive the outcomes were in patients who had no response to anything and were in bad shape.”

Q: What CML medications have you worked on?

A: “I’ve been involved with all of them. Imatinib early on, then I led trials with dasatinib and nilotinib. Then, I led the registration trials of bosutinib and ponatinib. More recently, I was part of the development of asciminib.”

Q: What were some of the biggest challenges in CML research?

A: “We had an opportunity to do a lot of analysis about TKIs [tyrosine kinase inhibitors] when these were new drugs. It was a very steep curve of learning, how to monitor and manage side effects.

Then patients were starting to have resistance to two to three TKIs. Ponatinib came along, and it was an incredibly effective drug. But after it was approved, we started to recognize the occurrence of heart attacks and strokes.

That was unexpected and not something that was known for any TKI. It was a big challenge. The drug was taken off the market for some time, and trials were put on hold by the FDA [US Food and Drug Administration].

We scrambled to understand the mechanism of action. For a year or two, it was a stressful time. But eventually we moved past it, and we learned a lot.”

Q: What sort of work have you done in Latin America?

A: “I’ve always been very close to Latin America. I have many good friends and colleagues there, and I’ve always been interested in working with them. 

We’ve done research and studies and created an organization called Latin American Leukemia Net to develop more trials in Latin America. The most rewarding thing has been the educational programs for patients that we’ve done, helping them understand the disease, the treatments, and the goals of treatment. 

We’ve conducted a number of programs, and they have been effective, well-attended, and well received. I still work with my colleagues to develop local guidelines and do collaborative research.”

Q: What convinced you to leave MD Anderson for Georgia?

A: “I never thought I’d leave MD Anderson. I had my well-oiled machine of clinical trials, my clinic, and my fellowship program. But the one thing that I wanted to see if I could try next was to develop an institution.

That was the goal here, to take the Georgia Cancer Center to NCI [National Cancer Institute] designation. So, I thought, ‘That’s a nice challenge.’ It may be a good opportunity to try a different aspect of what it means to be an oncologist.

There are days that you think, ‘What am I doing here?’ when you have to deal with budgets and personnel and all these things. But it’s part of the process. It’s still good to know that we have a goal, and that we’re going to make it. 

Also, I still see my patients, and I enjoy that I still do some research and mentoring.”

Q: What’s the current state of CML treatment?

A: “Many patients have a pretty much normal life expectancy while [on therapy]. Still, one of the goals of many patients is to stop therapy. But that’s a reality only for a small percentage of patients. How can we make that happen for more patients?”

Q: By stopping therapy, do you mean curing the cancer?

A: “Yes, pretty much. You have a good response, you stop the therapy, and it doesn’t come back.

There are also patients who really don’t do well. We hear about CML being with a disease with such a good outcome, but we have patients for whom nothing works. Is it a matter of [needing] another TKI, or do we need to look at something else?”

Q: What do you see on the horizon?

A: “We are developing new approaches like combination therapies. We’re scratching the surface on that. We need to understand which combinations work, and where and when.

And we can make more efficient uses of the drugs we have now in terms of which ones to use when, the doses, the safety profiles. I think we can do better.”

Cortes disclosed consulting for Amphivena, Astellas, Bio-Path, BioLineRx, Bristol Myers Squibb, Daiichi Sankyo, Jazz, Novartis, Pfizer, and Takeda and research funding from Astellas Pharma, Bristol Myers Squibb, Daiichi Sankyo, Immunogen, Jazz, Merus, Novartis, Pfizer, Sun Pharma, Takeda, Tolero and Trovagene.
 

A version of this article appeared on Medscape.com.

If the name of leukemia specialist Jorge Cortes, MD, appears any more often in PubMed, they’ll need to name a wing after him. 

Over 30 years, Cortes has led or coauthored hundreds of studies, including many trials of landmark drugs to treat chronic myeloid leukemia (CML). His work has helped transform CML into an often-survivable disease instead of one that took the lives of most patients within 5 years.

“It’s been remarkable to see the evolution in CML and to be part of that transition as a fellow, as faculty, and as leader of some of the trials,” said Cortes, who directs the Georgia Cancer Center at Augusta University. “I’m the luckiest person in the world.”

In an interview, Cortes talked about his youth in Mexico, his research path, and his close connections to cancer medicine in Latin America.

Q: You grew up in Mexico City. What was your family like?

A: “My father grew up very poor in a small town in Michoacán in the southwest part of Mexico. In Mexico City, he had a tiny grocery store in an old-fashioned market, and we were lower middle class.

One of the things I learned was to work hard. There’s nobody I know who worked as hard as my father. He opened his store every day of the year, [Mexican] Independence Day or New Year’s or Christmas. He worked hard so we could have a better life than he did.

We learned English from a very young age. My elementary school was called Westminster School because he wanted a school where we would learn English.

As for my mother, she stayed with us [at home] and made sure we did our homework and were taken care of. I learned about being honest and dedicating to what you were doing.”

Q: You trained at the Salvador Zubirán National Institute of Health Sciences and Nutrition in Mexico City. Then what happened? 

A: “Through encouragement by my dermatologist older brother and a mentor at the institution where I was training as a hematologist, I decided to come to the United States.

My initial focus was going to be on coagulation and thrombosis. I came to Houston (Texas) for a fellowship at the University of Texas Health Science Center.

Then I started doing my rotation for the malignant part of the fellowship at MD Anderson Cancer Center [Houston]. One of my first rotations was with Susan M. O’Brien, [MD,] who became my greatest mentor throughout my career. I really enjoyed my rotation. I thought she was great clinically, and she was doing research and teaching. That’s what I wanted for my career.”

Q: What drew you to leukemia specifically?

A: “Dr O’Brien worked in leukemia during my initial rotation, and I really loved it. It was hard work, but it was very inspiring to see the clinical research and the things you could for patients. She had a lot of joy doing that. 

I told my program director I’d change and transfer to MD Anderson, and I ended up staying at MD Anderson for 23 years.”

Q: What was leukemia research like in those days?

A: “We didn’t have the understanding of the biology and the new drugs that we have now. When I started in Mexico, we didn’t even have hydroxyurea. What we were doing was much more basic. But still, the field sounded like a great field to be involved with because they were doing so many trials and had an outstanding database. 

Because of the influence of Dr [Moshe] Talpaz, [MD,] I started getting very involved with CML. In my initial years as a young faculty, I started working with him on interferon. Then imatinib appeared. I saw even from the phase 1 study how impressive the outcomes were in patients who had no response to anything and were in bad shape.”

Q: What CML medications have you worked on?

A: “I’ve been involved with all of them. Imatinib early on, then I led trials with dasatinib and nilotinib. Then, I led the registration trials of bosutinib and ponatinib. More recently, I was part of the development of asciminib.”

Q: What were some of the biggest challenges in CML research?

A: “We had an opportunity to do a lot of analysis about TKIs [tyrosine kinase inhibitors] when these were new drugs. It was a very steep curve of learning, how to monitor and manage side effects.

Then patients were starting to have resistance to two to three TKIs. Ponatinib came along, and it was an incredibly effective drug. But after it was approved, we started to recognize the occurrence of heart attacks and strokes.

That was unexpected and not something that was known for any TKI. It was a big challenge. The drug was taken off the market for some time, and trials were put on hold by the FDA [US Food and Drug Administration].

We scrambled to understand the mechanism of action. For a year or two, it was a stressful time. But eventually we moved past it, and we learned a lot.”

Q: What sort of work have you done in Latin America?

A: “I’ve always been very close to Latin America. I have many good friends and colleagues there, and I’ve always been interested in working with them. 

We’ve done research and studies and created an organization called Latin American Leukemia Net to develop more trials in Latin America. The most rewarding thing has been the educational programs for patients that we’ve done, helping them understand the disease, the treatments, and the goals of treatment. 

We’ve conducted a number of programs, and they have been effective, well-attended, and well received. I still work with my colleagues to develop local guidelines and do collaborative research.”

Q: What convinced you to leave MD Anderson for Georgia?

A: “I never thought I’d leave MD Anderson. I had my well-oiled machine of clinical trials, my clinic, and my fellowship program. But the one thing that I wanted to see if I could try next was to develop an institution.

That was the goal here, to take the Georgia Cancer Center to NCI [National Cancer Institute] designation. So, I thought, ‘That’s a nice challenge.’ It may be a good opportunity to try a different aspect of what it means to be an oncologist.

There are days that you think, ‘What am I doing here?’ when you have to deal with budgets and personnel and all these things. But it’s part of the process. It’s still good to know that we have a goal, and that we’re going to make it. 

Also, I still see my patients, and I enjoy that I still do some research and mentoring.”

Q: What’s the current state of CML treatment?

A: “Many patients have a pretty much normal life expectancy while [on therapy]. Still, one of the goals of many patients is to stop therapy. But that’s a reality only for a small percentage of patients. How can we make that happen for more patients?”

Q: By stopping therapy, do you mean curing the cancer?

A: “Yes, pretty much. You have a good response, you stop the therapy, and it doesn’t come back.

There are also patients who really don’t do well. We hear about CML being with a disease with such a good outcome, but we have patients for whom nothing works. Is it a matter of [needing] another TKI, or do we need to look at something else?”

Q: What do you see on the horizon?

A: “We are developing new approaches like combination therapies. We’re scratching the surface on that. We need to understand which combinations work, and where and when.

And we can make more efficient uses of the drugs we have now in terms of which ones to use when, the doses, the safety profiles. I think we can do better.”

Cortes disclosed consulting for Amphivena, Astellas, Bio-Path, BioLineRx, Bristol Myers Squibb, Daiichi Sankyo, Jazz, Novartis, Pfizer, and Takeda and research funding from Astellas Pharma, Bristol Myers Squibb, Daiichi Sankyo, Immunogen, Jazz, Merus, Novartis, Pfizer, Sun Pharma, Takeda, Tolero and Trovagene.
 

A version of this article appeared on Medscape.com.