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Trump Nominations for US Health Agencies Spark Controversy, Criticism, Praise
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.
Eliminate Clozapine REMS, FDA Panels Say
Two Food and Drug Administration (FDA) advisory panels are urging the agency to eliminate the risk management program for the antipsychotic drug clozapine, saying that restrictions are limiting access to a life-changing and life-saving medication for people with schizophrenia.
Members of the Drug Safety and Risk Management and Psychopharmacologic Drugs advisory committees held a joint meeting on November 19 to address whether frequently revised restrictions that have been in place since clozapine was introduced in 1989 should be changed again. Clozapine — the only FDA-approved drug for treatment-resistant schizophrenia — can cause severe neutropenia, so is subject to a Risk Evaluation and Management Strategy (REMS).
Calling the current rules overly burdensome, a majority of committee members voted against continuing a requirement that pharmacies and physicians must provide documentation of a patient’s absolute neutrophil count (ANC) results through the REMS. Monitoring should continue, as directed in the labeling, said the panel.
Panelists also voted overwhelmingly that it is not necessary to mandate physician education about clozapine’s risk of neutropenia and the need for ANC monitoring.
The panel did not vote, however, on whether the REMS should be eliminated altogether. The FDA did not pose that as a voting question for the panels’ consideration.
Following intense lobbying by the American Psychiatric Association (APA), the National Alliance on Mental Illness, and others, the FDA announced in 2022 that the agency would exercise “enforcement discretion” by allowing prescribers and pharmacists to skirt the clozapine REMS rules. But the agency doesn’t know whether the program is meeting its goals, said Tiffany R. Farchione, MD, director of the division of psychiatry at the FDA’s Center for Drug Evaluation and Research.
Among other things, the REMS requires that physicians and pharmacists be certified to prescribe and dispense the drug, that patients be enrolled, and that patient status forms be submitted monthly, showing ANC levels and appropriateness of continuing treatment.
At the meeting, FDA officials said that 148,000 outpatient clozapine prescriptions were written in 2023. But an estimated 814,000–1.2 million Americans have treatment-resistant schizophrenia, the main indication for clozapine.
“We know the drug is being underutilized,” said Farchione, adding that the agency wants to ensure that physicians and pharmacists “can use the drug, use it safely and help the patients who need it.”
REMS a ‘Hindrance’
As reported by this news organization, research presented earlier this year at the APA annual meeting showed that the risk of moderate and severe neutropenia is low to minimal in people taking clozapine for treatment-resistant schizophrenia. Those findings prompted the study’s investigators to suggest clozapine REMS should be reconsidered.
In the November 19th committee meeting, many panelists said that clozapine was no more dangerous than many antipsychotics and that the administrative requirements were preventing clinicians from prescribing.
“I have fantasized for years about abolishing the clozapine REMS,” said Jacob S. Ballon, MD, MPH, a temporary panel member and associate professor of psychiatry at Stanford University in California.
Panelists Jess Fiedorowicz, MD, PhD, professor and senior research chair in adult psychiatry at the University of Ottawa, Canada; Megan J. Ehret, PharmD, MS, a panelist and professor at the University of Maryland School of Pharmacy, Baltimore; and Rajesh Narendran, MD, a professor in radiology and psychiatry at the University of Pittsburgh School of Medicine in Pennsylvania, agreed.
“I strongly feel that the REMS at this point is just a hindrance,” Narendran said. “I think you should get rid of the REMS.”
However, panelist Walter Dunn, MD, PhD, staff psychiatrist at the VA Greater Los Angeles Healthcare System, cautioned that modifying or eliminating the REMS might not necessarily increase prescribers. If monitoring ANC levels is still recommended in labeling, clinicians will still regard it as the standard of care, said Dunn. And “there are a whole host of other issues associated with clozapine,” that he said were “more concerning.”
Many patients are accessing clozapine without going through the REMS, which is also of concern to the FDA and drug manufacturers.
“We estimate about 42,000 patients are not participating in the REMS, said James Shamp, VP of data intelligence and program analytics at United BioSource, a company that supports drug makers.
Leah Hart, PharmD, a risk management analyst with the FDA, told the panel that the agency estimates that 25%-35% of patients taking clozapine may not be participating in the REMS.
“Today, prescribers, pharmacies, and patients do not have to participate in the REMS in order patients to obtain clozapine,” Hart said.
Public Testimony Sways Panel
But psychiatrists, pharmacists, families, and patients who testified during the 90-minute open portion of the meeting disagreed with that assessment, saying the REMS program had a devastatingly chilling effect on clozapine access.
Patty Taggart of Las Vegas said her daughter had nine suicide attempts over the past 14 years, while having tried eight different antipsychotics. In August, after the most-recent attempt, Taggart begged the psychiatrist to prescribe clozapine to her daughter. The clinician refused, citing the REMS. After her daughter’s discharge, Taggart said she found another provider who would prescribe the medication.
Lisa Castellanos said her son Daniel had been treated with a variety of antipsychotics but denied clozapine until he was arrested in 2012 for assault during a psychotic break. The state used the medication to improve Daniel’s mental state so he could stand trial. But when he went to jail after accepting a plea deal, the prison stopped the clozapine. Daniel has since deteriorated and was recently ruled ineligible for parole.
Patients and families also described being rejected at pharmacies — most of which, despite the FDA’s supposed “enforcement discretion” continue to rigorously follow REMS requirements.
Many panelists said they were moved by patients and family testimony. A dozen or more members of the public were wearing black t-shirts with white writing that declared: “Clozapine is the safest antipsychotic in the world.”
‘Blood-for-Drug Program’
Brian Barnett, MD, director of the psychiatric treatment-resistance program at the Cleveland Clinic in Ohio, said during the public portion of the meeting that “many pharmacies simply refuse to dispense clozapine likely because of the administrative burden and lack of financial incentives.”
Others want faxed lab results even when the results have been filed electronically, he said. “One of the most dangerous features of the current REMS system is its inflexibility, driven by the so-called ‘no blood, no drug’ ethos which has been baked into the minds of America’s pharmacists.”
“This is a blood-for-drug program,” agreed Rachel Strieff of Tempe, Arizona, who noted that her advocacy group, Angry Moms, and others had submitted 4,000 signatures calling for the end of the REMS. “The largest category of patients harmed by the clozapine REMS have never taken a single dose,” she said, noting that millions of eligible individuals are not getting the drug.
Panel chair James Floyd, MD, professor of medicine at the University of Washington, Seattle, said the public testimony was “very moving.” Families and patients had described “the intensity of suffering that people go through prior to getting to clozapine,” he added.
“We have to listen to that,” said Floyd.
“I want you to know that we hear you,” said Farchione. “We’re here today because of you and your loved ones. And your stories are important, and your experience is important, and what you’ve shared today will have an impact on regulatory decision making.”
While the FDA typically follows its panels’ advice, it’s unclear if the agency will do so for clozapine REMS or when it will release its final decision.
A version of this article appeared on Medscape.com.
Two Food and Drug Administration (FDA) advisory panels are urging the agency to eliminate the risk management program for the antipsychotic drug clozapine, saying that restrictions are limiting access to a life-changing and life-saving medication for people with schizophrenia.
Members of the Drug Safety and Risk Management and Psychopharmacologic Drugs advisory committees held a joint meeting on November 19 to address whether frequently revised restrictions that have been in place since clozapine was introduced in 1989 should be changed again. Clozapine — the only FDA-approved drug for treatment-resistant schizophrenia — can cause severe neutropenia, so is subject to a Risk Evaluation and Management Strategy (REMS).
Calling the current rules overly burdensome, a majority of committee members voted against continuing a requirement that pharmacies and physicians must provide documentation of a patient’s absolute neutrophil count (ANC) results through the REMS. Monitoring should continue, as directed in the labeling, said the panel.
Panelists also voted overwhelmingly that it is not necessary to mandate physician education about clozapine’s risk of neutropenia and the need for ANC monitoring.
The panel did not vote, however, on whether the REMS should be eliminated altogether. The FDA did not pose that as a voting question for the panels’ consideration.
Following intense lobbying by the American Psychiatric Association (APA), the National Alliance on Mental Illness, and others, the FDA announced in 2022 that the agency would exercise “enforcement discretion” by allowing prescribers and pharmacists to skirt the clozapine REMS rules. But the agency doesn’t know whether the program is meeting its goals, said Tiffany R. Farchione, MD, director of the division of psychiatry at the FDA’s Center for Drug Evaluation and Research.
Among other things, the REMS requires that physicians and pharmacists be certified to prescribe and dispense the drug, that patients be enrolled, and that patient status forms be submitted monthly, showing ANC levels and appropriateness of continuing treatment.
At the meeting, FDA officials said that 148,000 outpatient clozapine prescriptions were written in 2023. But an estimated 814,000–1.2 million Americans have treatment-resistant schizophrenia, the main indication for clozapine.
“We know the drug is being underutilized,” said Farchione, adding that the agency wants to ensure that physicians and pharmacists “can use the drug, use it safely and help the patients who need it.”
REMS a ‘Hindrance’
As reported by this news organization, research presented earlier this year at the APA annual meeting showed that the risk of moderate and severe neutropenia is low to minimal in people taking clozapine for treatment-resistant schizophrenia. Those findings prompted the study’s investigators to suggest clozapine REMS should be reconsidered.
In the November 19th committee meeting, many panelists said that clozapine was no more dangerous than many antipsychotics and that the administrative requirements were preventing clinicians from prescribing.
“I have fantasized for years about abolishing the clozapine REMS,” said Jacob S. Ballon, MD, MPH, a temporary panel member and associate professor of psychiatry at Stanford University in California.
Panelists Jess Fiedorowicz, MD, PhD, professor and senior research chair in adult psychiatry at the University of Ottawa, Canada; Megan J. Ehret, PharmD, MS, a panelist and professor at the University of Maryland School of Pharmacy, Baltimore; and Rajesh Narendran, MD, a professor in radiology and psychiatry at the University of Pittsburgh School of Medicine in Pennsylvania, agreed.
“I strongly feel that the REMS at this point is just a hindrance,” Narendran said. “I think you should get rid of the REMS.”
However, panelist Walter Dunn, MD, PhD, staff psychiatrist at the VA Greater Los Angeles Healthcare System, cautioned that modifying or eliminating the REMS might not necessarily increase prescribers. If monitoring ANC levels is still recommended in labeling, clinicians will still regard it as the standard of care, said Dunn. And “there are a whole host of other issues associated with clozapine,” that he said were “more concerning.”
Many patients are accessing clozapine without going through the REMS, which is also of concern to the FDA and drug manufacturers.
“We estimate about 42,000 patients are not participating in the REMS, said James Shamp, VP of data intelligence and program analytics at United BioSource, a company that supports drug makers.
Leah Hart, PharmD, a risk management analyst with the FDA, told the panel that the agency estimates that 25%-35% of patients taking clozapine may not be participating in the REMS.
“Today, prescribers, pharmacies, and patients do not have to participate in the REMS in order patients to obtain clozapine,” Hart said.
Public Testimony Sways Panel
But psychiatrists, pharmacists, families, and patients who testified during the 90-minute open portion of the meeting disagreed with that assessment, saying the REMS program had a devastatingly chilling effect on clozapine access.
Patty Taggart of Las Vegas said her daughter had nine suicide attempts over the past 14 years, while having tried eight different antipsychotics. In August, after the most-recent attempt, Taggart begged the psychiatrist to prescribe clozapine to her daughter. The clinician refused, citing the REMS. After her daughter’s discharge, Taggart said she found another provider who would prescribe the medication.
Lisa Castellanos said her son Daniel had been treated with a variety of antipsychotics but denied clozapine until he was arrested in 2012 for assault during a psychotic break. The state used the medication to improve Daniel’s mental state so he could stand trial. But when he went to jail after accepting a plea deal, the prison stopped the clozapine. Daniel has since deteriorated and was recently ruled ineligible for parole.
Patients and families also described being rejected at pharmacies — most of which, despite the FDA’s supposed “enforcement discretion” continue to rigorously follow REMS requirements.
Many panelists said they were moved by patients and family testimony. A dozen or more members of the public were wearing black t-shirts with white writing that declared: “Clozapine is the safest antipsychotic in the world.”
‘Blood-for-Drug Program’
Brian Barnett, MD, director of the psychiatric treatment-resistance program at the Cleveland Clinic in Ohio, said during the public portion of the meeting that “many pharmacies simply refuse to dispense clozapine likely because of the administrative burden and lack of financial incentives.”
Others want faxed lab results even when the results have been filed electronically, he said. “One of the most dangerous features of the current REMS system is its inflexibility, driven by the so-called ‘no blood, no drug’ ethos which has been baked into the minds of America’s pharmacists.”
“This is a blood-for-drug program,” agreed Rachel Strieff of Tempe, Arizona, who noted that her advocacy group, Angry Moms, and others had submitted 4,000 signatures calling for the end of the REMS. “The largest category of patients harmed by the clozapine REMS have never taken a single dose,” she said, noting that millions of eligible individuals are not getting the drug.
Panel chair James Floyd, MD, professor of medicine at the University of Washington, Seattle, said the public testimony was “very moving.” Families and patients had described “the intensity of suffering that people go through prior to getting to clozapine,” he added.
“We have to listen to that,” said Floyd.
“I want you to know that we hear you,” said Farchione. “We’re here today because of you and your loved ones. And your stories are important, and your experience is important, and what you’ve shared today will have an impact on regulatory decision making.”
While the FDA typically follows its panels’ advice, it’s unclear if the agency will do so for clozapine REMS or when it will release its final decision.
A version of this article appeared on Medscape.com.
Two Food and Drug Administration (FDA) advisory panels are urging the agency to eliminate the risk management program for the antipsychotic drug clozapine, saying that restrictions are limiting access to a life-changing and life-saving medication for people with schizophrenia.
Members of the Drug Safety and Risk Management and Psychopharmacologic Drugs advisory committees held a joint meeting on November 19 to address whether frequently revised restrictions that have been in place since clozapine was introduced in 1989 should be changed again. Clozapine — the only FDA-approved drug for treatment-resistant schizophrenia — can cause severe neutropenia, so is subject to a Risk Evaluation and Management Strategy (REMS).
Calling the current rules overly burdensome, a majority of committee members voted against continuing a requirement that pharmacies and physicians must provide documentation of a patient’s absolute neutrophil count (ANC) results through the REMS. Monitoring should continue, as directed in the labeling, said the panel.
Panelists also voted overwhelmingly that it is not necessary to mandate physician education about clozapine’s risk of neutropenia and the need for ANC monitoring.
The panel did not vote, however, on whether the REMS should be eliminated altogether. The FDA did not pose that as a voting question for the panels’ consideration.
Following intense lobbying by the American Psychiatric Association (APA), the National Alliance on Mental Illness, and others, the FDA announced in 2022 that the agency would exercise “enforcement discretion” by allowing prescribers and pharmacists to skirt the clozapine REMS rules. But the agency doesn’t know whether the program is meeting its goals, said Tiffany R. Farchione, MD, director of the division of psychiatry at the FDA’s Center for Drug Evaluation and Research.
Among other things, the REMS requires that physicians and pharmacists be certified to prescribe and dispense the drug, that patients be enrolled, and that patient status forms be submitted monthly, showing ANC levels and appropriateness of continuing treatment.
At the meeting, FDA officials said that 148,000 outpatient clozapine prescriptions were written in 2023. But an estimated 814,000–1.2 million Americans have treatment-resistant schizophrenia, the main indication for clozapine.
“We know the drug is being underutilized,” said Farchione, adding that the agency wants to ensure that physicians and pharmacists “can use the drug, use it safely and help the patients who need it.”
REMS a ‘Hindrance’
As reported by this news organization, research presented earlier this year at the APA annual meeting showed that the risk of moderate and severe neutropenia is low to minimal in people taking clozapine for treatment-resistant schizophrenia. Those findings prompted the study’s investigators to suggest clozapine REMS should be reconsidered.
In the November 19th committee meeting, many panelists said that clozapine was no more dangerous than many antipsychotics and that the administrative requirements were preventing clinicians from prescribing.
“I have fantasized for years about abolishing the clozapine REMS,” said Jacob S. Ballon, MD, MPH, a temporary panel member and associate professor of psychiatry at Stanford University in California.
Panelists Jess Fiedorowicz, MD, PhD, professor and senior research chair in adult psychiatry at the University of Ottawa, Canada; Megan J. Ehret, PharmD, MS, a panelist and professor at the University of Maryland School of Pharmacy, Baltimore; and Rajesh Narendran, MD, a professor in radiology and psychiatry at the University of Pittsburgh School of Medicine in Pennsylvania, agreed.
“I strongly feel that the REMS at this point is just a hindrance,” Narendran said. “I think you should get rid of the REMS.”
However, panelist Walter Dunn, MD, PhD, staff psychiatrist at the VA Greater Los Angeles Healthcare System, cautioned that modifying or eliminating the REMS might not necessarily increase prescribers. If monitoring ANC levels is still recommended in labeling, clinicians will still regard it as the standard of care, said Dunn. And “there are a whole host of other issues associated with clozapine,” that he said were “more concerning.”
Many patients are accessing clozapine without going through the REMS, which is also of concern to the FDA and drug manufacturers.
“We estimate about 42,000 patients are not participating in the REMS, said James Shamp, VP of data intelligence and program analytics at United BioSource, a company that supports drug makers.
Leah Hart, PharmD, a risk management analyst with the FDA, told the panel that the agency estimates that 25%-35% of patients taking clozapine may not be participating in the REMS.
“Today, prescribers, pharmacies, and patients do not have to participate in the REMS in order patients to obtain clozapine,” Hart said.
Public Testimony Sways Panel
But psychiatrists, pharmacists, families, and patients who testified during the 90-minute open portion of the meeting disagreed with that assessment, saying the REMS program had a devastatingly chilling effect on clozapine access.
Patty Taggart of Las Vegas said her daughter had nine suicide attempts over the past 14 years, while having tried eight different antipsychotics. In August, after the most-recent attempt, Taggart begged the psychiatrist to prescribe clozapine to her daughter. The clinician refused, citing the REMS. After her daughter’s discharge, Taggart said she found another provider who would prescribe the medication.
Lisa Castellanos said her son Daniel had been treated with a variety of antipsychotics but denied clozapine until he was arrested in 2012 for assault during a psychotic break. The state used the medication to improve Daniel’s mental state so he could stand trial. But when he went to jail after accepting a plea deal, the prison stopped the clozapine. Daniel has since deteriorated and was recently ruled ineligible for parole.
Patients and families also described being rejected at pharmacies — most of which, despite the FDA’s supposed “enforcement discretion” continue to rigorously follow REMS requirements.
Many panelists said they were moved by patients and family testimony. A dozen or more members of the public were wearing black t-shirts with white writing that declared: “Clozapine is the safest antipsychotic in the world.”
‘Blood-for-Drug Program’
Brian Barnett, MD, director of the psychiatric treatment-resistance program at the Cleveland Clinic in Ohio, said during the public portion of the meeting that “many pharmacies simply refuse to dispense clozapine likely because of the administrative burden and lack of financial incentives.”
Others want faxed lab results even when the results have been filed electronically, he said. “One of the most dangerous features of the current REMS system is its inflexibility, driven by the so-called ‘no blood, no drug’ ethos which has been baked into the minds of America’s pharmacists.”
“This is a blood-for-drug program,” agreed Rachel Strieff of Tempe, Arizona, who noted that her advocacy group, Angry Moms, and others had submitted 4,000 signatures calling for the end of the REMS. “The largest category of patients harmed by the clozapine REMS have never taken a single dose,” she said, noting that millions of eligible individuals are not getting the drug.
Panel chair James Floyd, MD, professor of medicine at the University of Washington, Seattle, said the public testimony was “very moving.” Families and patients had described “the intensity of suffering that people go through prior to getting to clozapine,” he added.
“We have to listen to that,” said Floyd.
“I want you to know that we hear you,” said Farchione. “We’re here today because of you and your loved ones. And your stories are important, and your experience is important, and what you’ve shared today will have an impact on regulatory decision making.”
While the FDA typically follows its panels’ advice, it’s unclear if the agency will do so for clozapine REMS or when it will release its final decision.
A version of this article appeared on Medscape.com.
MDMA Is Off the Table, So What’s Next for PTSD?
It has been 24 years since a pharmaceutical was last approved for posttraumatic stress disorder (PTSD). The condition is notoriously difficult to treat, with up to 40% patients finding no relief from symptoms through psychotherapy or current medications.
Many clinicians, advocates, and patients had pinned their hopes on the psychedelic drug midomafetamine with assisted therapy (MDMA-AT). However, in August, the US Food and Drug Administration (FDA) rejected it. At this point, it’s unclear when the therapy will be available, if ever.
“Not getting the FDA approval of any drug at this point is a setback for the field,” Lori Davis, MD, a senior research psychiatrist at the Birmingham Veterans Affairs (VA) Health Care System in Birmingham, Alabama, told Medscape Medical News.
Having an FDA-approved product would have helped increase public awareness of PTSD and driven interest in developing new therapies, said Davis, who is also adjunct professor of psychiatry at the Heersink School of Medicine, University of Alabama at Birmingham.
A Treatable Condition
So with MDMA-AT off the table, where does the field go next?
A public meeting in September hosted by the Reagan-Udall Foundation for the FDA in sought to answer that question. Agency officials joined representatives from the Department of Defense (DoD) and VA, patients, advocates, and industry representatives to discuss the current treatment landscape and what can be done to accelerate development of PTSD treatment.
Despite the common belief that PTSD is intractable, it “is a treatable condition,” Paula P. Schnurr, PhD, executive director of the VA National Center for PTSD, said at the meeting.
“There are effective treatments that work well for a lot of people, although not everyone has a satisfactory response,” she added.
The most effective psychotherapies are “trauma-focused,” and include cognitive processing therapy, eye movement desensitization and reprocessing, and prolonged exposure, according to the VA National Center for PTSD.
Three drugs have been approved by the FDA for PTSD: Venlafaxine (Effexor) in 1993, sertraline (Zoloft) in 1999, and paroxetine (Paxil) in 2000.
However, as the September meeting demonstrated, more therapies are needed.
“It’s clear to FDA and the federal government at large that there is an unmet need for safe and effective therapies to treat PTSD,” Bernard Fischer, MD, deputy director of the Division of Psychiatry in the Office of New Drugs at FDA’s Center for Drug Evaluation and Research, said at the meeting.
There is no shortage of research, Fischer added. Nearly 500 trials focused on PTSD are listed on clinicaltrials.gov are recruiting participants now or plan to soon.
Unsurprisingly, one of the primary drivers of PTSD therapeutics research is the VA. About 14% of the 5.7 million veterans who received care through the VA in 2023 had a diagnosis of PTSD.
“The US military is currently losing thousands of service members each year to PTSD- related disability discharges,” US Army Maj. Aaron Wolfgang, MD, a psychiatrist at the Walter Reed National Military Medical Center, said at the meeting. Only about 12%-20% of patients achieve remission with conventional therapies, added Wolfgang, who also is an assistant professor at the Uniformed Services University.
“For these reasons, establishing better treatments for PTSD is not only a matter of humanitarianism but also a pressing matter of national security,” he said.
The VA has committed at least $230 million to more than 140 active research projects in PTSD, Miriam J. Smyth, PhD, acting director of the clinical science, research and development service at the VA, said at the Reagan-Udall meeting.
One of the VA projects is the PTSD psychopharmacology initiative, which began in 2017 and now has 14 active clinical trials, said Smyth, who is also acting director for brain behavior and mental health at the VA. The first study should be finished by 2025.
The Million Veteran Program, with more than 1 million enrollees, has led to the discovery of genes related to re-experiencing traumatic memories and has confirmed that both PTSD and traumatic brain injury are risk factors for dementia, Smyth said.
The DoD has created a novel platform that establishes a common infrastructure for testing multiple drugs, called M-PACT. The platform allows sharing of placebo data across treatment arms. Drugs cycle off the platform if evidence indicates probability of success or failure.
Four trials are actively recruiting veterans and current service members. One is looking at vilazodone, approved in 2011 for major depressive disorder. It is being compared with placebo and fluoxetine in a trial that is currently recruiting.
Another trial will study daridorexant (sold as Quviviq), an orexin receptor antagonist, against placebo. The FDA approved daridorexant in 2022 as an insomnia treatment. A core issue in PTSD is sleep disruption, noted Davis.
New Therapies on the Way
Separately, Davis and colleagues are also studying methylphenidate, the stimulant used for attention-deficit/hyperactivity disorder. It may help with neurocognitive complaints and reduce PTSD symptoms, said Davis.
Because it is generic, few pharmaceutical manufacturers are likely to test it for PTSD, she said. But eventually, their work may lead a company to test newer stimulants for PTSD, she said.
Another potential therapeutic, BNC210, received Fast Track designation for PTSD from the FDA in 2019. Bionomics Limited in Australia will soon launch phase 3 trials of the investigational oral drug, which is a negative allosteric modulator of the alpha-7 nicotinic acetylcholine receptor. In late July, the company announced “ favorable feedback” from the agency on its phase 2 study, which led to the decision to move forward with larger trials.
Researchers at Brigham and Women’s Hospital have just reported that they may have found a target within the brain that will allow for transcranial magnetic stimulation (TMS) to ameliorate PTSD symptoms. They published results of a mapping effort in Nature Neuroscience and reported on one patient who had improved symptoms after receiving TMS for severe PTSD.
But perhaps one of the most promising treatments is a combination of sertraline and the new psychiatric medication brexpiprazole.
Brexpiprazole was developed by Otsuka Pharmaceutical and approved in the United States in 2015 as an adjunctive therapy to antidepressants for major depressive disorder and as a treatment for schizophrenia. In 2023, the FDA approved it for Alzheimer’s-related agitation. However, according to Otsuka, its mechanism of action is unknown.
Its efficacy may be mediated through a combination of partial agonist activity at serotonin 5-HT1A and dopamine D2 receptors, antagonist activity at serotonin 5-HT2A receptors, as well as antagonism of alpha-1B/2C receptors, said the company.
“It is the combination, rather than either alone, that’s going to have that broad synergistic pharmacology that is obviously potent for ameliorating the symptoms of PTSD,” said Davis, who has received consulting fees from Otsuka. “That’s an exciting development.”
Otsuka and partner Lundbeck Pharmaceuticals reported results in May from the companies’ phase 2 and 3 randomized clinical trials. The therapy achieved a statistically significant reduction (P <.05) in PTSD symptoms compared with sertraline plus placebo. This was without any supplemental psychotherapy.
The FDA accepted the companies’ new drug application in June and is expected to make a decision on approval in February 2025.
The Potential of Psychedelics
Though Lykos Therapeutics may have to go back to the drawing board on its MDMA-AT, psychedelics still have potential as PTSD therapies, Smyth said, who added that the VA is continuing to encourage study of MDMA and other psychedelic agents.
The VA issued a call for proposals for research on psychedelics in January, focused on MDMA or psilocybin in combination with psychotherapy. The administration received the first wave of applications early in the summer.
Scientific peer review panels made up of research experts from within and outside the VA have reviewed the applications and funding announcements are expected this fall, Smyth said.
Wolfgang, the Army psychiatrist, said, “Under the psychedelic treatment research clinical trial award, we welcome investigators to apply to what we anticipate will usher in a new era of innovation and hope for service members and their families who need it the most.”
Psychedelic studies are also proceeding without VA funding, as they have for years, when most of the trials were backed by universities or foundations or other private money. Johns Hopkins University is recruiting for a study in which patients would receive psilocybin along with trauma-focused psychotherapy, as is Ohio State University.
London-based Compass Pathways said in May that it successfully completed a phase 2 trial of Comp360, its synthetic psilocybin, in PTSD. The company has started a phase 3 study in treatment-resistant depression but has not given any further updates on PTSD.
Davis said that she believes that the FDA’s rejection of Lykos won’t lead to a shutdown of exploration of psychedelics.
“I think it informs these designs going forward, but it doesn’t eliminate that whole field of research,” she said.
Davis reported receiving consulting fees from Boehringer Ingelheim and Otsuka and research funding from Alkermes, the Patient-Centered Outcomes Research Institute, and the VA. Schnurr, Fischer, Smyth, and Wolfgang reported no relevant disclosures.
A version of this article appeared on Medscape.com.
It has been 24 years since a pharmaceutical was last approved for posttraumatic stress disorder (PTSD). The condition is notoriously difficult to treat, with up to 40% patients finding no relief from symptoms through psychotherapy or current medications.
Many clinicians, advocates, and patients had pinned their hopes on the psychedelic drug midomafetamine with assisted therapy (MDMA-AT). However, in August, the US Food and Drug Administration (FDA) rejected it. At this point, it’s unclear when the therapy will be available, if ever.
“Not getting the FDA approval of any drug at this point is a setback for the field,” Lori Davis, MD, a senior research psychiatrist at the Birmingham Veterans Affairs (VA) Health Care System in Birmingham, Alabama, told Medscape Medical News.
Having an FDA-approved product would have helped increase public awareness of PTSD and driven interest in developing new therapies, said Davis, who is also adjunct professor of psychiatry at the Heersink School of Medicine, University of Alabama at Birmingham.
A Treatable Condition
So with MDMA-AT off the table, where does the field go next?
A public meeting in September hosted by the Reagan-Udall Foundation for the FDA in sought to answer that question. Agency officials joined representatives from the Department of Defense (DoD) and VA, patients, advocates, and industry representatives to discuss the current treatment landscape and what can be done to accelerate development of PTSD treatment.
Despite the common belief that PTSD is intractable, it “is a treatable condition,” Paula P. Schnurr, PhD, executive director of the VA National Center for PTSD, said at the meeting.
“There are effective treatments that work well for a lot of people, although not everyone has a satisfactory response,” she added.
The most effective psychotherapies are “trauma-focused,” and include cognitive processing therapy, eye movement desensitization and reprocessing, and prolonged exposure, according to the VA National Center for PTSD.
Three drugs have been approved by the FDA for PTSD: Venlafaxine (Effexor) in 1993, sertraline (Zoloft) in 1999, and paroxetine (Paxil) in 2000.
However, as the September meeting demonstrated, more therapies are needed.
“It’s clear to FDA and the federal government at large that there is an unmet need for safe and effective therapies to treat PTSD,” Bernard Fischer, MD, deputy director of the Division of Psychiatry in the Office of New Drugs at FDA’s Center for Drug Evaluation and Research, said at the meeting.
There is no shortage of research, Fischer added. Nearly 500 trials focused on PTSD are listed on clinicaltrials.gov are recruiting participants now or plan to soon.
Unsurprisingly, one of the primary drivers of PTSD therapeutics research is the VA. About 14% of the 5.7 million veterans who received care through the VA in 2023 had a diagnosis of PTSD.
“The US military is currently losing thousands of service members each year to PTSD- related disability discharges,” US Army Maj. Aaron Wolfgang, MD, a psychiatrist at the Walter Reed National Military Medical Center, said at the meeting. Only about 12%-20% of patients achieve remission with conventional therapies, added Wolfgang, who also is an assistant professor at the Uniformed Services University.
“For these reasons, establishing better treatments for PTSD is not only a matter of humanitarianism but also a pressing matter of national security,” he said.
The VA has committed at least $230 million to more than 140 active research projects in PTSD, Miriam J. Smyth, PhD, acting director of the clinical science, research and development service at the VA, said at the Reagan-Udall meeting.
One of the VA projects is the PTSD psychopharmacology initiative, which began in 2017 and now has 14 active clinical trials, said Smyth, who is also acting director for brain behavior and mental health at the VA. The first study should be finished by 2025.
The Million Veteran Program, with more than 1 million enrollees, has led to the discovery of genes related to re-experiencing traumatic memories and has confirmed that both PTSD and traumatic brain injury are risk factors for dementia, Smyth said.
The DoD has created a novel platform that establishes a common infrastructure for testing multiple drugs, called M-PACT. The platform allows sharing of placebo data across treatment arms. Drugs cycle off the platform if evidence indicates probability of success or failure.
Four trials are actively recruiting veterans and current service members. One is looking at vilazodone, approved in 2011 for major depressive disorder. It is being compared with placebo and fluoxetine in a trial that is currently recruiting.
Another trial will study daridorexant (sold as Quviviq), an orexin receptor antagonist, against placebo. The FDA approved daridorexant in 2022 as an insomnia treatment. A core issue in PTSD is sleep disruption, noted Davis.
New Therapies on the Way
Separately, Davis and colleagues are also studying methylphenidate, the stimulant used for attention-deficit/hyperactivity disorder. It may help with neurocognitive complaints and reduce PTSD symptoms, said Davis.
Because it is generic, few pharmaceutical manufacturers are likely to test it for PTSD, she said. But eventually, their work may lead a company to test newer stimulants for PTSD, she said.
Another potential therapeutic, BNC210, received Fast Track designation for PTSD from the FDA in 2019. Bionomics Limited in Australia will soon launch phase 3 trials of the investigational oral drug, which is a negative allosteric modulator of the alpha-7 nicotinic acetylcholine receptor. In late July, the company announced “ favorable feedback” from the agency on its phase 2 study, which led to the decision to move forward with larger trials.
Researchers at Brigham and Women’s Hospital have just reported that they may have found a target within the brain that will allow for transcranial magnetic stimulation (TMS) to ameliorate PTSD symptoms. They published results of a mapping effort in Nature Neuroscience and reported on one patient who had improved symptoms after receiving TMS for severe PTSD.
But perhaps one of the most promising treatments is a combination of sertraline and the new psychiatric medication brexpiprazole.
Brexpiprazole was developed by Otsuka Pharmaceutical and approved in the United States in 2015 as an adjunctive therapy to antidepressants for major depressive disorder and as a treatment for schizophrenia. In 2023, the FDA approved it for Alzheimer’s-related agitation. However, according to Otsuka, its mechanism of action is unknown.
Its efficacy may be mediated through a combination of partial agonist activity at serotonin 5-HT1A and dopamine D2 receptors, antagonist activity at serotonin 5-HT2A receptors, as well as antagonism of alpha-1B/2C receptors, said the company.
“It is the combination, rather than either alone, that’s going to have that broad synergistic pharmacology that is obviously potent for ameliorating the symptoms of PTSD,” said Davis, who has received consulting fees from Otsuka. “That’s an exciting development.”
Otsuka and partner Lundbeck Pharmaceuticals reported results in May from the companies’ phase 2 and 3 randomized clinical trials. The therapy achieved a statistically significant reduction (P <.05) in PTSD symptoms compared with sertraline plus placebo. This was without any supplemental psychotherapy.
The FDA accepted the companies’ new drug application in June and is expected to make a decision on approval in February 2025.
The Potential of Psychedelics
Though Lykos Therapeutics may have to go back to the drawing board on its MDMA-AT, psychedelics still have potential as PTSD therapies, Smyth said, who added that the VA is continuing to encourage study of MDMA and other psychedelic agents.
The VA issued a call for proposals for research on psychedelics in January, focused on MDMA or psilocybin in combination with psychotherapy. The administration received the first wave of applications early in the summer.
Scientific peer review panels made up of research experts from within and outside the VA have reviewed the applications and funding announcements are expected this fall, Smyth said.
Wolfgang, the Army psychiatrist, said, “Under the psychedelic treatment research clinical trial award, we welcome investigators to apply to what we anticipate will usher in a new era of innovation and hope for service members and their families who need it the most.”
Psychedelic studies are also proceeding without VA funding, as they have for years, when most of the trials were backed by universities or foundations or other private money. Johns Hopkins University is recruiting for a study in which patients would receive psilocybin along with trauma-focused psychotherapy, as is Ohio State University.
London-based Compass Pathways said in May that it successfully completed a phase 2 trial of Comp360, its synthetic psilocybin, in PTSD. The company has started a phase 3 study in treatment-resistant depression but has not given any further updates on PTSD.
Davis said that she believes that the FDA’s rejection of Lykos won’t lead to a shutdown of exploration of psychedelics.
“I think it informs these designs going forward, but it doesn’t eliminate that whole field of research,” she said.
Davis reported receiving consulting fees from Boehringer Ingelheim and Otsuka and research funding from Alkermes, the Patient-Centered Outcomes Research Institute, and the VA. Schnurr, Fischer, Smyth, and Wolfgang reported no relevant disclosures.
A version of this article appeared on Medscape.com.
It has been 24 years since a pharmaceutical was last approved for posttraumatic stress disorder (PTSD). The condition is notoriously difficult to treat, with up to 40% patients finding no relief from symptoms through psychotherapy or current medications.
Many clinicians, advocates, and patients had pinned their hopes on the psychedelic drug midomafetamine with assisted therapy (MDMA-AT). However, in August, the US Food and Drug Administration (FDA) rejected it. At this point, it’s unclear when the therapy will be available, if ever.
“Not getting the FDA approval of any drug at this point is a setback for the field,” Lori Davis, MD, a senior research psychiatrist at the Birmingham Veterans Affairs (VA) Health Care System in Birmingham, Alabama, told Medscape Medical News.
Having an FDA-approved product would have helped increase public awareness of PTSD and driven interest in developing new therapies, said Davis, who is also adjunct professor of psychiatry at the Heersink School of Medicine, University of Alabama at Birmingham.
A Treatable Condition
So with MDMA-AT off the table, where does the field go next?
A public meeting in September hosted by the Reagan-Udall Foundation for the FDA in sought to answer that question. Agency officials joined representatives from the Department of Defense (DoD) and VA, patients, advocates, and industry representatives to discuss the current treatment landscape and what can be done to accelerate development of PTSD treatment.
Despite the common belief that PTSD is intractable, it “is a treatable condition,” Paula P. Schnurr, PhD, executive director of the VA National Center for PTSD, said at the meeting.
“There are effective treatments that work well for a lot of people, although not everyone has a satisfactory response,” she added.
The most effective psychotherapies are “trauma-focused,” and include cognitive processing therapy, eye movement desensitization and reprocessing, and prolonged exposure, according to the VA National Center for PTSD.
Three drugs have been approved by the FDA for PTSD: Venlafaxine (Effexor) in 1993, sertraline (Zoloft) in 1999, and paroxetine (Paxil) in 2000.
However, as the September meeting demonstrated, more therapies are needed.
“It’s clear to FDA and the federal government at large that there is an unmet need for safe and effective therapies to treat PTSD,” Bernard Fischer, MD, deputy director of the Division of Psychiatry in the Office of New Drugs at FDA’s Center for Drug Evaluation and Research, said at the meeting.
There is no shortage of research, Fischer added. Nearly 500 trials focused on PTSD are listed on clinicaltrials.gov are recruiting participants now or plan to soon.
Unsurprisingly, one of the primary drivers of PTSD therapeutics research is the VA. About 14% of the 5.7 million veterans who received care through the VA in 2023 had a diagnosis of PTSD.
“The US military is currently losing thousands of service members each year to PTSD- related disability discharges,” US Army Maj. Aaron Wolfgang, MD, a psychiatrist at the Walter Reed National Military Medical Center, said at the meeting. Only about 12%-20% of patients achieve remission with conventional therapies, added Wolfgang, who also is an assistant professor at the Uniformed Services University.
“For these reasons, establishing better treatments for PTSD is not only a matter of humanitarianism but also a pressing matter of national security,” he said.
The VA has committed at least $230 million to more than 140 active research projects in PTSD, Miriam J. Smyth, PhD, acting director of the clinical science, research and development service at the VA, said at the Reagan-Udall meeting.
One of the VA projects is the PTSD psychopharmacology initiative, which began in 2017 and now has 14 active clinical trials, said Smyth, who is also acting director for brain behavior and mental health at the VA. The first study should be finished by 2025.
The Million Veteran Program, with more than 1 million enrollees, has led to the discovery of genes related to re-experiencing traumatic memories and has confirmed that both PTSD and traumatic brain injury are risk factors for dementia, Smyth said.
The DoD has created a novel platform that establishes a common infrastructure for testing multiple drugs, called M-PACT. The platform allows sharing of placebo data across treatment arms. Drugs cycle off the platform if evidence indicates probability of success or failure.
Four trials are actively recruiting veterans and current service members. One is looking at vilazodone, approved in 2011 for major depressive disorder. It is being compared with placebo and fluoxetine in a trial that is currently recruiting.
Another trial will study daridorexant (sold as Quviviq), an orexin receptor antagonist, against placebo. The FDA approved daridorexant in 2022 as an insomnia treatment. A core issue in PTSD is sleep disruption, noted Davis.
New Therapies on the Way
Separately, Davis and colleagues are also studying methylphenidate, the stimulant used for attention-deficit/hyperactivity disorder. It may help with neurocognitive complaints and reduce PTSD symptoms, said Davis.
Because it is generic, few pharmaceutical manufacturers are likely to test it for PTSD, she said. But eventually, their work may lead a company to test newer stimulants for PTSD, she said.
Another potential therapeutic, BNC210, received Fast Track designation for PTSD from the FDA in 2019. Bionomics Limited in Australia will soon launch phase 3 trials of the investigational oral drug, which is a negative allosteric modulator of the alpha-7 nicotinic acetylcholine receptor. In late July, the company announced “ favorable feedback” from the agency on its phase 2 study, which led to the decision to move forward with larger trials.
Researchers at Brigham and Women’s Hospital have just reported that they may have found a target within the brain that will allow for transcranial magnetic stimulation (TMS) to ameliorate PTSD symptoms. They published results of a mapping effort in Nature Neuroscience and reported on one patient who had improved symptoms after receiving TMS for severe PTSD.
But perhaps one of the most promising treatments is a combination of sertraline and the new psychiatric medication brexpiprazole.
Brexpiprazole was developed by Otsuka Pharmaceutical and approved in the United States in 2015 as an adjunctive therapy to antidepressants for major depressive disorder and as a treatment for schizophrenia. In 2023, the FDA approved it for Alzheimer’s-related agitation. However, according to Otsuka, its mechanism of action is unknown.
Its efficacy may be mediated through a combination of partial agonist activity at serotonin 5-HT1A and dopamine D2 receptors, antagonist activity at serotonin 5-HT2A receptors, as well as antagonism of alpha-1B/2C receptors, said the company.
“It is the combination, rather than either alone, that’s going to have that broad synergistic pharmacology that is obviously potent for ameliorating the symptoms of PTSD,” said Davis, who has received consulting fees from Otsuka. “That’s an exciting development.”
Otsuka and partner Lundbeck Pharmaceuticals reported results in May from the companies’ phase 2 and 3 randomized clinical trials. The therapy achieved a statistically significant reduction (P <.05) in PTSD symptoms compared with sertraline plus placebo. This was without any supplemental psychotherapy.
The FDA accepted the companies’ new drug application in June and is expected to make a decision on approval in February 2025.
The Potential of Psychedelics
Though Lykos Therapeutics may have to go back to the drawing board on its MDMA-AT, psychedelics still have potential as PTSD therapies, Smyth said, who added that the VA is continuing to encourage study of MDMA and other psychedelic agents.
The VA issued a call for proposals for research on psychedelics in January, focused on MDMA or psilocybin in combination with psychotherapy. The administration received the first wave of applications early in the summer.
Scientific peer review panels made up of research experts from within and outside the VA have reviewed the applications and funding announcements are expected this fall, Smyth said.
Wolfgang, the Army psychiatrist, said, “Under the psychedelic treatment research clinical trial award, we welcome investigators to apply to what we anticipate will usher in a new era of innovation and hope for service members and their families who need it the most.”
Psychedelic studies are also proceeding without VA funding, as they have for years, when most of the trials were backed by universities or foundations or other private money. Johns Hopkins University is recruiting for a study in which patients would receive psilocybin along with trauma-focused psychotherapy, as is Ohio State University.
London-based Compass Pathways said in May that it successfully completed a phase 2 trial of Comp360, its synthetic psilocybin, in PTSD. The company has started a phase 3 study in treatment-resistant depression but has not given any further updates on PTSD.
Davis said that she believes that the FDA’s rejection of Lykos won’t lead to a shutdown of exploration of psychedelics.
“I think it informs these designs going forward, but it doesn’t eliminate that whole field of research,” she said.
Davis reported receiving consulting fees from Boehringer Ingelheim and Otsuka and research funding from Alkermes, the Patient-Centered Outcomes Research Institute, and the VA. Schnurr, Fischer, Smyth, and Wolfgang reported no relevant disclosures.
A version of this article appeared on Medscape.com.
Feds Sue Three Biggest Pharmacy Benefit Managers Over Insulin Costs
The US Federal Trade Commission (FTC) has sued the nation’s three largest pharmacy benefit managers (PBMs), alleging that they have steered patients to buying higher-priced insulins so that they can reap more profits.
The three PBMs administer 80% of prescriptions in the United States, the FTC said in a statement announcing the action.
The agency filed an administrative complaint, which means its allegations will be tried in a formal hearing before an administrative law judge. It will not be heard in a criminal court.
The three PBMs “have extracted millions of dollars off the backs of patients who need life-saving medications,” Rahul Rao, deputy director of the FTC’s Bureau of Competition, said in the statement.
The FTC action is not the first taken by a government agency against PBMs. Ohio Attorney General Dave Yost sued Express Scripts and Prime Therapeutics in March 2023, alleging antitrust violations.
The FTC’s complaint, which is not yet public, alleges that PBMs excluded lower-priced insulins from their formularies “in favor of high list price, highly rebated insulin products.”
The FTC describes a market in which PBMs, as they consolidated market power, began to extract higher rebates from drug makers. In turn, insulin manufacturers started raising their prices. That allowed PBMs to collect larger rebates, even as drug makers profited, according to the FTC.
The PBMs “engaged in unfair methods of competition and unfair acts or practices under Section 5 of the FTC Act by incentivizing manufacturers to inflate insulin list prices, restricting patients’ access to more affordable insulins on drug formularies and shifting the cost of high list price insulins to vulnerable patient populations,” said the FTC, in its statement.
Andrea Nelson, chief legal officer for The Cigna Group, said in a statement that the lawsuit “continues a troubling pattern from the FTC of unsubstantiated and ideologically-driven attacks on pharmacy benefit managers, following the FTC’s biased and misleading July 2024 report, which Express Scripts demanded the Commission retract earlier this week.”
Conduct ‘Raises Serious Concerns’
Drug makers are not off the hook, said the FTC. Mr. Rao said in a separate statement that “all drug manufacturers should be on notice that their participation in the type of conduct challenged here raises serious concerns and that the Bureau of Competition may recommend suing drug manufacturers in any future enforcement actions.”
The lawsuit comes on the heels of a report issued by the FTC in July, in which it accused the industry of driving small pharmacies out of business and of having extraordinary control over where Americans access prescription drugs and how much they pay.
The agency also noted in that report that some PBMs had still not responded to its requests for information, some 2 years after first asking.
Cigna’s Express Scripts sued the FTC on September 17, 2024, claiming that the report hurt the company’s reputation.
The report is “74 pages of unsupported innuendo leveled against Express Scripts and other PBMs under a false and defamatory headline and accompanied by a false and defamatory press release,” said the Cigna suit.
Cigna is seeking to have the report scrubbed from the FTC website and an injunction that would bar FTC Chairwoman Lina Khan from participating in any FTC business relating to Express Scripts.
Cigna’s Ms. Nelson accused the FTC of trying to “score political points” and said that forcing PBMs to include some drugs on its formularies “will drive drug prices higher in this country.”
CVS Health’s Caremark and UnitedHealth’s Optum also pushed back, as did the industry trade group, the Pharmaceutical Care Management Association.
“This action not only fails to accurately consider the role of the entire prescription drug supply chain, but disregards positive progress, supported by PBMs, in making insulin more affordable for patients,” the association said in a statement. “In contrast to the rhetoric, the current insulin market is actually working, with PBMs effectively leveraging greater competition to drive down insulin prices and doing their part to make insulin affordable for patients through innovative programs,” said the group.
“The FTC has missed the mark entirely,” David Whitrap, vice president for external affairs at CVS Health, said in a statement emailed to this news organization.
CVS Health members “on average pay less than $25, far below list prices and far below the Biden Administration’s $35 cap,” said Mr. Whitrap, who added that the PBM had protected customers from “pharma price-gouging.”
UnitedHealth’s Optum also said that it had reduced insulin prices for members to an average of less than $18 per month. “This baseless action demonstrates a profound misunderstanding of how drug pricing works,” wrote Elizabeth Hoff, a spokesperson for UnitedHealth’s Optum Rx, in an email to this news organization.
A version of this article appeared on Medscape.com.
The US Federal Trade Commission (FTC) has sued the nation’s three largest pharmacy benefit managers (PBMs), alleging that they have steered patients to buying higher-priced insulins so that they can reap more profits.
The three PBMs administer 80% of prescriptions in the United States, the FTC said in a statement announcing the action.
The agency filed an administrative complaint, which means its allegations will be tried in a formal hearing before an administrative law judge. It will not be heard in a criminal court.
The three PBMs “have extracted millions of dollars off the backs of patients who need life-saving medications,” Rahul Rao, deputy director of the FTC’s Bureau of Competition, said in the statement.
The FTC action is not the first taken by a government agency against PBMs. Ohio Attorney General Dave Yost sued Express Scripts and Prime Therapeutics in March 2023, alleging antitrust violations.
The FTC’s complaint, which is not yet public, alleges that PBMs excluded lower-priced insulins from their formularies “in favor of high list price, highly rebated insulin products.”
The FTC describes a market in which PBMs, as they consolidated market power, began to extract higher rebates from drug makers. In turn, insulin manufacturers started raising their prices. That allowed PBMs to collect larger rebates, even as drug makers profited, according to the FTC.
The PBMs “engaged in unfair methods of competition and unfair acts or practices under Section 5 of the FTC Act by incentivizing manufacturers to inflate insulin list prices, restricting patients’ access to more affordable insulins on drug formularies and shifting the cost of high list price insulins to vulnerable patient populations,” said the FTC, in its statement.
Andrea Nelson, chief legal officer for The Cigna Group, said in a statement that the lawsuit “continues a troubling pattern from the FTC of unsubstantiated and ideologically-driven attacks on pharmacy benefit managers, following the FTC’s biased and misleading July 2024 report, which Express Scripts demanded the Commission retract earlier this week.”
Conduct ‘Raises Serious Concerns’
Drug makers are not off the hook, said the FTC. Mr. Rao said in a separate statement that “all drug manufacturers should be on notice that their participation in the type of conduct challenged here raises serious concerns and that the Bureau of Competition may recommend suing drug manufacturers in any future enforcement actions.”
The lawsuit comes on the heels of a report issued by the FTC in July, in which it accused the industry of driving small pharmacies out of business and of having extraordinary control over where Americans access prescription drugs and how much they pay.
The agency also noted in that report that some PBMs had still not responded to its requests for information, some 2 years after first asking.
Cigna’s Express Scripts sued the FTC on September 17, 2024, claiming that the report hurt the company’s reputation.
The report is “74 pages of unsupported innuendo leveled against Express Scripts and other PBMs under a false and defamatory headline and accompanied by a false and defamatory press release,” said the Cigna suit.
Cigna is seeking to have the report scrubbed from the FTC website and an injunction that would bar FTC Chairwoman Lina Khan from participating in any FTC business relating to Express Scripts.
Cigna’s Ms. Nelson accused the FTC of trying to “score political points” and said that forcing PBMs to include some drugs on its formularies “will drive drug prices higher in this country.”
CVS Health’s Caremark and UnitedHealth’s Optum also pushed back, as did the industry trade group, the Pharmaceutical Care Management Association.
“This action not only fails to accurately consider the role of the entire prescription drug supply chain, but disregards positive progress, supported by PBMs, in making insulin more affordable for patients,” the association said in a statement. “In contrast to the rhetoric, the current insulin market is actually working, with PBMs effectively leveraging greater competition to drive down insulin prices and doing their part to make insulin affordable for patients through innovative programs,” said the group.
“The FTC has missed the mark entirely,” David Whitrap, vice president for external affairs at CVS Health, said in a statement emailed to this news organization.
CVS Health members “on average pay less than $25, far below list prices and far below the Biden Administration’s $35 cap,” said Mr. Whitrap, who added that the PBM had protected customers from “pharma price-gouging.”
UnitedHealth’s Optum also said that it had reduced insulin prices for members to an average of less than $18 per month. “This baseless action demonstrates a profound misunderstanding of how drug pricing works,” wrote Elizabeth Hoff, a spokesperson for UnitedHealth’s Optum Rx, in an email to this news organization.
A version of this article appeared on Medscape.com.
The US Federal Trade Commission (FTC) has sued the nation’s three largest pharmacy benefit managers (PBMs), alleging that they have steered patients to buying higher-priced insulins so that they can reap more profits.
The three PBMs administer 80% of prescriptions in the United States, the FTC said in a statement announcing the action.
The agency filed an administrative complaint, which means its allegations will be tried in a formal hearing before an administrative law judge. It will not be heard in a criminal court.
The three PBMs “have extracted millions of dollars off the backs of patients who need life-saving medications,” Rahul Rao, deputy director of the FTC’s Bureau of Competition, said in the statement.
The FTC action is not the first taken by a government agency against PBMs. Ohio Attorney General Dave Yost sued Express Scripts and Prime Therapeutics in March 2023, alleging antitrust violations.
The FTC’s complaint, which is not yet public, alleges that PBMs excluded lower-priced insulins from their formularies “in favor of high list price, highly rebated insulin products.”
The FTC describes a market in which PBMs, as they consolidated market power, began to extract higher rebates from drug makers. In turn, insulin manufacturers started raising their prices. That allowed PBMs to collect larger rebates, even as drug makers profited, according to the FTC.
The PBMs “engaged in unfair methods of competition and unfair acts or practices under Section 5 of the FTC Act by incentivizing manufacturers to inflate insulin list prices, restricting patients’ access to more affordable insulins on drug formularies and shifting the cost of high list price insulins to vulnerable patient populations,” said the FTC, in its statement.
Andrea Nelson, chief legal officer for The Cigna Group, said in a statement that the lawsuit “continues a troubling pattern from the FTC of unsubstantiated and ideologically-driven attacks on pharmacy benefit managers, following the FTC’s biased and misleading July 2024 report, which Express Scripts demanded the Commission retract earlier this week.”
Conduct ‘Raises Serious Concerns’
Drug makers are not off the hook, said the FTC. Mr. Rao said in a separate statement that “all drug manufacturers should be on notice that their participation in the type of conduct challenged here raises serious concerns and that the Bureau of Competition may recommend suing drug manufacturers in any future enforcement actions.”
The lawsuit comes on the heels of a report issued by the FTC in July, in which it accused the industry of driving small pharmacies out of business and of having extraordinary control over where Americans access prescription drugs and how much they pay.
The agency also noted in that report that some PBMs had still not responded to its requests for information, some 2 years after first asking.
Cigna’s Express Scripts sued the FTC on September 17, 2024, claiming that the report hurt the company’s reputation.
The report is “74 pages of unsupported innuendo leveled against Express Scripts and other PBMs under a false and defamatory headline and accompanied by a false and defamatory press release,” said the Cigna suit.
Cigna is seeking to have the report scrubbed from the FTC website and an injunction that would bar FTC Chairwoman Lina Khan from participating in any FTC business relating to Express Scripts.
Cigna’s Ms. Nelson accused the FTC of trying to “score political points” and said that forcing PBMs to include some drugs on its formularies “will drive drug prices higher in this country.”
CVS Health’s Caremark and UnitedHealth’s Optum also pushed back, as did the industry trade group, the Pharmaceutical Care Management Association.
“This action not only fails to accurately consider the role of the entire prescription drug supply chain, but disregards positive progress, supported by PBMs, in making insulin more affordable for patients,” the association said in a statement. “In contrast to the rhetoric, the current insulin market is actually working, with PBMs effectively leveraging greater competition to drive down insulin prices and doing their part to make insulin affordable for patients through innovative programs,” said the group.
“The FTC has missed the mark entirely,” David Whitrap, vice president for external affairs at CVS Health, said in a statement emailed to this news organization.
CVS Health members “on average pay less than $25, far below list prices and far below the Biden Administration’s $35 cap,” said Mr. Whitrap, who added that the PBM had protected customers from “pharma price-gouging.”
UnitedHealth’s Optum also said that it had reduced insulin prices for members to an average of less than $18 per month. “This baseless action demonstrates a profound misunderstanding of how drug pricing works,” wrote Elizabeth Hoff, a spokesperson for UnitedHealth’s Optum Rx, in an email to this news organization.
A version of this article appeared on Medscape.com.
Semaglutide Bests Liraglutide in Long-Term Weight Loss
Patients with obesity or type 2 diabetes (T2D) who stuck with their medication for a year lost more weight with semaglutide than with liraglutide, a new study reported.
Researchers at the Cleveland Clinic reviewed records for 3389 adult patients with obesity who were prescribed one of the glucagon-like peptide 1 (GLP-1) medications for either T2D or obesity between 2015 and 2022. They found that patients who took either semaglutide or liraglutide for obesity were more likely to lose weight than those prescribed the medications for T2D and that semaglutide was associated with greater weight loss.
The study, published in JAMA Network Open, identified “key characteristics that could inform the probability of achieving sustained weight loss of a magnitude large enough to provide clinically significant health benefits,” said lead author Hamlet Gasoyan, PhD, a staff investigator at the Center for Value-Based Care Research in the Department of Internal Medicine of Primary Care Institute, Cleveland Clinic, Cleveland.
Only about 40% of patients continued to take the medications at 1 year. Those who did not continue did not achieve the same level of weight loss, Dr. Gasoyan told this news organization. He and his colleagues will study the factors that lead patients to stop taking the medications in a future paper.
The results from the current paper give patients and clinicians reasonable expectations on the trajectory of weight loss when the drugs are prescribed for diabetes vs obesity, said Dr. Gasoyan, assistant professor of medicine at Cleveland Clinic Lerner College of Medicine of Case Western Reserve University, Cleveland.
Semaglutide Superior
Because of the study’s timeframe, the majority of GLP-1s were prescribed for T2D. Liraglutide was approved (as Saxenda) for obesity in December 2020 and semaglutide (as Wegovy) for obesity in June 2021.
The authors were able to capture fills under the brand names and doses approved by the US Food and Drug Administration (FDA) for obesity (Wegovy, 1.7 or 2.4 mg; Saxenda, 3.0 mg), as well as those approved for T2D (Ozempic, 0.5, 1.0, or 2.0 mg; Victoza, 1.2 or 1.8 mg).
The researchers reported that among the 3389 patients, 1341 (39.6%) were prescribed semaglutide and 1444 (42.6%) were prescribed liraglutide for T2D. For obesity, 227 (6.7%) were prescribed liraglutide, and 377 (11.1%) were prescribed semaglutide.
Overall, those with diabetes had a −3.2% mean weight change compared with those with obesity who had a −5.9% mean weight change.
Semaglutide consistently outperformed liraglutide, particularly in obesity.
Overall, at 1 year, the mean percentage weight change among those with obesity was −5.1% with semaglutide compared with −2.2% with liraglutide (P < .001).
At 1 year, among those with obesity who were persistent in semaglutide use (defined as 90-275 medication days) had a mean body weight of −12.9% vs −5.6% in those taking liraglutide.
Overall, about 40% of patients were persistent at 1 year. But the figure was higher for semaglutide (45.8%) and lower for liraglutide (35.6%).
Liraglutide requires daily injections compared with semaglutide that requires weekly injections. The authors did not study the reasons for medication adherence or discontinuation.
Key factors for achieving a greater than 10% weight loss — considered clinically meaningful — included taking semaglutide, receiving a GLP-1 for obesity, persistent medication use, high dosage, and being female.
Real-World Data Welcomed
Michael Weintraub, MD, an obesity medicine specialist and clinical assistant professor at NYU Langone Health, New York City, said that having real-world data on GLP-1 effectiveness has been much needed.
The researchers “did a really good job at stratifying these patients,” he told this news organization, saying that the study “adds to the literature in terms of what we might expect and what things we should look out for when we want to obtain the maximum degree of weight loss and attain overall better metabolic health for our patients.”
One strength: The researchers were able to capture when someone actually filled a prescription, he said. Clinicians don’t always know whether a prescription for a GLP-1 has been filled because patients might go without the drug because of insurance hurdles or supply issues, he said.
Dr. Weintraub was not surprised that the study showed that both GLP-1s produced more weight loss in those with obesity than in those with T2D, as that has become a common finding. No one has been able to explain why there is such a difference, said Dr. Weintraub. “As a field, we actually don’t know the reason behind that yet,” he said.
Given the small number of patients prescribed semaglutide for obesity, that “limits the generalizability,” he said.
Even so, semaglutide is increasingly proving superior, Dr. Weintraub said. “I would reach towards semaglutide every time either for individuals with type 2 diabetes or individuals with obesity,” he said. “The major limitation, though, is insurance coverage rather than, unfortunately, my clinical decision-making.”
He also still sees a role for liraglutide. It will go off patent soon and that could “lead to a lower price point and hopefully greater access for patients,” he said.
Dr. Gasoyan and Dr. Weintraub reported no relevant financial relationships. One coauthor reported receiving advisory board fees from Novo Nordisk and research funding from Eli Lilly during the conduct of the study.
A version of this article first appeared on Medscape.com.
Patients with obesity or type 2 diabetes (T2D) who stuck with their medication for a year lost more weight with semaglutide than with liraglutide, a new study reported.
Researchers at the Cleveland Clinic reviewed records for 3389 adult patients with obesity who were prescribed one of the glucagon-like peptide 1 (GLP-1) medications for either T2D or obesity between 2015 and 2022. They found that patients who took either semaglutide or liraglutide for obesity were more likely to lose weight than those prescribed the medications for T2D and that semaglutide was associated with greater weight loss.
The study, published in JAMA Network Open, identified “key characteristics that could inform the probability of achieving sustained weight loss of a magnitude large enough to provide clinically significant health benefits,” said lead author Hamlet Gasoyan, PhD, a staff investigator at the Center for Value-Based Care Research in the Department of Internal Medicine of Primary Care Institute, Cleveland Clinic, Cleveland.
Only about 40% of patients continued to take the medications at 1 year. Those who did not continue did not achieve the same level of weight loss, Dr. Gasoyan told this news organization. He and his colleagues will study the factors that lead patients to stop taking the medications in a future paper.
The results from the current paper give patients and clinicians reasonable expectations on the trajectory of weight loss when the drugs are prescribed for diabetes vs obesity, said Dr. Gasoyan, assistant professor of medicine at Cleveland Clinic Lerner College of Medicine of Case Western Reserve University, Cleveland.
Semaglutide Superior
Because of the study’s timeframe, the majority of GLP-1s were prescribed for T2D. Liraglutide was approved (as Saxenda) for obesity in December 2020 and semaglutide (as Wegovy) for obesity in June 2021.
The authors were able to capture fills under the brand names and doses approved by the US Food and Drug Administration (FDA) for obesity (Wegovy, 1.7 or 2.4 mg; Saxenda, 3.0 mg), as well as those approved for T2D (Ozempic, 0.5, 1.0, or 2.0 mg; Victoza, 1.2 or 1.8 mg).
The researchers reported that among the 3389 patients, 1341 (39.6%) were prescribed semaglutide and 1444 (42.6%) were prescribed liraglutide for T2D. For obesity, 227 (6.7%) were prescribed liraglutide, and 377 (11.1%) were prescribed semaglutide.
Overall, those with diabetes had a −3.2% mean weight change compared with those with obesity who had a −5.9% mean weight change.
Semaglutide consistently outperformed liraglutide, particularly in obesity.
Overall, at 1 year, the mean percentage weight change among those with obesity was −5.1% with semaglutide compared with −2.2% with liraglutide (P < .001).
At 1 year, among those with obesity who were persistent in semaglutide use (defined as 90-275 medication days) had a mean body weight of −12.9% vs −5.6% in those taking liraglutide.
Overall, about 40% of patients were persistent at 1 year. But the figure was higher for semaglutide (45.8%) and lower for liraglutide (35.6%).
Liraglutide requires daily injections compared with semaglutide that requires weekly injections. The authors did not study the reasons for medication adherence or discontinuation.
Key factors for achieving a greater than 10% weight loss — considered clinically meaningful — included taking semaglutide, receiving a GLP-1 for obesity, persistent medication use, high dosage, and being female.
Real-World Data Welcomed
Michael Weintraub, MD, an obesity medicine specialist and clinical assistant professor at NYU Langone Health, New York City, said that having real-world data on GLP-1 effectiveness has been much needed.
The researchers “did a really good job at stratifying these patients,” he told this news organization, saying that the study “adds to the literature in terms of what we might expect and what things we should look out for when we want to obtain the maximum degree of weight loss and attain overall better metabolic health for our patients.”
One strength: The researchers were able to capture when someone actually filled a prescription, he said. Clinicians don’t always know whether a prescription for a GLP-1 has been filled because patients might go without the drug because of insurance hurdles or supply issues, he said.
Dr. Weintraub was not surprised that the study showed that both GLP-1s produced more weight loss in those with obesity than in those with T2D, as that has become a common finding. No one has been able to explain why there is such a difference, said Dr. Weintraub. “As a field, we actually don’t know the reason behind that yet,” he said.
Given the small number of patients prescribed semaglutide for obesity, that “limits the generalizability,” he said.
Even so, semaglutide is increasingly proving superior, Dr. Weintraub said. “I would reach towards semaglutide every time either for individuals with type 2 diabetes or individuals with obesity,” he said. “The major limitation, though, is insurance coverage rather than, unfortunately, my clinical decision-making.”
He also still sees a role for liraglutide. It will go off patent soon and that could “lead to a lower price point and hopefully greater access for patients,” he said.
Dr. Gasoyan and Dr. Weintraub reported no relevant financial relationships. One coauthor reported receiving advisory board fees from Novo Nordisk and research funding from Eli Lilly during the conduct of the study.
A version of this article first appeared on Medscape.com.
Patients with obesity or type 2 diabetes (T2D) who stuck with their medication for a year lost more weight with semaglutide than with liraglutide, a new study reported.
Researchers at the Cleveland Clinic reviewed records for 3389 adult patients with obesity who were prescribed one of the glucagon-like peptide 1 (GLP-1) medications for either T2D or obesity between 2015 and 2022. They found that patients who took either semaglutide or liraglutide for obesity were more likely to lose weight than those prescribed the medications for T2D and that semaglutide was associated with greater weight loss.
The study, published in JAMA Network Open, identified “key characteristics that could inform the probability of achieving sustained weight loss of a magnitude large enough to provide clinically significant health benefits,” said lead author Hamlet Gasoyan, PhD, a staff investigator at the Center for Value-Based Care Research in the Department of Internal Medicine of Primary Care Institute, Cleveland Clinic, Cleveland.
Only about 40% of patients continued to take the medications at 1 year. Those who did not continue did not achieve the same level of weight loss, Dr. Gasoyan told this news organization. He and his colleagues will study the factors that lead patients to stop taking the medications in a future paper.
The results from the current paper give patients and clinicians reasonable expectations on the trajectory of weight loss when the drugs are prescribed for diabetes vs obesity, said Dr. Gasoyan, assistant professor of medicine at Cleveland Clinic Lerner College of Medicine of Case Western Reserve University, Cleveland.
Semaglutide Superior
Because of the study’s timeframe, the majority of GLP-1s were prescribed for T2D. Liraglutide was approved (as Saxenda) for obesity in December 2020 and semaglutide (as Wegovy) for obesity in June 2021.
The authors were able to capture fills under the brand names and doses approved by the US Food and Drug Administration (FDA) for obesity (Wegovy, 1.7 or 2.4 mg; Saxenda, 3.0 mg), as well as those approved for T2D (Ozempic, 0.5, 1.0, or 2.0 mg; Victoza, 1.2 or 1.8 mg).
The researchers reported that among the 3389 patients, 1341 (39.6%) were prescribed semaglutide and 1444 (42.6%) were prescribed liraglutide for T2D. For obesity, 227 (6.7%) were prescribed liraglutide, and 377 (11.1%) were prescribed semaglutide.
Overall, those with diabetes had a −3.2% mean weight change compared with those with obesity who had a −5.9% mean weight change.
Semaglutide consistently outperformed liraglutide, particularly in obesity.
Overall, at 1 year, the mean percentage weight change among those with obesity was −5.1% with semaglutide compared with −2.2% with liraglutide (P < .001).
At 1 year, among those with obesity who were persistent in semaglutide use (defined as 90-275 medication days) had a mean body weight of −12.9% vs −5.6% in those taking liraglutide.
Overall, about 40% of patients were persistent at 1 year. But the figure was higher for semaglutide (45.8%) and lower for liraglutide (35.6%).
Liraglutide requires daily injections compared with semaglutide that requires weekly injections. The authors did not study the reasons for medication adherence or discontinuation.
Key factors for achieving a greater than 10% weight loss — considered clinically meaningful — included taking semaglutide, receiving a GLP-1 for obesity, persistent medication use, high dosage, and being female.
Real-World Data Welcomed
Michael Weintraub, MD, an obesity medicine specialist and clinical assistant professor at NYU Langone Health, New York City, said that having real-world data on GLP-1 effectiveness has been much needed.
The researchers “did a really good job at stratifying these patients,” he told this news organization, saying that the study “adds to the literature in terms of what we might expect and what things we should look out for when we want to obtain the maximum degree of weight loss and attain overall better metabolic health for our patients.”
One strength: The researchers were able to capture when someone actually filled a prescription, he said. Clinicians don’t always know whether a prescription for a GLP-1 has been filled because patients might go without the drug because of insurance hurdles or supply issues, he said.
Dr. Weintraub was not surprised that the study showed that both GLP-1s produced more weight loss in those with obesity than in those with T2D, as that has become a common finding. No one has been able to explain why there is such a difference, said Dr. Weintraub. “As a field, we actually don’t know the reason behind that yet,” he said.
Given the small number of patients prescribed semaglutide for obesity, that “limits the generalizability,” he said.
Even so, semaglutide is increasingly proving superior, Dr. Weintraub said. “I would reach towards semaglutide every time either for individuals with type 2 diabetes or individuals with obesity,” he said. “The major limitation, though, is insurance coverage rather than, unfortunately, my clinical decision-making.”
He also still sees a role for liraglutide. It will go off patent soon and that could “lead to a lower price point and hopefully greater access for patients,” he said.
Dr. Gasoyan and Dr. Weintraub reported no relevant financial relationships. One coauthor reported receiving advisory board fees from Novo Nordisk and research funding from Eli Lilly during the conduct of the study.
A version of this article first appeared on Medscape.com.
FROM JAMA NETWORK OPEN
UVA Defends Medical School Dean, Hospital CEO After Docs Call for Their Removal
The University of Virginia (UVA) is defending the CEO of its health system and its medical school dean in the wake of a very public call for their removal.
At least 128 members of the University of Virginia faculty who are employed by both the medical school and the UVA Physicians Group wrote to the UVA Board of Visitors and its peer-elected faculty leaders, expressing no confidence in K. Craig Kent, MD, CEO of UVA Health and executive vice president for health affairs, and Melina Kibbe, MD, dean of the medical school and chief health affairs officer.
Dr. Kibbe, a vascular surgeon and researcher, is also the editor in chief of JAMA Surgery.
“We call for the immediate removal of Craig Kent and Melina Kibbe,” wrote the physicians.
The letter alleged that patient safety was compromised because doctors, nurses, and other staff were pressured to abstain from reporting safety concerns and that physicians had been hired “despite concerns regarding integrity and quality.” Those who raised safety concerns faced “explicit and implicit threats and retaliation,” including delays and denials of promotion and tenure, said the letter.
The September 5 letter did not include signatures. The authors said that names were being protected, but that they would share the names with a limited audience.
UVA President Jim Ryan took issue with the notion that the signees were anonymous. He said in his own letter to medical school faculty that some of the accusations were about matters that had already been addressed or that were being worked on. As far as allegations that he was not previously aware of, “we will do our best to investigate,” he said.
The faculty who signed the letter “have besmirched the reputations of not just Melina and Craig,” wrote Mr. Ryan. “They have unfairly — and I trust unwittingly — cast a shadow over the great work of the entire health system and medical school.”
The authors claimed that reports about bullying and harassment of trainees had been “suppressed, minimized, and subsequently altered.”
And they said that spending on leadership was prioritized over addressing clinical and technical staff shortages. Whistleblowers who reported fraud were not protected, and clinicians were pressured to modify patient records to “obfuscate adverse outcomes and boost productivity metrics,” they wrote.
The 128 members of the UVA Physicians Group who signed the letter represent about 10% of the 1400 medical school faculty members.
It is not the first time that Dr. Kent has been given a vote of no confidence. In 2017, when he was the dean of the College of Medicine at the Ohio State University, Dr. Kent was accused in a “no confidence” letter from 25 physicians and faculty of helping to undermine the school’s mission and taking actions that led to resignations and early retirements of many staff, the Columbus Dispatch reported.
William G. Crutchfield Jr., a member of the UVA Health System Board, defended Dr. Kent and Dr. Kibbe in a lengthy statement shared with this news organization. He said that UVA Health’s four hospitals had received “A” ratings for safety, and that the system has a 5.1% turnover rate compared with a national average of 8.3%.
Dr. Kent and Dr. Kibbe have recruited faculty from top academic medical centers, Mr. Crutchfield wrote.
“If our work environment were so toxic, these people would not have joined our faculty,” he wrote.
Mr. Crutchfield credited Dr. Kent and Dr. Kibbe with crafting a new 10-year strategic plan and for hiring a chief strategy officer to lead the plan — a move that replaced “expensive outside consultants.”
Mr. Ryan said in his letter that his inbox “is overflowing with testimonials from some of the 1200-plus faculty who did not sign the letter, who attest that the health system today — under Melina and Craig’s leadership — is in the best shape it has ever been in, and that they have addressed changes that have needed to be made for more than two decades.”
A request to see some of these positive testimonials was not answered by press time.
Mr. Crutchfield, like Mr. Ryan, said that the letter writers were doing more harm than good.
“If a small cabal of people hiding behind anonymity can force outstanding leaders out of UVA, it will make it extremely difficult to recruit outstanding new physicians, nurses, technicians, and administrators,” he wrote.
A version of this article first appeared on Medscape.com.
The University of Virginia (UVA) is defending the CEO of its health system and its medical school dean in the wake of a very public call for their removal.
At least 128 members of the University of Virginia faculty who are employed by both the medical school and the UVA Physicians Group wrote to the UVA Board of Visitors and its peer-elected faculty leaders, expressing no confidence in K. Craig Kent, MD, CEO of UVA Health and executive vice president for health affairs, and Melina Kibbe, MD, dean of the medical school and chief health affairs officer.
Dr. Kibbe, a vascular surgeon and researcher, is also the editor in chief of JAMA Surgery.
“We call for the immediate removal of Craig Kent and Melina Kibbe,” wrote the physicians.
The letter alleged that patient safety was compromised because doctors, nurses, and other staff were pressured to abstain from reporting safety concerns and that physicians had been hired “despite concerns regarding integrity and quality.” Those who raised safety concerns faced “explicit and implicit threats and retaliation,” including delays and denials of promotion and tenure, said the letter.
The September 5 letter did not include signatures. The authors said that names were being protected, but that they would share the names with a limited audience.
UVA President Jim Ryan took issue with the notion that the signees were anonymous. He said in his own letter to medical school faculty that some of the accusations were about matters that had already been addressed or that were being worked on. As far as allegations that he was not previously aware of, “we will do our best to investigate,” he said.
The faculty who signed the letter “have besmirched the reputations of not just Melina and Craig,” wrote Mr. Ryan. “They have unfairly — and I trust unwittingly — cast a shadow over the great work of the entire health system and medical school.”
The authors claimed that reports about bullying and harassment of trainees had been “suppressed, minimized, and subsequently altered.”
And they said that spending on leadership was prioritized over addressing clinical and technical staff shortages. Whistleblowers who reported fraud were not protected, and clinicians were pressured to modify patient records to “obfuscate adverse outcomes and boost productivity metrics,” they wrote.
The 128 members of the UVA Physicians Group who signed the letter represent about 10% of the 1400 medical school faculty members.
It is not the first time that Dr. Kent has been given a vote of no confidence. In 2017, when he was the dean of the College of Medicine at the Ohio State University, Dr. Kent was accused in a “no confidence” letter from 25 physicians and faculty of helping to undermine the school’s mission and taking actions that led to resignations and early retirements of many staff, the Columbus Dispatch reported.
William G. Crutchfield Jr., a member of the UVA Health System Board, defended Dr. Kent and Dr. Kibbe in a lengthy statement shared with this news organization. He said that UVA Health’s four hospitals had received “A” ratings for safety, and that the system has a 5.1% turnover rate compared with a national average of 8.3%.
Dr. Kent and Dr. Kibbe have recruited faculty from top academic medical centers, Mr. Crutchfield wrote.
“If our work environment were so toxic, these people would not have joined our faculty,” he wrote.
Mr. Crutchfield credited Dr. Kent and Dr. Kibbe with crafting a new 10-year strategic plan and for hiring a chief strategy officer to lead the plan — a move that replaced “expensive outside consultants.”
Mr. Ryan said in his letter that his inbox “is overflowing with testimonials from some of the 1200-plus faculty who did not sign the letter, who attest that the health system today — under Melina and Craig’s leadership — is in the best shape it has ever been in, and that they have addressed changes that have needed to be made for more than two decades.”
A request to see some of these positive testimonials was not answered by press time.
Mr. Crutchfield, like Mr. Ryan, said that the letter writers were doing more harm than good.
“If a small cabal of people hiding behind anonymity can force outstanding leaders out of UVA, it will make it extremely difficult to recruit outstanding new physicians, nurses, technicians, and administrators,” he wrote.
A version of this article first appeared on Medscape.com.
The University of Virginia (UVA) is defending the CEO of its health system and its medical school dean in the wake of a very public call for their removal.
At least 128 members of the University of Virginia faculty who are employed by both the medical school and the UVA Physicians Group wrote to the UVA Board of Visitors and its peer-elected faculty leaders, expressing no confidence in K. Craig Kent, MD, CEO of UVA Health and executive vice president for health affairs, and Melina Kibbe, MD, dean of the medical school and chief health affairs officer.
Dr. Kibbe, a vascular surgeon and researcher, is also the editor in chief of JAMA Surgery.
“We call for the immediate removal of Craig Kent and Melina Kibbe,” wrote the physicians.
The letter alleged that patient safety was compromised because doctors, nurses, and other staff were pressured to abstain from reporting safety concerns and that physicians had been hired “despite concerns regarding integrity and quality.” Those who raised safety concerns faced “explicit and implicit threats and retaliation,” including delays and denials of promotion and tenure, said the letter.
The September 5 letter did not include signatures. The authors said that names were being protected, but that they would share the names with a limited audience.
UVA President Jim Ryan took issue with the notion that the signees were anonymous. He said in his own letter to medical school faculty that some of the accusations were about matters that had already been addressed or that were being worked on. As far as allegations that he was not previously aware of, “we will do our best to investigate,” he said.
The faculty who signed the letter “have besmirched the reputations of not just Melina and Craig,” wrote Mr. Ryan. “They have unfairly — and I trust unwittingly — cast a shadow over the great work of the entire health system and medical school.”
The authors claimed that reports about bullying and harassment of trainees had been “suppressed, minimized, and subsequently altered.”
And they said that spending on leadership was prioritized over addressing clinical and technical staff shortages. Whistleblowers who reported fraud were not protected, and clinicians were pressured to modify patient records to “obfuscate adverse outcomes and boost productivity metrics,” they wrote.
The 128 members of the UVA Physicians Group who signed the letter represent about 10% of the 1400 medical school faculty members.
It is not the first time that Dr. Kent has been given a vote of no confidence. In 2017, when he was the dean of the College of Medicine at the Ohio State University, Dr. Kent was accused in a “no confidence” letter from 25 physicians and faculty of helping to undermine the school’s mission and taking actions that led to resignations and early retirements of many staff, the Columbus Dispatch reported.
William G. Crutchfield Jr., a member of the UVA Health System Board, defended Dr. Kent and Dr. Kibbe in a lengthy statement shared with this news organization. He said that UVA Health’s four hospitals had received “A” ratings for safety, and that the system has a 5.1% turnover rate compared with a national average of 8.3%.
Dr. Kent and Dr. Kibbe have recruited faculty from top academic medical centers, Mr. Crutchfield wrote.
“If our work environment were so toxic, these people would not have joined our faculty,” he wrote.
Mr. Crutchfield credited Dr. Kent and Dr. Kibbe with crafting a new 10-year strategic plan and for hiring a chief strategy officer to lead the plan — a move that replaced “expensive outside consultants.”
Mr. Ryan said in his letter that his inbox “is overflowing with testimonials from some of the 1200-plus faculty who did not sign the letter, who attest that the health system today — under Melina and Craig’s leadership — is in the best shape it has ever been in, and that they have addressed changes that have needed to be made for more than two decades.”
A request to see some of these positive testimonials was not answered by press time.
Mr. Crutchfield, like Mr. Ryan, said that the letter writers were doing more harm than good.
“If a small cabal of people hiding behind anonymity can force outstanding leaders out of UVA, it will make it extremely difficult to recruit outstanding new physicians, nurses, technicians, and administrators,” he wrote.
A version of this article first appeared on Medscape.com.
FDA Rejects MDMA-AT for PTSD, but Lykos, Others, Vow to Push on
The Food and Drug Administration’s (FDA) decision not to approve midomafetamine-assisted therapy (MDMA-AT) for posttraumatic stress disorder (PTSD) puts the therapy’s near-term future in doubt, but officials say the rejection may not knock it out of contention as an eventual therapeutic tool for a variety of conditions.
In August the agency declined to approve the drug with currently available study data and requested that the company conduct an additional phase 3 trial. The agency’s action had potentially devastating consequences for MDMA-AT’s sponsor, Lykos Therapeutics, and was a huge disappointment for researchers, clinicians, and patients who were optimistic that it would be a new option for a condition that affects 13-17 million Americans.
For now, no other company is poised to imminently seek FDA approval for MDMA.
Despite the setback, research into MDMA that combines different psychotherapeutic approaches continues. Currently, there are seven US studies actively recruiting participants, and another 13 are registered with an eye toward starting recruitment, as reported on ClinicalTrials.gov.
The lack of FDA approval “actually increases the opportunity now for us to do trials,” said Michael Ostacher, MD, professor of psychiatry and behavioral sciences at Stanford Medicine in California. Researchers won’t have to be sponsored by Lykos to get access to MDMA.
“There’s a lot of energy and interest in doing these studies,” he said in an interview, adding that philanthropic organizations and Veterans Affairs (VA) are contributing funds to support such studies.
The VA provided a statement saying that it “intends to gather rigorous scientific evidence on the potential efficacy and safety of psychedelic compounds when used in conjunction with psychotherapy.” It also noted that “these studies will be conducted under stringent safety protocols and will mark the first time since the 1960’s that VA is funding research on such compounds.”
Rachel Yehuda, PhD, director of the Center for Psychedelic Therapy Research at Icahn School of Medicine at Mount Sinai in New York City, said in an interview that the FDA rejection “raises questions about how to keep the work going.”
Without the FDA’s imprimatur, MDMA remains a schedule 1 drug, which means it has no valid medical use.
“It’s a lot more complicated and expensive to work with a scheduled compound than to work with a compound that has been approved,” Dr. Yehuda said.
Also, without Lykos or another drug company sponsor, investigators have to find an acceptable MDMA source on their own, said Dr. Yehuda, who was an investigator on a study in which Lykos provided MDMA but was not involved in study design, data collection, analysis, or manuscript preparation.
Lykos in Disarray
Within a week of the FDA’s decision, Lykos announced it was cutting its staff by 75% and that Rick Doblin, PhD, the founder and president of the Multidisciplinary Association for Psychedelic Studies (MAPS) that gave rise to Lykos, had resigned from the Lykos board.
A frequently controversial figure, Doblin has been attempting to legitimize MDMA as a therapy since the mid-1980s. He formed a public benefit corporation (PBC) in 2014 with an eye toward FDA approval. The PBC fully separated from MAPS in 2024 and became Lykos.
Although the FDA has left the door open to approval, Lykos has not released the agency’s complete response letter, so it’s not clear exactly what the FDA is seeking. In a statement, the company said it believes the issues “can be addressed with existing data, postapproval requirements, or through reference to the scientific literature.”
Lykos said in an email that it is working on “securing the meeting with the FDA” and that it “will work with the agency to determine what needs to be done to fulfill their requests.”
Soon after the FDA decision, Lykos was hit with another blow. The journal Psychopharmacology retracted an article that pooled six Lykos phase 2 studies, claiming the paper’s authors knew about unethical conduct before submission but did not inform the publisher.
Lykos said the issues could have been addressed through a correction and that it has filed a complaint with the Committee on Publication Ethics. It also noted that the misconduct at issue was reported to the FDA and Health Canada.
“However, we did not disclose the violations to the journal itself, an additional step we should have taken and regret not doing,” the company said. It added that the efficacy data in the paper were not part of the FDA submission.
Author Allison A. Feduccia, PhD, cofounder of Psychedelic Support, agreed with the retraction but disagreed with the wording. In a post on LinkedIn, she said she and other authors were not informed about the misconduct until years after the study’s submission.
Four authors — including Dr. Doblin — disagreed with the retraction.
Dr. Doblin said in a statement that he’d resigned from Lykos to escape the restrictions that came with being a fiduciary. “Now I can advocate and speak freely,” he said, adding that he could also return to his activist roots.
He predicted that Lykos would eventually gain FDA approval. But if Lykos can’t convince the agency, it have the necessary data already in hand; “potential FDA approval is now at least 2 years away, possibly more,” Dr. Doblin said in his statement.
Research Continues
Lykos is not the only company hoping to commercialize MDMA. Toronto-based Awakn Life Sciences has an MDMA preclinical development program for addiction. In addition, some companies are offering MDMA therapy through clinics, such as Numinus in Utah and Sunstone Therapies in Rockville, Maryland.
But Lykos was the closest to bringing a product to market. The company is still a sponsor of four MDMA-related clinical trials, three of which appear to be on hold. One study at the VA San Diego Healthcare System, San Diego, that is actively recruiting is an open-label trial to assess MDMA-AT in combination with brief Cognitive-Behavioral Conjoint Therapy for PTSD.
Those studies are among 13 US trials listed in ClinicalTrials.gov that have not yet begun recruiting and 7 that are actively recruiting.
Among them is a study of MDMA plus exposure therapy, funded by and conducted at Emory University in Atlanta. One of the Emory principal investigators, Barbara Rothbaum, MD, has also been named to a Lykos’ panel that would help ensure oversight of MDMA-AT post FDA approval.
Dr. Ostacher is an investigator in a study planned at VA Palo Alto Health Care System in California, that will compare MDMA-AT with cognitive processing therapy in veterans with severe PTSD. He said it will be open label in an effort to minimize expectation bias and issues with blinding — both problems that tripped up the Lykos application. Although placebo-controlled trials are the gold standard, it’s not ideal when “the purpose of the drug is for it to change how you see the world and yourself,” Dr. Ostacher said.
The study aims to see whether MDMA-AT is better than “a much shorter, less onerous, but quite evidence-based psychotherapy for PTSD,” he said.
The FDA’s decision is not the end of the road, said Dr. Ostacher. “Even though I think this makes for an obvious delay, I don’t think that it’s a permanent one,” he said.
Dr. Yehuda also said she is not ready to give up.
“We don’t plan on stopping — we plan on finding a way,” she said.
“In our experience, this is a very powerful approach that helps a lot of people that haven’t found help using other approaches, and when it’s in the hands of really trusted, experienced, ethical clinicians in a trusted environment, this could be a real game changer for people who have not been able to find belief by traditional methods,” she said.
Dr. Ostacher reported no relevant financial relationships. Dr. Yahuda is the principal investigator on clinical trials for the Center for Psychedelic Psychotherapy and Trauma Research that are sponsored by the Multidisciplinary Association for Psychedelic Studies and COMPASS Pathways.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration’s (FDA) decision not to approve midomafetamine-assisted therapy (MDMA-AT) for posttraumatic stress disorder (PTSD) puts the therapy’s near-term future in doubt, but officials say the rejection may not knock it out of contention as an eventual therapeutic tool for a variety of conditions.
In August the agency declined to approve the drug with currently available study data and requested that the company conduct an additional phase 3 trial. The agency’s action had potentially devastating consequences for MDMA-AT’s sponsor, Lykos Therapeutics, and was a huge disappointment for researchers, clinicians, and patients who were optimistic that it would be a new option for a condition that affects 13-17 million Americans.
For now, no other company is poised to imminently seek FDA approval for MDMA.
Despite the setback, research into MDMA that combines different psychotherapeutic approaches continues. Currently, there are seven US studies actively recruiting participants, and another 13 are registered with an eye toward starting recruitment, as reported on ClinicalTrials.gov.
The lack of FDA approval “actually increases the opportunity now for us to do trials,” said Michael Ostacher, MD, professor of psychiatry and behavioral sciences at Stanford Medicine in California. Researchers won’t have to be sponsored by Lykos to get access to MDMA.
“There’s a lot of energy and interest in doing these studies,” he said in an interview, adding that philanthropic organizations and Veterans Affairs (VA) are contributing funds to support such studies.
The VA provided a statement saying that it “intends to gather rigorous scientific evidence on the potential efficacy and safety of psychedelic compounds when used in conjunction with psychotherapy.” It also noted that “these studies will be conducted under stringent safety protocols and will mark the first time since the 1960’s that VA is funding research on such compounds.”
Rachel Yehuda, PhD, director of the Center for Psychedelic Therapy Research at Icahn School of Medicine at Mount Sinai in New York City, said in an interview that the FDA rejection “raises questions about how to keep the work going.”
Without the FDA’s imprimatur, MDMA remains a schedule 1 drug, which means it has no valid medical use.
“It’s a lot more complicated and expensive to work with a scheduled compound than to work with a compound that has been approved,” Dr. Yehuda said.
Also, without Lykos or another drug company sponsor, investigators have to find an acceptable MDMA source on their own, said Dr. Yehuda, who was an investigator on a study in which Lykos provided MDMA but was not involved in study design, data collection, analysis, or manuscript preparation.
Lykos in Disarray
Within a week of the FDA’s decision, Lykos announced it was cutting its staff by 75% and that Rick Doblin, PhD, the founder and president of the Multidisciplinary Association for Psychedelic Studies (MAPS) that gave rise to Lykos, had resigned from the Lykos board.
A frequently controversial figure, Doblin has been attempting to legitimize MDMA as a therapy since the mid-1980s. He formed a public benefit corporation (PBC) in 2014 with an eye toward FDA approval. The PBC fully separated from MAPS in 2024 and became Lykos.
Although the FDA has left the door open to approval, Lykos has not released the agency’s complete response letter, so it’s not clear exactly what the FDA is seeking. In a statement, the company said it believes the issues “can be addressed with existing data, postapproval requirements, or through reference to the scientific literature.”
Lykos said in an email that it is working on “securing the meeting with the FDA” and that it “will work with the agency to determine what needs to be done to fulfill their requests.”
Soon after the FDA decision, Lykos was hit with another blow. The journal Psychopharmacology retracted an article that pooled six Lykos phase 2 studies, claiming the paper’s authors knew about unethical conduct before submission but did not inform the publisher.
Lykos said the issues could have been addressed through a correction and that it has filed a complaint with the Committee on Publication Ethics. It also noted that the misconduct at issue was reported to the FDA and Health Canada.
“However, we did not disclose the violations to the journal itself, an additional step we should have taken and regret not doing,” the company said. It added that the efficacy data in the paper were not part of the FDA submission.
Author Allison A. Feduccia, PhD, cofounder of Psychedelic Support, agreed with the retraction but disagreed with the wording. In a post on LinkedIn, she said she and other authors were not informed about the misconduct until years after the study’s submission.
Four authors — including Dr. Doblin — disagreed with the retraction.
Dr. Doblin said in a statement that he’d resigned from Lykos to escape the restrictions that came with being a fiduciary. “Now I can advocate and speak freely,” he said, adding that he could also return to his activist roots.
He predicted that Lykos would eventually gain FDA approval. But if Lykos can’t convince the agency, it have the necessary data already in hand; “potential FDA approval is now at least 2 years away, possibly more,” Dr. Doblin said in his statement.
Research Continues
Lykos is not the only company hoping to commercialize MDMA. Toronto-based Awakn Life Sciences has an MDMA preclinical development program for addiction. In addition, some companies are offering MDMA therapy through clinics, such as Numinus in Utah and Sunstone Therapies in Rockville, Maryland.
But Lykos was the closest to bringing a product to market. The company is still a sponsor of four MDMA-related clinical trials, three of which appear to be on hold. One study at the VA San Diego Healthcare System, San Diego, that is actively recruiting is an open-label trial to assess MDMA-AT in combination with brief Cognitive-Behavioral Conjoint Therapy for PTSD.
Those studies are among 13 US trials listed in ClinicalTrials.gov that have not yet begun recruiting and 7 that are actively recruiting.
Among them is a study of MDMA plus exposure therapy, funded by and conducted at Emory University in Atlanta. One of the Emory principal investigators, Barbara Rothbaum, MD, has also been named to a Lykos’ panel that would help ensure oversight of MDMA-AT post FDA approval.
Dr. Ostacher is an investigator in a study planned at VA Palo Alto Health Care System in California, that will compare MDMA-AT with cognitive processing therapy in veterans with severe PTSD. He said it will be open label in an effort to minimize expectation bias and issues with blinding — both problems that tripped up the Lykos application. Although placebo-controlled trials are the gold standard, it’s not ideal when “the purpose of the drug is for it to change how you see the world and yourself,” Dr. Ostacher said.
The study aims to see whether MDMA-AT is better than “a much shorter, less onerous, but quite evidence-based psychotherapy for PTSD,” he said.
The FDA’s decision is not the end of the road, said Dr. Ostacher. “Even though I think this makes for an obvious delay, I don’t think that it’s a permanent one,” he said.
Dr. Yehuda also said she is not ready to give up.
“We don’t plan on stopping — we plan on finding a way,” she said.
“In our experience, this is a very powerful approach that helps a lot of people that haven’t found help using other approaches, and when it’s in the hands of really trusted, experienced, ethical clinicians in a trusted environment, this could be a real game changer for people who have not been able to find belief by traditional methods,” she said.
Dr. Ostacher reported no relevant financial relationships. Dr. Yahuda is the principal investigator on clinical trials for the Center for Psychedelic Psychotherapy and Trauma Research that are sponsored by the Multidisciplinary Association for Psychedelic Studies and COMPASS Pathways.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration’s (FDA) decision not to approve midomafetamine-assisted therapy (MDMA-AT) for posttraumatic stress disorder (PTSD) puts the therapy’s near-term future in doubt, but officials say the rejection may not knock it out of contention as an eventual therapeutic tool for a variety of conditions.
In August the agency declined to approve the drug with currently available study data and requested that the company conduct an additional phase 3 trial. The agency’s action had potentially devastating consequences for MDMA-AT’s sponsor, Lykos Therapeutics, and was a huge disappointment for researchers, clinicians, and patients who were optimistic that it would be a new option for a condition that affects 13-17 million Americans.
For now, no other company is poised to imminently seek FDA approval for MDMA.
Despite the setback, research into MDMA that combines different psychotherapeutic approaches continues. Currently, there are seven US studies actively recruiting participants, and another 13 are registered with an eye toward starting recruitment, as reported on ClinicalTrials.gov.
The lack of FDA approval “actually increases the opportunity now for us to do trials,” said Michael Ostacher, MD, professor of psychiatry and behavioral sciences at Stanford Medicine in California. Researchers won’t have to be sponsored by Lykos to get access to MDMA.
“There’s a lot of energy and interest in doing these studies,” he said in an interview, adding that philanthropic organizations and Veterans Affairs (VA) are contributing funds to support such studies.
The VA provided a statement saying that it “intends to gather rigorous scientific evidence on the potential efficacy and safety of psychedelic compounds when used in conjunction with psychotherapy.” It also noted that “these studies will be conducted under stringent safety protocols and will mark the first time since the 1960’s that VA is funding research on such compounds.”
Rachel Yehuda, PhD, director of the Center for Psychedelic Therapy Research at Icahn School of Medicine at Mount Sinai in New York City, said in an interview that the FDA rejection “raises questions about how to keep the work going.”
Without the FDA’s imprimatur, MDMA remains a schedule 1 drug, which means it has no valid medical use.
“It’s a lot more complicated and expensive to work with a scheduled compound than to work with a compound that has been approved,” Dr. Yehuda said.
Also, without Lykos or another drug company sponsor, investigators have to find an acceptable MDMA source on their own, said Dr. Yehuda, who was an investigator on a study in which Lykos provided MDMA but was not involved in study design, data collection, analysis, or manuscript preparation.
Lykos in Disarray
Within a week of the FDA’s decision, Lykos announced it was cutting its staff by 75% and that Rick Doblin, PhD, the founder and president of the Multidisciplinary Association for Psychedelic Studies (MAPS) that gave rise to Lykos, had resigned from the Lykos board.
A frequently controversial figure, Doblin has been attempting to legitimize MDMA as a therapy since the mid-1980s. He formed a public benefit corporation (PBC) in 2014 with an eye toward FDA approval. The PBC fully separated from MAPS in 2024 and became Lykos.
Although the FDA has left the door open to approval, Lykos has not released the agency’s complete response letter, so it’s not clear exactly what the FDA is seeking. In a statement, the company said it believes the issues “can be addressed with existing data, postapproval requirements, or through reference to the scientific literature.”
Lykos said in an email that it is working on “securing the meeting with the FDA” and that it “will work with the agency to determine what needs to be done to fulfill their requests.”
Soon after the FDA decision, Lykos was hit with another blow. The journal Psychopharmacology retracted an article that pooled six Lykos phase 2 studies, claiming the paper’s authors knew about unethical conduct before submission but did not inform the publisher.
Lykos said the issues could have been addressed through a correction and that it has filed a complaint with the Committee on Publication Ethics. It also noted that the misconduct at issue was reported to the FDA and Health Canada.
“However, we did not disclose the violations to the journal itself, an additional step we should have taken and regret not doing,” the company said. It added that the efficacy data in the paper were not part of the FDA submission.
Author Allison A. Feduccia, PhD, cofounder of Psychedelic Support, agreed with the retraction but disagreed with the wording. In a post on LinkedIn, she said she and other authors were not informed about the misconduct until years after the study’s submission.
Four authors — including Dr. Doblin — disagreed with the retraction.
Dr. Doblin said in a statement that he’d resigned from Lykos to escape the restrictions that came with being a fiduciary. “Now I can advocate and speak freely,” he said, adding that he could also return to his activist roots.
He predicted that Lykos would eventually gain FDA approval. But if Lykos can’t convince the agency, it have the necessary data already in hand; “potential FDA approval is now at least 2 years away, possibly more,” Dr. Doblin said in his statement.
Research Continues
Lykos is not the only company hoping to commercialize MDMA. Toronto-based Awakn Life Sciences has an MDMA preclinical development program for addiction. In addition, some companies are offering MDMA therapy through clinics, such as Numinus in Utah and Sunstone Therapies in Rockville, Maryland.
But Lykos was the closest to bringing a product to market. The company is still a sponsor of four MDMA-related clinical trials, three of which appear to be on hold. One study at the VA San Diego Healthcare System, San Diego, that is actively recruiting is an open-label trial to assess MDMA-AT in combination with brief Cognitive-Behavioral Conjoint Therapy for PTSD.
Those studies are among 13 US trials listed in ClinicalTrials.gov that have not yet begun recruiting and 7 that are actively recruiting.
Among them is a study of MDMA plus exposure therapy, funded by and conducted at Emory University in Atlanta. One of the Emory principal investigators, Barbara Rothbaum, MD, has also been named to a Lykos’ panel that would help ensure oversight of MDMA-AT post FDA approval.
Dr. Ostacher is an investigator in a study planned at VA Palo Alto Health Care System in California, that will compare MDMA-AT with cognitive processing therapy in veterans with severe PTSD. He said it will be open label in an effort to minimize expectation bias and issues with blinding — both problems that tripped up the Lykos application. Although placebo-controlled trials are the gold standard, it’s not ideal when “the purpose of the drug is for it to change how you see the world and yourself,” Dr. Ostacher said.
The study aims to see whether MDMA-AT is better than “a much shorter, less onerous, but quite evidence-based psychotherapy for PTSD,” he said.
The FDA’s decision is not the end of the road, said Dr. Ostacher. “Even though I think this makes for an obvious delay, I don’t think that it’s a permanent one,” he said.
Dr. Yehuda also said she is not ready to give up.
“We don’t plan on stopping — we plan on finding a way,” she said.
“In our experience, this is a very powerful approach that helps a lot of people that haven’t found help using other approaches, and when it’s in the hands of really trusted, experienced, ethical clinicians in a trusted environment, this could be a real game changer for people who have not been able to find belief by traditional methods,” she said.
Dr. Ostacher reported no relevant financial relationships. Dr. Yahuda is the principal investigator on clinical trials for the Center for Psychedelic Psychotherapy and Trauma Research that are sponsored by the Multidisciplinary Association for Psychedelic Studies and COMPASS Pathways.
A version of this article first appeared on Medscape.com.
Do Neurology Patient Advocacy Groups Wield Too Much Power?
Advocacy groups for patients with neurologic disorders have become a common feature in the landscape of drug and device development and federal research funding allocation.
On Capitol Hill, advocates have racked up some impressive legislative wins that aim to set a federal agenda for developing new medications.
At the Food and Drug Administration (FDA), advocacy groups played a significant role in several recent high-profile and controversial approvals for drugs for Alzheimer’s disease, Duchenne muscular dystrophy (DMD), and amyotrophic lateral sclerosis (ALS).
Such gains suggest these groups are growing in power. But with these wins come questions about whether large advocacy organizations — some of which receive significant industry funding — wield too much influence.
“You need to think very carefully about how you open these processes up to greater patient involvement,” Matthew S. McCoy, PhD, assistant professor of medical ethics and health policy at the University of Pennsylvania, Philadelphia, told this news organization. It’s important not to “end up with a situation where it’s the best-connected, the most well-resourced, the most-savvy patient organizations that are able to exercise outsize influence.”
Just because a group has deep pockets does not mean that its priorities align with the disease burden. And not every patient population is represented by a professionalized patient advocacy organization, Dr. McCoy noted. “There is the potential for the rich to get richer.”
A Seat at the Table
Long ago, the FDA and the National Institutes of Health (NIH) began giving patients a seat at the table, in part because of the path blazed by AIDS activists in the late 1980s and early 1990s, said Dr. McCoy.
Patient advocacy is often visible during FDA advisory committee meetings. The agency usually allows an hour, sometimes more, for members of the public to express support or concerns about the product being reviewed. Patients and caregivers — often aided by advocacy organizations — also submit hundreds, sometimes thousands, of letters before a product review.
The Alzheimer’s Association spent years advocating for approval of the anti-amyloid agent aducanumab (Aduhelm, Biogen/Eisai). In 2020, the organization urged patients and caregivers to submit written and oral testimony to the FDA advisory panel that was reviewing the drug. Despite patients’ pleas, the panel ultimately declined to support the drug’s approval, citing safety concerns and limited evidence of efficacy.
As controversy swirled around the medication — which had the potential for life-threatening brain swelling — advocates continued to apply pressure. Going against the expert panel’s recommendation, in June 2021, the FDA granted accelerated approval prompting three of the panelists to resign in protest.
Aducanumab’s initial price — $56,000 a year — was seen as a major threat to the viability of Medicare. Still, the Alzheimer’s Association stood behind the decision to approve the drug. But by early 2024, Biogen/Eisai said they would stop selling aducanumab, citing other priorities.
Once again patient advocates showed up in March 2022 when the FDA advisers were reviewing Amylyx Pharmaceuticals’ ALS drug Relyvrio (sodium phenylbutyrate and taurursodiol). Trials had showed limited efficacy, but patients testified they would accept greater risk for a chance to be treated with the drug. The committee ultimately voted against approval; 6 months later, the FDA approved Relyvrio anyway.
In April 2024, Amylyx removed Relyvrio from the market following phase 3 trial results that showed no difference between the treatment and placebo.
The drug manufacturer Sarepta Therapeutics, which develops treatments for genetic conditions such as DMD, has a history of working with — and funding — patient advocacy groups. The company encourages nonprofits to apply for grants or sponsorship on its website. At a 2016 advisory committee, when Sarepta was seeking approval of its first DMD therapy eteplirsen (Exondys 51), 52 speakers, most from patient advocacy groups, pleaded for the drug’s approval. When the panel voted no, Sarepta mobilized families to pressure the agency. Exondys was eventually approved.
In June, Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, unilaterally gave final expanded approval to Sarepta Therapeutics’ gene therapy Elevidys for DMD. Dr. Marks overrode his own FDA reviewers, who said the product lacked substantial evidence of efficacy. He acknowledged the drug had not met its primary endpoint but said he found secondary and exploratory endpoints “compelling” and cited an unmet medical need.
In an opinion piece in The Washington Post, Aaron Kesselheim, MD, JD, MPH, the director of the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, Boston, Massachusetts, and a former member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, questioned the approval stating that it undermined both public trust and manufacturers’ incentives to do the hard work of proving effectiveness.
Patient Voices the ‘Secret Sauce’
Drugmakers aren’t alone in seeing the value of having patients speak directly to government entities. When the Michael J. Fox Foundation wanted to gather cosponsors for the National Plan to End Parkinson’s Act, which President Joe Biden signed into law in July, it recruited and trained patients and caregivers for congressional meetings, said Ted Thompson, senior vice president of public policy at the foundation.
Having those individuals “making the personal case for how this disease affects their families ... was really the secret sauce,” in garnering a large number of cosponsors and getting legislation signed into law within 2 years of its introduction, Mr. Thompson told this news organization.
ALS advocacy groups launched a similar campaign to secure passage of the Accelerating Access to Critical Therapies for ALS Act in 2021.
Both pieces of legislation seek to set a federal agenda for developing new therapies in neurodegenerative diseases, in part by directing the FDA and NIH to fund research, engage patients more directly, and form public-private partnerships and councils to spur innovation.
But some said patient advocates are still coming far too late to the party.
“By the time you hear from patient groups at the meetings at the FDA, often the best opportunities for their input are long past,” Leah Zoe Gibson Rand, DPhil, a research scientist with the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, told this news organization. There should be more focus on the patient perspective earlier in drug development and trial design.
“There are some things that the patient voice could uniquely tell the agency,” said Holly Fernandez Lynch, JD, associate professor of medical ethics and health policy at the University of Pennsylvania. Patients can give insight on what it means to live with a disease, what symptoms are particularly burdensome, and which endpoints matter.
But, she said, “listening to the patient voice cannot mean that FDA just steps aside and lets anything on the market that patients are willing to try.” Individuals “who lack good treatment options have a very good reason to want to try things that haven’t yet been proven.”
If the FDA allows drugs on the market just because patients are willing to try, “5 or 10 years down the road, it’s not at all clear that we would end up with drugs that are better, or drugs that work, or drugs that we know anything more about,” said Dr. Lynch.
Does Taking Industry Money Equal Conflicts of Interest?
Many patient advocacy organizations receive funding from drug companies, medical device makers, or other industry sources, but they aren’t always transparent about how much or from which companies, according to studies.
The Alzheimer’s Association continued to push for the approval of aducanumab, even as the group received millions of dollars from the drugmakers. The association was accused of failing to disclose the potential conflict. It still lobbied for approval, even after the FDA advisers in 2020 voted against the drug.
It is not uncommon for individuals who speak in favor of a product’s approval to receive money for transportation and/or lodging from the drug’s manufacturer. In 2018, Dr. McCoy and colleagues reported in JAMA Internal Medicine that, between 2009 and 2017, a quarter of the speakers at the Anesthetic and Analgesic Drug Products Advisory Committee had conflicts of interest (COIs), mostly from industry, and that they were not disclosed in approximately 20% of the instances.
In a 2017 study of 104 large patient advocacy organizations published in The New England Journal of Medicine, Dr. McCoy and colleagues reported that 83% had received funds from industry. At least 39% had a current or former industry executive on the governing board, and 12% had a current or former industry executive in a board leadership position. Of the 104, 38 were focused on cancer and 13 on neurologic conditions. Of these, only 12% had published policies for managing institutional COIs.
Dr. McCoy emphasized the industry’s reliance on partnering with patient groups, particularly during FDA advisory committee meetings. “The sponsors wouldn’t be paying for patients to show up and give these testimonies if they didn’t think it made a difference. The audience isn’t just panel members; it’s also agency officials and maybe elected officials as well.”
“The Fox Foundation, with a $300 million-plus budget, gets about $5-$6 million a year from industry,” said Mr. Thompson. The money is earmarked for the organization’s Parkinson’s Disease Education Consortium; none goes toward advocacy. And, “the foundation has never specifically endorsed a product or device.”
When organizations that receive industry funding back a particular product, “it does appear to be [a conflict], and whether it is an actual one or not, appearances sometimes are all that matter,” said Mr. Thompson.
Dr. Lynch said accepting industry money “is a really significant conflict.” While advocates might need that money to fund advocacy efforts or make grants to advance research priorities, the acceptance might hinder willingness to demand evidence or to complain about a product’s price tag. “You don’t want to bite the hand that feeds you, right?”
Both Dr. McCoy and Dr. Lynch said patient groups — and individual patients — should at a minimum disclose industry funding, especially when speaking at an advisory committee.
Federal agencies and members of Congress actively seek patient input when considering legislation and funding priorities. But the individuals testifying at an advisory committee aren’t likely to represent all patients, and there’s a danger that they are just the loudest voices, said Dr. McCoy.
“We need to think more carefully about how we actually understand the preferences of a big, diverse patient population,” he said.
Dr. Lynch agreed.
Within the ALS community, “a lot of people who take different perspectives than some of those that are the leading voices get shouted down, and their voices get drowned out, and they get attacked on social media,” she said.
The group may be at the table, “but they’re just one voice at the table,” she said.
Dr. McCoy reported that his wife works for the Leukemia & Lymphoma Society, a patient advocacy organization. Dr. Rand reported no relevant financial relationships. Dr. Lynch received funding from Arnold Ventures and the Greenwall Foundation for work related to the FDA and patient advocacy.
A version of this article first appeared on Medscape.com.
Advocacy groups for patients with neurologic disorders have become a common feature in the landscape of drug and device development and federal research funding allocation.
On Capitol Hill, advocates have racked up some impressive legislative wins that aim to set a federal agenda for developing new medications.
At the Food and Drug Administration (FDA), advocacy groups played a significant role in several recent high-profile and controversial approvals for drugs for Alzheimer’s disease, Duchenne muscular dystrophy (DMD), and amyotrophic lateral sclerosis (ALS).
Such gains suggest these groups are growing in power. But with these wins come questions about whether large advocacy organizations — some of which receive significant industry funding — wield too much influence.
“You need to think very carefully about how you open these processes up to greater patient involvement,” Matthew S. McCoy, PhD, assistant professor of medical ethics and health policy at the University of Pennsylvania, Philadelphia, told this news organization. It’s important not to “end up with a situation where it’s the best-connected, the most well-resourced, the most-savvy patient organizations that are able to exercise outsize influence.”
Just because a group has deep pockets does not mean that its priorities align with the disease burden. And not every patient population is represented by a professionalized patient advocacy organization, Dr. McCoy noted. “There is the potential for the rich to get richer.”
A Seat at the Table
Long ago, the FDA and the National Institutes of Health (NIH) began giving patients a seat at the table, in part because of the path blazed by AIDS activists in the late 1980s and early 1990s, said Dr. McCoy.
Patient advocacy is often visible during FDA advisory committee meetings. The agency usually allows an hour, sometimes more, for members of the public to express support or concerns about the product being reviewed. Patients and caregivers — often aided by advocacy organizations — also submit hundreds, sometimes thousands, of letters before a product review.
The Alzheimer’s Association spent years advocating for approval of the anti-amyloid agent aducanumab (Aduhelm, Biogen/Eisai). In 2020, the organization urged patients and caregivers to submit written and oral testimony to the FDA advisory panel that was reviewing the drug. Despite patients’ pleas, the panel ultimately declined to support the drug’s approval, citing safety concerns and limited evidence of efficacy.
As controversy swirled around the medication — which had the potential for life-threatening brain swelling — advocates continued to apply pressure. Going against the expert panel’s recommendation, in June 2021, the FDA granted accelerated approval prompting three of the panelists to resign in protest.
Aducanumab’s initial price — $56,000 a year — was seen as a major threat to the viability of Medicare. Still, the Alzheimer’s Association stood behind the decision to approve the drug. But by early 2024, Biogen/Eisai said they would stop selling aducanumab, citing other priorities.
Once again patient advocates showed up in March 2022 when the FDA advisers were reviewing Amylyx Pharmaceuticals’ ALS drug Relyvrio (sodium phenylbutyrate and taurursodiol). Trials had showed limited efficacy, but patients testified they would accept greater risk for a chance to be treated with the drug. The committee ultimately voted against approval; 6 months later, the FDA approved Relyvrio anyway.
In April 2024, Amylyx removed Relyvrio from the market following phase 3 trial results that showed no difference between the treatment and placebo.
The drug manufacturer Sarepta Therapeutics, which develops treatments for genetic conditions such as DMD, has a history of working with — and funding — patient advocacy groups. The company encourages nonprofits to apply for grants or sponsorship on its website. At a 2016 advisory committee, when Sarepta was seeking approval of its first DMD therapy eteplirsen (Exondys 51), 52 speakers, most from patient advocacy groups, pleaded for the drug’s approval. When the panel voted no, Sarepta mobilized families to pressure the agency. Exondys was eventually approved.
In June, Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, unilaterally gave final expanded approval to Sarepta Therapeutics’ gene therapy Elevidys for DMD. Dr. Marks overrode his own FDA reviewers, who said the product lacked substantial evidence of efficacy. He acknowledged the drug had not met its primary endpoint but said he found secondary and exploratory endpoints “compelling” and cited an unmet medical need.
In an opinion piece in The Washington Post, Aaron Kesselheim, MD, JD, MPH, the director of the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, Boston, Massachusetts, and a former member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, questioned the approval stating that it undermined both public trust and manufacturers’ incentives to do the hard work of proving effectiveness.
Patient Voices the ‘Secret Sauce’
Drugmakers aren’t alone in seeing the value of having patients speak directly to government entities. When the Michael J. Fox Foundation wanted to gather cosponsors for the National Plan to End Parkinson’s Act, which President Joe Biden signed into law in July, it recruited and trained patients and caregivers for congressional meetings, said Ted Thompson, senior vice president of public policy at the foundation.
Having those individuals “making the personal case for how this disease affects their families ... was really the secret sauce,” in garnering a large number of cosponsors and getting legislation signed into law within 2 years of its introduction, Mr. Thompson told this news organization.
ALS advocacy groups launched a similar campaign to secure passage of the Accelerating Access to Critical Therapies for ALS Act in 2021.
Both pieces of legislation seek to set a federal agenda for developing new therapies in neurodegenerative diseases, in part by directing the FDA and NIH to fund research, engage patients more directly, and form public-private partnerships and councils to spur innovation.
But some said patient advocates are still coming far too late to the party.
“By the time you hear from patient groups at the meetings at the FDA, often the best opportunities for their input are long past,” Leah Zoe Gibson Rand, DPhil, a research scientist with the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, told this news organization. There should be more focus on the patient perspective earlier in drug development and trial design.
“There are some things that the patient voice could uniquely tell the agency,” said Holly Fernandez Lynch, JD, associate professor of medical ethics and health policy at the University of Pennsylvania. Patients can give insight on what it means to live with a disease, what symptoms are particularly burdensome, and which endpoints matter.
But, she said, “listening to the patient voice cannot mean that FDA just steps aside and lets anything on the market that patients are willing to try.” Individuals “who lack good treatment options have a very good reason to want to try things that haven’t yet been proven.”
If the FDA allows drugs on the market just because patients are willing to try, “5 or 10 years down the road, it’s not at all clear that we would end up with drugs that are better, or drugs that work, or drugs that we know anything more about,” said Dr. Lynch.
Does Taking Industry Money Equal Conflicts of Interest?
Many patient advocacy organizations receive funding from drug companies, medical device makers, or other industry sources, but they aren’t always transparent about how much or from which companies, according to studies.
The Alzheimer’s Association continued to push for the approval of aducanumab, even as the group received millions of dollars from the drugmakers. The association was accused of failing to disclose the potential conflict. It still lobbied for approval, even after the FDA advisers in 2020 voted against the drug.
It is not uncommon for individuals who speak in favor of a product’s approval to receive money for transportation and/or lodging from the drug’s manufacturer. In 2018, Dr. McCoy and colleagues reported in JAMA Internal Medicine that, between 2009 and 2017, a quarter of the speakers at the Anesthetic and Analgesic Drug Products Advisory Committee had conflicts of interest (COIs), mostly from industry, and that they were not disclosed in approximately 20% of the instances.
In a 2017 study of 104 large patient advocacy organizations published in The New England Journal of Medicine, Dr. McCoy and colleagues reported that 83% had received funds from industry. At least 39% had a current or former industry executive on the governing board, and 12% had a current or former industry executive in a board leadership position. Of the 104, 38 were focused on cancer and 13 on neurologic conditions. Of these, only 12% had published policies for managing institutional COIs.
Dr. McCoy emphasized the industry’s reliance on partnering with patient groups, particularly during FDA advisory committee meetings. “The sponsors wouldn’t be paying for patients to show up and give these testimonies if they didn’t think it made a difference. The audience isn’t just panel members; it’s also agency officials and maybe elected officials as well.”
“The Fox Foundation, with a $300 million-plus budget, gets about $5-$6 million a year from industry,” said Mr. Thompson. The money is earmarked for the organization’s Parkinson’s Disease Education Consortium; none goes toward advocacy. And, “the foundation has never specifically endorsed a product or device.”
When organizations that receive industry funding back a particular product, “it does appear to be [a conflict], and whether it is an actual one or not, appearances sometimes are all that matter,” said Mr. Thompson.
Dr. Lynch said accepting industry money “is a really significant conflict.” While advocates might need that money to fund advocacy efforts or make grants to advance research priorities, the acceptance might hinder willingness to demand evidence or to complain about a product’s price tag. “You don’t want to bite the hand that feeds you, right?”
Both Dr. McCoy and Dr. Lynch said patient groups — and individual patients — should at a minimum disclose industry funding, especially when speaking at an advisory committee.
Federal agencies and members of Congress actively seek patient input when considering legislation and funding priorities. But the individuals testifying at an advisory committee aren’t likely to represent all patients, and there’s a danger that they are just the loudest voices, said Dr. McCoy.
“We need to think more carefully about how we actually understand the preferences of a big, diverse patient population,” he said.
Dr. Lynch agreed.
Within the ALS community, “a lot of people who take different perspectives than some of those that are the leading voices get shouted down, and their voices get drowned out, and they get attacked on social media,” she said.
The group may be at the table, “but they’re just one voice at the table,” she said.
Dr. McCoy reported that his wife works for the Leukemia & Lymphoma Society, a patient advocacy organization. Dr. Rand reported no relevant financial relationships. Dr. Lynch received funding from Arnold Ventures and the Greenwall Foundation for work related to the FDA and patient advocacy.
A version of this article first appeared on Medscape.com.
Advocacy groups for patients with neurologic disorders have become a common feature in the landscape of drug and device development and federal research funding allocation.
On Capitol Hill, advocates have racked up some impressive legislative wins that aim to set a federal agenda for developing new medications.
At the Food and Drug Administration (FDA), advocacy groups played a significant role in several recent high-profile and controversial approvals for drugs for Alzheimer’s disease, Duchenne muscular dystrophy (DMD), and amyotrophic lateral sclerosis (ALS).
Such gains suggest these groups are growing in power. But with these wins come questions about whether large advocacy organizations — some of which receive significant industry funding — wield too much influence.
“You need to think very carefully about how you open these processes up to greater patient involvement,” Matthew S. McCoy, PhD, assistant professor of medical ethics and health policy at the University of Pennsylvania, Philadelphia, told this news organization. It’s important not to “end up with a situation where it’s the best-connected, the most well-resourced, the most-savvy patient organizations that are able to exercise outsize influence.”
Just because a group has deep pockets does not mean that its priorities align with the disease burden. And not every patient population is represented by a professionalized patient advocacy organization, Dr. McCoy noted. “There is the potential for the rich to get richer.”
A Seat at the Table
Long ago, the FDA and the National Institutes of Health (NIH) began giving patients a seat at the table, in part because of the path blazed by AIDS activists in the late 1980s and early 1990s, said Dr. McCoy.
Patient advocacy is often visible during FDA advisory committee meetings. The agency usually allows an hour, sometimes more, for members of the public to express support or concerns about the product being reviewed. Patients and caregivers — often aided by advocacy organizations — also submit hundreds, sometimes thousands, of letters before a product review.
The Alzheimer’s Association spent years advocating for approval of the anti-amyloid agent aducanumab (Aduhelm, Biogen/Eisai). In 2020, the organization urged patients and caregivers to submit written and oral testimony to the FDA advisory panel that was reviewing the drug. Despite patients’ pleas, the panel ultimately declined to support the drug’s approval, citing safety concerns and limited evidence of efficacy.
As controversy swirled around the medication — which had the potential for life-threatening brain swelling — advocates continued to apply pressure. Going against the expert panel’s recommendation, in June 2021, the FDA granted accelerated approval prompting three of the panelists to resign in protest.
Aducanumab’s initial price — $56,000 a year — was seen as a major threat to the viability of Medicare. Still, the Alzheimer’s Association stood behind the decision to approve the drug. But by early 2024, Biogen/Eisai said they would stop selling aducanumab, citing other priorities.
Once again patient advocates showed up in March 2022 when the FDA advisers were reviewing Amylyx Pharmaceuticals’ ALS drug Relyvrio (sodium phenylbutyrate and taurursodiol). Trials had showed limited efficacy, but patients testified they would accept greater risk for a chance to be treated with the drug. The committee ultimately voted against approval; 6 months later, the FDA approved Relyvrio anyway.
In April 2024, Amylyx removed Relyvrio from the market following phase 3 trial results that showed no difference between the treatment and placebo.
The drug manufacturer Sarepta Therapeutics, which develops treatments for genetic conditions such as DMD, has a history of working with — and funding — patient advocacy groups. The company encourages nonprofits to apply for grants or sponsorship on its website. At a 2016 advisory committee, when Sarepta was seeking approval of its first DMD therapy eteplirsen (Exondys 51), 52 speakers, most from patient advocacy groups, pleaded for the drug’s approval. When the panel voted no, Sarepta mobilized families to pressure the agency. Exondys was eventually approved.
In June, Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, unilaterally gave final expanded approval to Sarepta Therapeutics’ gene therapy Elevidys for DMD. Dr. Marks overrode his own FDA reviewers, who said the product lacked substantial evidence of efficacy. He acknowledged the drug had not met its primary endpoint but said he found secondary and exploratory endpoints “compelling” and cited an unmet medical need.
In an opinion piece in The Washington Post, Aaron Kesselheim, MD, JD, MPH, the director of the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, Boston, Massachusetts, and a former member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, questioned the approval stating that it undermined both public trust and manufacturers’ incentives to do the hard work of proving effectiveness.
Patient Voices the ‘Secret Sauce’
Drugmakers aren’t alone in seeing the value of having patients speak directly to government entities. When the Michael J. Fox Foundation wanted to gather cosponsors for the National Plan to End Parkinson’s Act, which President Joe Biden signed into law in July, it recruited and trained patients and caregivers for congressional meetings, said Ted Thompson, senior vice president of public policy at the foundation.
Having those individuals “making the personal case for how this disease affects their families ... was really the secret sauce,” in garnering a large number of cosponsors and getting legislation signed into law within 2 years of its introduction, Mr. Thompson told this news organization.
ALS advocacy groups launched a similar campaign to secure passage of the Accelerating Access to Critical Therapies for ALS Act in 2021.
Both pieces of legislation seek to set a federal agenda for developing new therapies in neurodegenerative diseases, in part by directing the FDA and NIH to fund research, engage patients more directly, and form public-private partnerships and councils to spur innovation.
But some said patient advocates are still coming far too late to the party.
“By the time you hear from patient groups at the meetings at the FDA, often the best opportunities for their input are long past,” Leah Zoe Gibson Rand, DPhil, a research scientist with the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, told this news organization. There should be more focus on the patient perspective earlier in drug development and trial design.
“There are some things that the patient voice could uniquely tell the agency,” said Holly Fernandez Lynch, JD, associate professor of medical ethics and health policy at the University of Pennsylvania. Patients can give insight on what it means to live with a disease, what symptoms are particularly burdensome, and which endpoints matter.
But, she said, “listening to the patient voice cannot mean that FDA just steps aside and lets anything on the market that patients are willing to try.” Individuals “who lack good treatment options have a very good reason to want to try things that haven’t yet been proven.”
If the FDA allows drugs on the market just because patients are willing to try, “5 or 10 years down the road, it’s not at all clear that we would end up with drugs that are better, or drugs that work, or drugs that we know anything more about,” said Dr. Lynch.
Does Taking Industry Money Equal Conflicts of Interest?
Many patient advocacy organizations receive funding from drug companies, medical device makers, or other industry sources, but they aren’t always transparent about how much or from which companies, according to studies.
The Alzheimer’s Association continued to push for the approval of aducanumab, even as the group received millions of dollars from the drugmakers. The association was accused of failing to disclose the potential conflict. It still lobbied for approval, even after the FDA advisers in 2020 voted against the drug.
It is not uncommon for individuals who speak in favor of a product’s approval to receive money for transportation and/or lodging from the drug’s manufacturer. In 2018, Dr. McCoy and colleagues reported in JAMA Internal Medicine that, between 2009 and 2017, a quarter of the speakers at the Anesthetic and Analgesic Drug Products Advisory Committee had conflicts of interest (COIs), mostly from industry, and that they were not disclosed in approximately 20% of the instances.
In a 2017 study of 104 large patient advocacy organizations published in The New England Journal of Medicine, Dr. McCoy and colleagues reported that 83% had received funds from industry. At least 39% had a current or former industry executive on the governing board, and 12% had a current or former industry executive in a board leadership position. Of the 104, 38 were focused on cancer and 13 on neurologic conditions. Of these, only 12% had published policies for managing institutional COIs.
Dr. McCoy emphasized the industry’s reliance on partnering with patient groups, particularly during FDA advisory committee meetings. “The sponsors wouldn’t be paying for patients to show up and give these testimonies if they didn’t think it made a difference. The audience isn’t just panel members; it’s also agency officials and maybe elected officials as well.”
“The Fox Foundation, with a $300 million-plus budget, gets about $5-$6 million a year from industry,” said Mr. Thompson. The money is earmarked for the organization’s Parkinson’s Disease Education Consortium; none goes toward advocacy. And, “the foundation has never specifically endorsed a product or device.”
When organizations that receive industry funding back a particular product, “it does appear to be [a conflict], and whether it is an actual one or not, appearances sometimes are all that matter,” said Mr. Thompson.
Dr. Lynch said accepting industry money “is a really significant conflict.” While advocates might need that money to fund advocacy efforts or make grants to advance research priorities, the acceptance might hinder willingness to demand evidence or to complain about a product’s price tag. “You don’t want to bite the hand that feeds you, right?”
Both Dr. McCoy and Dr. Lynch said patient groups — and individual patients — should at a minimum disclose industry funding, especially when speaking at an advisory committee.
Federal agencies and members of Congress actively seek patient input when considering legislation and funding priorities. But the individuals testifying at an advisory committee aren’t likely to represent all patients, and there’s a danger that they are just the loudest voices, said Dr. McCoy.
“We need to think more carefully about how we actually understand the preferences of a big, diverse patient population,” he said.
Dr. Lynch agreed.
Within the ALS community, “a lot of people who take different perspectives than some of those that are the leading voices get shouted down, and their voices get drowned out, and they get attacked on social media,” she said.
The group may be at the table, “but they’re just one voice at the table,” she said.
Dr. McCoy reported that his wife works for the Leukemia & Lymphoma Society, a patient advocacy organization. Dr. Rand reported no relevant financial relationships. Dr. Lynch received funding from Arnold Ventures and the Greenwall Foundation for work related to the FDA and patient advocacy.
A version of this article first appeared on Medscape.com.
ABIM Revokes Two Physicians’ Certifications Over Accusations of COVID Misinformation
The American Board of Internal Medicine (ABIM) has revoked certification for two physicians known for leading an organization that promotes ivermectin as a treatment for COVID-19.
Pierre Kory, MD, is no longer certified in critical care medicine, pulmonary disease, and internal medicine, according to the ABIM website. Paul Ellis Marik, MD, is no longer certified in critical care medicine or internal medicine.
Dr. Marik is the chief scientific officer and Dr. Kory is president emeritus of the Front Line COVID-19 Critical Care Alliance, a group they founded in March 2020. and also offers treatments for Lyme disease.
Ivermectin was proven to not be of use in treating COVID. Studies purporting to show a benefit were later linked to errors, and some were found to have been based on potentially fraudulent research.
The ABIM declined to comment when asked by this news organization about its action. Its website indicates that “revoked” indicates “loss of certification due to disciplinary action for which ABIM has determined that the conduct underlying the sanction does not warrant a defined pathway for restoration of certification at the time of disciplinary sanction.”
In a statement emailed to this news organization, Dr. Kory and Dr. Marik said, “we believe this decision represents a dangerous shift away from the foundation principles of medical discourse and scientific debate that have historically been the bedrock of medical education associations.”
The FLCCC said in the statement that it, along with Dr. Kory and Dr. Marik, are “evaluating options to challenge these decisions.”
Dr. Kory and Dr. Marik said they were notified in May 2022 that they were facing a potential ABIM disciplinary action. An ABIM committee recommended the revocation in July 2023, saying the two men were spreading “false or inaccurate medical information,” according to FLCCC. Dr. Kory and Dr. Marik lost an appeal.
In a 2023 statement, Dr. Kory and Dr. Marik called the ABIM action an “attack on freedom of speech.”
“This isn’t a free speech question,” said Arthur L. Caplan, PhD, the Drs. William F. and Virginia Connolly Mitty Professor of Bioethics at NYU Grossman School of Medicine’s Department of Population Health, New York City. “You do have the right to free speech, but you don’t have the right to practice outside of the standard of care boundaries,” he told this news organization.
The ABIM action “is the field standing up and saying, ‘These are the limits of what you can do,’” said Dr. Caplan. It means the profession is rejecting those “who are involved in things that harm patients or delay them getting accepted treatments,” he said. Caplan noted that a disciplinary action had been a long time in coming — 3 years since the first battles over ivermectin.
Wendy Parmet, JD, Matthews Distinguished University Professor of Law at Northeastern University School of Public Policy and Urban Affairs, Boston, said that misinformation spread by physicians is especially harmful because it comes with an air of credibility.
“We certainly want people to be able to dissent,” Ms. Parmet told this news organization. To engender trust, any sanctions by a professional board should be done in a deliberative process with a strong evidentiary base, she said.
“You want to leave sufficient room for discourse and discussion within the profession, and you don’t want the board to enforce a narrow, rigid orthodoxy,” she said. But in cases where people are “peddling information that is way outside the consensus” or are “profiting off of it, for the profession to take no action, that is, I think, detrimental also to the trust in the profession,” she said.
She was not surprised that Dr. Kory and Dr. Marik would fight to retain certification. “Board certification is an important, very worthwhile thing to have,” she said. “Losing it is not trivial.”
Dr. Kory, who is licensed in California, New York, and Wisconsin, “does not require this certification for his independent practice but is evaluating next steps with attorneys,” according to the statement from FLCCC.
Dr. Marik, whose Virginia medical license expired in 2022, “is no longer treating patients and has dedicated his time and efforts to the FLCCC Alliance,” the statement said.
Dr. Caplan served as a director, officer, partner, employee, advisor, consultant, or trustee for Johnson & Johnson’s Panel for Compassionate Drug Use (unpaid position) and is a contributing author and advisor for this news organization. Ms. Parmet reports no relevant financial relationships.
A version of this article appeared on Medscape.com.
The American Board of Internal Medicine (ABIM) has revoked certification for two physicians known for leading an organization that promotes ivermectin as a treatment for COVID-19.
Pierre Kory, MD, is no longer certified in critical care medicine, pulmonary disease, and internal medicine, according to the ABIM website. Paul Ellis Marik, MD, is no longer certified in critical care medicine or internal medicine.
Dr. Marik is the chief scientific officer and Dr. Kory is president emeritus of the Front Line COVID-19 Critical Care Alliance, a group they founded in March 2020. and also offers treatments for Lyme disease.
Ivermectin was proven to not be of use in treating COVID. Studies purporting to show a benefit were later linked to errors, and some were found to have been based on potentially fraudulent research.
The ABIM declined to comment when asked by this news organization about its action. Its website indicates that “revoked” indicates “loss of certification due to disciplinary action for which ABIM has determined that the conduct underlying the sanction does not warrant a defined pathway for restoration of certification at the time of disciplinary sanction.”
In a statement emailed to this news organization, Dr. Kory and Dr. Marik said, “we believe this decision represents a dangerous shift away from the foundation principles of medical discourse and scientific debate that have historically been the bedrock of medical education associations.”
The FLCCC said in the statement that it, along with Dr. Kory and Dr. Marik, are “evaluating options to challenge these decisions.”
Dr. Kory and Dr. Marik said they were notified in May 2022 that they were facing a potential ABIM disciplinary action. An ABIM committee recommended the revocation in July 2023, saying the two men were spreading “false or inaccurate medical information,” according to FLCCC. Dr. Kory and Dr. Marik lost an appeal.
In a 2023 statement, Dr. Kory and Dr. Marik called the ABIM action an “attack on freedom of speech.”
“This isn’t a free speech question,” said Arthur L. Caplan, PhD, the Drs. William F. and Virginia Connolly Mitty Professor of Bioethics at NYU Grossman School of Medicine’s Department of Population Health, New York City. “You do have the right to free speech, but you don’t have the right to practice outside of the standard of care boundaries,” he told this news organization.
The ABIM action “is the field standing up and saying, ‘These are the limits of what you can do,’” said Dr. Caplan. It means the profession is rejecting those “who are involved in things that harm patients or delay them getting accepted treatments,” he said. Caplan noted that a disciplinary action had been a long time in coming — 3 years since the first battles over ivermectin.
Wendy Parmet, JD, Matthews Distinguished University Professor of Law at Northeastern University School of Public Policy and Urban Affairs, Boston, said that misinformation spread by physicians is especially harmful because it comes with an air of credibility.
“We certainly want people to be able to dissent,” Ms. Parmet told this news organization. To engender trust, any sanctions by a professional board should be done in a deliberative process with a strong evidentiary base, she said.
“You want to leave sufficient room for discourse and discussion within the profession, and you don’t want the board to enforce a narrow, rigid orthodoxy,” she said. But in cases where people are “peddling information that is way outside the consensus” or are “profiting off of it, for the profession to take no action, that is, I think, detrimental also to the trust in the profession,” she said.
She was not surprised that Dr. Kory and Dr. Marik would fight to retain certification. “Board certification is an important, very worthwhile thing to have,” she said. “Losing it is not trivial.”
Dr. Kory, who is licensed in California, New York, and Wisconsin, “does not require this certification for his independent practice but is evaluating next steps with attorneys,” according to the statement from FLCCC.
Dr. Marik, whose Virginia medical license expired in 2022, “is no longer treating patients and has dedicated his time and efforts to the FLCCC Alliance,” the statement said.
Dr. Caplan served as a director, officer, partner, employee, advisor, consultant, or trustee for Johnson & Johnson’s Panel for Compassionate Drug Use (unpaid position) and is a contributing author and advisor for this news organization. Ms. Parmet reports no relevant financial relationships.
A version of this article appeared on Medscape.com.
The American Board of Internal Medicine (ABIM) has revoked certification for two physicians known for leading an organization that promotes ivermectin as a treatment for COVID-19.
Pierre Kory, MD, is no longer certified in critical care medicine, pulmonary disease, and internal medicine, according to the ABIM website. Paul Ellis Marik, MD, is no longer certified in critical care medicine or internal medicine.
Dr. Marik is the chief scientific officer and Dr. Kory is president emeritus of the Front Line COVID-19 Critical Care Alliance, a group they founded in March 2020. and also offers treatments for Lyme disease.
Ivermectin was proven to not be of use in treating COVID. Studies purporting to show a benefit were later linked to errors, and some were found to have been based on potentially fraudulent research.
The ABIM declined to comment when asked by this news organization about its action. Its website indicates that “revoked” indicates “loss of certification due to disciplinary action for which ABIM has determined that the conduct underlying the sanction does not warrant a defined pathway for restoration of certification at the time of disciplinary sanction.”
In a statement emailed to this news organization, Dr. Kory and Dr. Marik said, “we believe this decision represents a dangerous shift away from the foundation principles of medical discourse and scientific debate that have historically been the bedrock of medical education associations.”
The FLCCC said in the statement that it, along with Dr. Kory and Dr. Marik, are “evaluating options to challenge these decisions.”
Dr. Kory and Dr. Marik said they were notified in May 2022 that they were facing a potential ABIM disciplinary action. An ABIM committee recommended the revocation in July 2023, saying the two men were spreading “false or inaccurate medical information,” according to FLCCC. Dr. Kory and Dr. Marik lost an appeal.
In a 2023 statement, Dr. Kory and Dr. Marik called the ABIM action an “attack on freedom of speech.”
“This isn’t a free speech question,” said Arthur L. Caplan, PhD, the Drs. William F. and Virginia Connolly Mitty Professor of Bioethics at NYU Grossman School of Medicine’s Department of Population Health, New York City. “You do have the right to free speech, but you don’t have the right to practice outside of the standard of care boundaries,” he told this news organization.
The ABIM action “is the field standing up and saying, ‘These are the limits of what you can do,’” said Dr. Caplan. It means the profession is rejecting those “who are involved in things that harm patients or delay them getting accepted treatments,” he said. Caplan noted that a disciplinary action had been a long time in coming — 3 years since the first battles over ivermectin.
Wendy Parmet, JD, Matthews Distinguished University Professor of Law at Northeastern University School of Public Policy and Urban Affairs, Boston, said that misinformation spread by physicians is especially harmful because it comes with an air of credibility.
“We certainly want people to be able to dissent,” Ms. Parmet told this news organization. To engender trust, any sanctions by a professional board should be done in a deliberative process with a strong evidentiary base, she said.
“You want to leave sufficient room for discourse and discussion within the profession, and you don’t want the board to enforce a narrow, rigid orthodoxy,” she said. But in cases where people are “peddling information that is way outside the consensus” or are “profiting off of it, for the profession to take no action, that is, I think, detrimental also to the trust in the profession,” she said.
She was not surprised that Dr. Kory and Dr. Marik would fight to retain certification. “Board certification is an important, very worthwhile thing to have,” she said. “Losing it is not trivial.”
Dr. Kory, who is licensed in California, New York, and Wisconsin, “does not require this certification for his independent practice but is evaluating next steps with attorneys,” according to the statement from FLCCC.
Dr. Marik, whose Virginia medical license expired in 2022, “is no longer treating patients and has dedicated his time and efforts to the FLCCC Alliance,” the statement said.
Dr. Caplan served as a director, officer, partner, employee, advisor, consultant, or trustee for Johnson & Johnson’s Panel for Compassionate Drug Use (unpaid position) and is a contributing author and advisor for this news organization. Ms. Parmet reports no relevant financial relationships.
A version of this article appeared on Medscape.com.
New Guidance on Genetic Testing for Kidney Disease
A new consensus statement recommended genetic testing for all categories of kidney diseases whenever a genetic cause is suspected and offered guidance on who to test, which tests are the most useful, and how to talk to patients about results.
The statement, published online in the American Journal of Kidney Diseases, is the work of four dozen authors — including patients, nephrologists, experts in clinical and laboratory genetics, kidney pathology, genetic counseling, and ethics. The experts were brought together by the National Kidney Foundation (NKF) with the goal of broadening use and understanding of the tests.
About 10% or more of kidney diseases in adults and 70% of selected chronic kidney diseases (CKDs) in children have genetic causes. But nephrologists have reported a lack of education about genetic testing, and other barriers to wider use, including limited access to testing, cost, insurance coverage, and a small number of genetic counselors who are versed in kidney genetics.
Genetic testing “in the kidney field is a little less developed than in other fields,” said co–lead author Nora Franceschini, MD, MPH, a professor of epidemiology at the University of North Carolina Gillings School of Global Public Health, Chapel Hill, and a nephrologist who studies the genetic epidemiology of hypertension and kidney and cardiovascular diseases.
There are already many known variants that play a role in various kidney diseases and more are on the horizon, Dr. Franceschini told this news organization. More genetic tests will be available in the near future. “The workforce needs to be prepared,” she said.
The statement is an initial step that gets clinicians thinking about testing in a more systematic way, said Dr. Franceschini. “Genetic testing is just another test that physicians can use to complete the story when evaluating patients.
“I think clinicians are ready to implement” testing, said Dr. Franceschini. “We just need to have better guidance.”
Who, When, What to Test
The NKF statement is not the first to try to address gaps in use and knowledge. A European Renal Association Working Group published guidelines in 2022.
The NKF Working Group came up with 56 recommendations and separate algorithms to guide testing for adult and pediatric individuals who are considered at-risk (and currently asymptomatic) and for those who already have clinical disease.
Testing can help determine a cause if there’s an atypical clinical presentation, and it can help avoid biopsies, said the group. Tests can also guide choice of therapy.
For at-risk individuals, there are two broad situations in which testing might be considered: In family members of a patient who already has kidney disease and in potential kidney donors. But testing at-risk children younger than 18 years should only be done if there is an intervention available that could prevent, treat, or slow progression of disease, said the authors.
For patients with an established genetic diagnosis, at-risk family members should be tested with the known single-gene variant diagnostic instead of a broad panel, said the group.
Single-gene variant testing is most appropriate in situations when clinical disease is already evident or when there is known genetic disease in the family, according to the NKF panel. A large diagnostic panel that covers the many common genetic causes of kidney disease is recommended for the majority of patients.
The group recommended that apolipoprotein L1 (APOL1) testing should be included in gene panels for CKD, and it should be offered to any patient “with clinical findings suggestive of APOL1-association nephropathy, regardless of race and ethnicity.”
High-risk APOL1 genotypes confer a 5- to 10-fold increased risk for CKD and are found in one out of seven individuals of African ancestry, which means the focus has largely been on testing those with that ancestry.
However, with many unknowns about APOL1, the NKF panel did not want to “profile” individuals and suggest that testing should not be based on skin color or race/ethnicity, said Dr. Franceschini.
In addition, only about 10% of those with the variant develop disease, so testing is not currently warranted for those who do not already have kidney disease, said the group.
They also recommended against the use of polygenic risk scores, saying that there are not enough data from diverse populations in genome-wide association studies for kidney disease or on their clinical utility.
More Education Needed; Many Barriers
The authors acknowledged that nephrologists generally receive little education in genetics and lack support for interpreting and discussing results.
“Nephrologists should be provided with training and best practice resources to interpret genetic testing and discuss the results with individuals and their families,” they wrote, adding that there’s a need for genomic medicine boards at academic centers that would be available to help nephrologists interpret results and plot clinical management.
The group did not, however, cite some of the other barriers to adoption of testing, including a limited number of sites offering testing, cost, and lack of insurance coverage for the diagnostics.
Medicare may cover genetic testing for kidney disease when an individual has symptoms and there is a Food and Drug Administration–approved test. Joseph Vassalotti, MD, chief medical officer for the NKF, said private insurance may cover the testing if the nephrologist deems it medically necessary, but that he usually confirms coverage before initiating testing. The often-used Renasight panel, which tests for 385 genes related to kidney diseases, costs $300-$400 out of pocket, Dr. Vassalotti told this news organization.
In a survey of 149 nephrologists conducted in 2021, both users (46%) and nonusers of the tests (69%) said that high cost was the most significant perceived barrier to implementing widespread testing. A third of users and almost two thirds of nonusers said that poor availability or lack of ease of testing was the second most significant barrier.
Clinics that test for kidney genes “are largely confined to large academic centers and some specialty clinics,” said Dominic Raj, MD, the Bert B. Brooks chair, and Divya Shankaranarayanan, MD, director of the Kidney Precision Medicine Clinic, both at George Washington University School of Medicine & Health Sciences, Washington, DC, in an email.
Testing is also limited by cultural barriers, lack of genetic literacy, and patients’ concerns that a positive result could lead to a loss of health insurance coverage, said Dr. Raj and Dr. Shankaranarayanan.
Paper Will Help Expand Use
A lack of consensus has also held back expansion. The new statement “may lead to increased and possibly judicious utilization of genetic testing in nephrology practices,” said Dr. Raj and Dr. Shankaranarayanan. “Most importantly, the panel has given specific guidance as to what type of genetic test platform is likely to yield the best and most cost-effective yield.”
The most effective use is “in monogenic kidney diseases and to a lesser extent in oligogenic kidney disease,” said Dr. Raj and Dr. Shankaranarayanan, adding that testing is of less-certain utility in polygenic kidney diseases, “where complex genetic and epigenetic factors determine the phenotype.”
Genetic testing might be especially useful “in atypical clinical presentations” and can help clinicians avoid unnecessary expensive and extensive investigations when multiple organ systems are involved, they said.
“Most importantly, [testing] might prevent unnecessary and potentially harmful treatment and enable targeted specific treatment, when available,” said Dr. Raj and Dr. Shankaranarayanan.
Dr. Franceschini and Dr. Shankaranarayanan reported no relevant financial relationships. Dr. Raj disclosed that he received consulting fees and honoraria from Novo Nordisk and is a national leader for the company’s Zeus trial, studying whether ziltivekimab reduces the risk for cardiovascular events in cardiovascular disease, CKD, and inflammation. He also participated in a study of Natera’s Renasight, a 385-gene panel for kidney disease.
A version of this article first appeared on Medscape.com.
A new consensus statement recommended genetic testing for all categories of kidney diseases whenever a genetic cause is suspected and offered guidance on who to test, which tests are the most useful, and how to talk to patients about results.
The statement, published online in the American Journal of Kidney Diseases, is the work of four dozen authors — including patients, nephrologists, experts in clinical and laboratory genetics, kidney pathology, genetic counseling, and ethics. The experts were brought together by the National Kidney Foundation (NKF) with the goal of broadening use and understanding of the tests.
About 10% or more of kidney diseases in adults and 70% of selected chronic kidney diseases (CKDs) in children have genetic causes. But nephrologists have reported a lack of education about genetic testing, and other barriers to wider use, including limited access to testing, cost, insurance coverage, and a small number of genetic counselors who are versed in kidney genetics.
Genetic testing “in the kidney field is a little less developed than in other fields,” said co–lead author Nora Franceschini, MD, MPH, a professor of epidemiology at the University of North Carolina Gillings School of Global Public Health, Chapel Hill, and a nephrologist who studies the genetic epidemiology of hypertension and kidney and cardiovascular diseases.
There are already many known variants that play a role in various kidney diseases and more are on the horizon, Dr. Franceschini told this news organization. More genetic tests will be available in the near future. “The workforce needs to be prepared,” she said.
The statement is an initial step that gets clinicians thinking about testing in a more systematic way, said Dr. Franceschini. “Genetic testing is just another test that physicians can use to complete the story when evaluating patients.
“I think clinicians are ready to implement” testing, said Dr. Franceschini. “We just need to have better guidance.”
Who, When, What to Test
The NKF statement is not the first to try to address gaps in use and knowledge. A European Renal Association Working Group published guidelines in 2022.
The NKF Working Group came up with 56 recommendations and separate algorithms to guide testing for adult and pediatric individuals who are considered at-risk (and currently asymptomatic) and for those who already have clinical disease.
Testing can help determine a cause if there’s an atypical clinical presentation, and it can help avoid biopsies, said the group. Tests can also guide choice of therapy.
For at-risk individuals, there are two broad situations in which testing might be considered: In family members of a patient who already has kidney disease and in potential kidney donors. But testing at-risk children younger than 18 years should only be done if there is an intervention available that could prevent, treat, or slow progression of disease, said the authors.
For patients with an established genetic diagnosis, at-risk family members should be tested with the known single-gene variant diagnostic instead of a broad panel, said the group.
Single-gene variant testing is most appropriate in situations when clinical disease is already evident or when there is known genetic disease in the family, according to the NKF panel. A large diagnostic panel that covers the many common genetic causes of kidney disease is recommended for the majority of patients.
The group recommended that apolipoprotein L1 (APOL1) testing should be included in gene panels for CKD, and it should be offered to any patient “with clinical findings suggestive of APOL1-association nephropathy, regardless of race and ethnicity.”
High-risk APOL1 genotypes confer a 5- to 10-fold increased risk for CKD and are found in one out of seven individuals of African ancestry, which means the focus has largely been on testing those with that ancestry.
However, with many unknowns about APOL1, the NKF panel did not want to “profile” individuals and suggest that testing should not be based on skin color or race/ethnicity, said Dr. Franceschini.
In addition, only about 10% of those with the variant develop disease, so testing is not currently warranted for those who do not already have kidney disease, said the group.
They also recommended against the use of polygenic risk scores, saying that there are not enough data from diverse populations in genome-wide association studies for kidney disease or on their clinical utility.
More Education Needed; Many Barriers
The authors acknowledged that nephrologists generally receive little education in genetics and lack support for interpreting and discussing results.
“Nephrologists should be provided with training and best practice resources to interpret genetic testing and discuss the results with individuals and their families,” they wrote, adding that there’s a need for genomic medicine boards at academic centers that would be available to help nephrologists interpret results and plot clinical management.
The group did not, however, cite some of the other barriers to adoption of testing, including a limited number of sites offering testing, cost, and lack of insurance coverage for the diagnostics.
Medicare may cover genetic testing for kidney disease when an individual has symptoms and there is a Food and Drug Administration–approved test. Joseph Vassalotti, MD, chief medical officer for the NKF, said private insurance may cover the testing if the nephrologist deems it medically necessary, but that he usually confirms coverage before initiating testing. The often-used Renasight panel, which tests for 385 genes related to kidney diseases, costs $300-$400 out of pocket, Dr. Vassalotti told this news organization.
In a survey of 149 nephrologists conducted in 2021, both users (46%) and nonusers of the tests (69%) said that high cost was the most significant perceived barrier to implementing widespread testing. A third of users and almost two thirds of nonusers said that poor availability or lack of ease of testing was the second most significant barrier.
Clinics that test for kidney genes “are largely confined to large academic centers and some specialty clinics,” said Dominic Raj, MD, the Bert B. Brooks chair, and Divya Shankaranarayanan, MD, director of the Kidney Precision Medicine Clinic, both at George Washington University School of Medicine & Health Sciences, Washington, DC, in an email.
Testing is also limited by cultural barriers, lack of genetic literacy, and patients’ concerns that a positive result could lead to a loss of health insurance coverage, said Dr. Raj and Dr. Shankaranarayanan.
Paper Will Help Expand Use
A lack of consensus has also held back expansion. The new statement “may lead to increased and possibly judicious utilization of genetic testing in nephrology practices,” said Dr. Raj and Dr. Shankaranarayanan. “Most importantly, the panel has given specific guidance as to what type of genetic test platform is likely to yield the best and most cost-effective yield.”
The most effective use is “in monogenic kidney diseases and to a lesser extent in oligogenic kidney disease,” said Dr. Raj and Dr. Shankaranarayanan, adding that testing is of less-certain utility in polygenic kidney diseases, “where complex genetic and epigenetic factors determine the phenotype.”
Genetic testing might be especially useful “in atypical clinical presentations” and can help clinicians avoid unnecessary expensive and extensive investigations when multiple organ systems are involved, they said.
“Most importantly, [testing] might prevent unnecessary and potentially harmful treatment and enable targeted specific treatment, when available,” said Dr. Raj and Dr. Shankaranarayanan.
Dr. Franceschini and Dr. Shankaranarayanan reported no relevant financial relationships. Dr. Raj disclosed that he received consulting fees and honoraria from Novo Nordisk and is a national leader for the company’s Zeus trial, studying whether ziltivekimab reduces the risk for cardiovascular events in cardiovascular disease, CKD, and inflammation. He also participated in a study of Natera’s Renasight, a 385-gene panel for kidney disease.
A version of this article first appeared on Medscape.com.
A new consensus statement recommended genetic testing for all categories of kidney diseases whenever a genetic cause is suspected and offered guidance on who to test, which tests are the most useful, and how to talk to patients about results.
The statement, published online in the American Journal of Kidney Diseases, is the work of four dozen authors — including patients, nephrologists, experts in clinical and laboratory genetics, kidney pathology, genetic counseling, and ethics. The experts were brought together by the National Kidney Foundation (NKF) with the goal of broadening use and understanding of the tests.
About 10% or more of kidney diseases in adults and 70% of selected chronic kidney diseases (CKDs) in children have genetic causes. But nephrologists have reported a lack of education about genetic testing, and other barriers to wider use, including limited access to testing, cost, insurance coverage, and a small number of genetic counselors who are versed in kidney genetics.
Genetic testing “in the kidney field is a little less developed than in other fields,” said co–lead author Nora Franceschini, MD, MPH, a professor of epidemiology at the University of North Carolina Gillings School of Global Public Health, Chapel Hill, and a nephrologist who studies the genetic epidemiology of hypertension and kidney and cardiovascular diseases.
There are already many known variants that play a role in various kidney diseases and more are on the horizon, Dr. Franceschini told this news organization. More genetic tests will be available in the near future. “The workforce needs to be prepared,” she said.
The statement is an initial step that gets clinicians thinking about testing in a more systematic way, said Dr. Franceschini. “Genetic testing is just another test that physicians can use to complete the story when evaluating patients.
“I think clinicians are ready to implement” testing, said Dr. Franceschini. “We just need to have better guidance.”
Who, When, What to Test
The NKF statement is not the first to try to address gaps in use and knowledge. A European Renal Association Working Group published guidelines in 2022.
The NKF Working Group came up with 56 recommendations and separate algorithms to guide testing for adult and pediatric individuals who are considered at-risk (and currently asymptomatic) and for those who already have clinical disease.
Testing can help determine a cause if there’s an atypical clinical presentation, and it can help avoid biopsies, said the group. Tests can also guide choice of therapy.
For at-risk individuals, there are two broad situations in which testing might be considered: In family members of a patient who already has kidney disease and in potential kidney donors. But testing at-risk children younger than 18 years should only be done if there is an intervention available that could prevent, treat, or slow progression of disease, said the authors.
For patients with an established genetic diagnosis, at-risk family members should be tested with the known single-gene variant diagnostic instead of a broad panel, said the group.
Single-gene variant testing is most appropriate in situations when clinical disease is already evident or when there is known genetic disease in the family, according to the NKF panel. A large diagnostic panel that covers the many common genetic causes of kidney disease is recommended for the majority of patients.
The group recommended that apolipoprotein L1 (APOL1) testing should be included in gene panels for CKD, and it should be offered to any patient “with clinical findings suggestive of APOL1-association nephropathy, regardless of race and ethnicity.”
High-risk APOL1 genotypes confer a 5- to 10-fold increased risk for CKD and are found in one out of seven individuals of African ancestry, which means the focus has largely been on testing those with that ancestry.
However, with many unknowns about APOL1, the NKF panel did not want to “profile” individuals and suggest that testing should not be based on skin color or race/ethnicity, said Dr. Franceschini.
In addition, only about 10% of those with the variant develop disease, so testing is not currently warranted for those who do not already have kidney disease, said the group.
They also recommended against the use of polygenic risk scores, saying that there are not enough data from diverse populations in genome-wide association studies for kidney disease or on their clinical utility.
More Education Needed; Many Barriers
The authors acknowledged that nephrologists generally receive little education in genetics and lack support for interpreting and discussing results.
“Nephrologists should be provided with training and best practice resources to interpret genetic testing and discuss the results with individuals and their families,” they wrote, adding that there’s a need for genomic medicine boards at academic centers that would be available to help nephrologists interpret results and plot clinical management.
The group did not, however, cite some of the other barriers to adoption of testing, including a limited number of sites offering testing, cost, and lack of insurance coverage for the diagnostics.
Medicare may cover genetic testing for kidney disease when an individual has symptoms and there is a Food and Drug Administration–approved test. Joseph Vassalotti, MD, chief medical officer for the NKF, said private insurance may cover the testing if the nephrologist deems it medically necessary, but that he usually confirms coverage before initiating testing. The often-used Renasight panel, which tests for 385 genes related to kidney diseases, costs $300-$400 out of pocket, Dr. Vassalotti told this news organization.
In a survey of 149 nephrologists conducted in 2021, both users (46%) and nonusers of the tests (69%) said that high cost was the most significant perceived barrier to implementing widespread testing. A third of users and almost two thirds of nonusers said that poor availability or lack of ease of testing was the second most significant barrier.
Clinics that test for kidney genes “are largely confined to large academic centers and some specialty clinics,” said Dominic Raj, MD, the Bert B. Brooks chair, and Divya Shankaranarayanan, MD, director of the Kidney Precision Medicine Clinic, both at George Washington University School of Medicine & Health Sciences, Washington, DC, in an email.
Testing is also limited by cultural barriers, lack of genetic literacy, and patients’ concerns that a positive result could lead to a loss of health insurance coverage, said Dr. Raj and Dr. Shankaranarayanan.
Paper Will Help Expand Use
A lack of consensus has also held back expansion. The new statement “may lead to increased and possibly judicious utilization of genetic testing in nephrology practices,” said Dr. Raj and Dr. Shankaranarayanan. “Most importantly, the panel has given specific guidance as to what type of genetic test platform is likely to yield the best and most cost-effective yield.”
The most effective use is “in monogenic kidney diseases and to a lesser extent in oligogenic kidney disease,” said Dr. Raj and Dr. Shankaranarayanan, adding that testing is of less-certain utility in polygenic kidney diseases, “where complex genetic and epigenetic factors determine the phenotype.”
Genetic testing might be especially useful “in atypical clinical presentations” and can help clinicians avoid unnecessary expensive and extensive investigations when multiple organ systems are involved, they said.
“Most importantly, [testing] might prevent unnecessary and potentially harmful treatment and enable targeted specific treatment, when available,” said Dr. Raj and Dr. Shankaranarayanan.
Dr. Franceschini and Dr. Shankaranarayanan reported no relevant financial relationships. Dr. Raj disclosed that he received consulting fees and honoraria from Novo Nordisk and is a national leader for the company’s Zeus trial, studying whether ziltivekimab reduces the risk for cardiovascular events in cardiovascular disease, CKD, and inflammation. He also participated in a study of Natera’s Renasight, a 385-gene panel for kidney disease.
A version of this article first appeared on Medscape.com.
FROM THE AMERICAN JOURNAL OF KIDNEY DISEASES