It’s time for the Trump administration to put back in place the safety regulations on mifepristone without delay
U.S. Food and Drug Administration (FDA) Commissioner Marty Makary recently told a D.C. confab that he has “no plans to take action” on mifepristone, the chemical abortion drug responsible for more than 70 percent of abortions in the United States
That’s a decidedly different answer than the one he gave me under oath
Makary pledged “to review the totality of data” with respect to mifepristone
He also said that if “the data suggests something or tells us that there’s a real signal
we can’t promise we’re not going to act on that data.”
And it’s sending a signal that can’t be missed: Mifepristone is not safe
Last Monday, the Ethics and Public Policy Center published a massive, in-depth report examining more than 865,000 cases of prescribed mifepristone abortions between 2017 and 2023
The data comes from insurance claims records and constitutes the largest study on mifepristone ever undertaken
Nearly 11 percent of women who use the drug to induce an abortion suffer an “adverse health event” — as in a major
potentially life-threatening medical disaster
The kind of things that could lead to death
And the data reveals this happens to more than 1 in 10 women taking mifepristone
and they have been largely hidden from the public
The rate of serious health events revealed by the EPPC study is some 22 times greater than the numbers reported on the FDA-approved drug label
rely on clinical trials more than four decades old
All of which means this: It’s time for the Trump administration to put back in place the safety regulations on mifepristone without delay
The federal government once acknowledged the serious risks of this dangerous drug
the government imposed a host of safety measures
Those included prescription by physician only
a requirement that physicians dispense the drug
and even a “black box” warning alerting women to the drug’s many dangers
And all this was done by the Clinton administration
launched a campaign to roll back the safeguards
President Obama reduced the number of required in-person visits and removed the physician-prescription requirement
in an attempt to mask the drug’s true risks
his administration ended the mandatory adverse-event reporting
meaning doctors no longer had to admit when the drug sent their patients to the hospital
The Biden administration eliminated in-person checkups entirely and even did away with the long-standing mandate that physicians dispense the drug in person
So mifepristone could be sent through the mail
they have “revolutionized abortion access in the U.S.” in a manner pro-lifers “will likely not be able to stop.”
Except we can stop this abortion-on-demand bonanza
reinstate the full complement of mifepristone safety regulations immediately
Only doctors should be able to prescribe the drug
Only doctors should be able to dispense and administer it
And there should be mandatory safety checkups afterward
Congress should make all of these rules permanent by writing them into law
The new data speaks for itself: Mifepristone is a remarkably dangerous drug that threatens the lives of both baby and mother
Those who have pretended otherwise have lied to us
Now is the time to set the record straight and protect the lives of millions of Americans
A wholly independent division of FDRLST Media
Food and Drug Administration Commissioner Martin Makary said Thursday he has “no plans to take action” to restrict the availability of mifepristone
the pill used in most medication abortions in the US
Speaking at the Semafor World Economy Summit
Makary took a firmer stance on the issue than he did in his Senate confirmation hearing last month
when he said he would “do a review of the data” and didn’t commit to specific action — leading some to wonder whether the Donald Trump administration would ultimately move to roll back access to the pill
Makary still left the door open to future action on the medication
“There is an ongoing set of data that is coming into the FDA on mifepristone,” he said
“So if the data suggests something or tells us that there’s a real signal
The FDA considers mifepristone safe and effective
After the US Supreme Court overturned Roe v
some Republicans have moved to crack down on the pill
which some people have accessed through telemedicine even in states with abortion bans
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By Lizzy Lawrence and Sarah Todd
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Lizzy Lawrence leads STAT’s coverage of the Food and Drug Administration
She was previously a medical devices reporter
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Sarah Todd returned to reporting in January 2025 after being assignment editor at STAT since October 2022
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His first email to staff summarized his resume
“A national leader in medicine with impeccable credentials
Makary is imminently qualified to lead the FDA at this important time in the health of our nation,” the email
who will need to earn trust from employees in order to achieve health secretary Robert F
Kennedy Jr.’s lofty goal of tackling the root causes of chronic disease to make the nation healthier
The vast number of open leadership roles also gives Makary and Kennedy the opportunity to remake the FDA as they see fit
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WASHINGTON — The Food and Drug Administration will remove industry representatives from advisory committees and replace them with patients and caregivers
Commissioner Marty Makary announced Thursday
It’s the latest effort by Makary and his boss
to reduce the influence of the pharmaceutical industry
Makary announced the change during his first interview as commissioner
He spent much of the conversation with Kelly lambasting his agency for its ties to industry
and said he was shocked to learn that drug industry representatives are allowed to sit on FDA advisory committees
but may mean the administration stops actively making things worse
a Norstella company Pharma Intelligence UK Limited is a company registered in England and Wales with company number 13787459 whose registered
Screen shot from Marty Makary’s first media interview as FDA commissioner
The Megyn Kelly Show
BioSpace strives for finely balanced reporting
equally considering the many voices currently weighing in on our fractured political reality—within which the biopharma space is caught squarely in the crosshairs
we sought clarification on a few key points made by the new commissioner and unpacked the particulars of others
Statement: “All the [FDA workforce] cuts were limited to communications staff
There were no layoffs to scientists or food inspectors.”
There is no debate over the massive layoffs made to the communications teams across the Department of Health and Human Services
including all of the employees at the FDA’s main communications office
but there have been reports of culls beyond those staffers
Some of these non-communications staff were given the option for reassignment
adding that FOIA staff and travel arrangers who helped inspectors travel overseas were also let go
“I declined the offer (as ridiculous) so am on administrative leave,” Stein told the publication
Center for Biologics Evaluation and Research Director Peter Marks was also reportedly forced to choose between retirement and being fired
Even if no direct cuts were made to scientists and inspectors, as Makary claimed, it’s misleading to suggest that this wouldn’t affect the overall operations at FDA, Genevieve Kanter, an associate professor of public policy at the University of Southern California Price School of Public Policy, told BioSpace earlier this month
She likened the layoffs of FDA support staff to a clinic that keeps only the doctors
“The idea that FDA reviews won’t be adversely affected after such a draconian staffing cut is like firing all the staff at a clinic and saying that patient care won’t be affected because we haven’t fired the doctors,” she said
Statement: “I don’t think VAERS [the Vaccines Adverse Event Reporting System] is a good database because it is self-reported and not rates.”
Jefferies’ Andrew Tsai agreed with Makary’s comments
we don’t really give too much credence on self-reported data,” he told BioSpace
Notably, VAERS was at the center of Marks’ departure
Marks said after he left the agency that he had denied Kennedy’s team access to the system
“Why wouldn’t we [grant access]? Because frankly we don’t trust [them],” Marks said, according to the AP
“They’d write over it or erase the whole database.”
Makary said the former CBER chief “resigned before I came to office.”
adding that “Makary was briefed on the plan to oust Marks.” Pink Sheet cited “multiple sources,” including a former agency official
Per an FDA news release, Makary was confirmed as commissioner by the U.S
Senate on March 25 and had taken the oath of office as of April 1
BioSpace has reached out to HHS for further details on this timing
Statement: “We are removing industry members
Coinciding with this interview, on April 17, the FDA announced a policy directive limiting pharmaceutical company employees from serving as members on its independent advisory committees
The possibility of conflicts of interest—or so-called “regulatory capture” of the FDA by the pharma industry—has been debated for decades and was alluded to by Kennedy at a recent visit to the FDA’s White Oaks, Maryland campus where he called staffers the industry’s “sock puppet.”
This directive would appear to be the administration’s opening salvo toward addressing this issue. However, experts who spoke with BioSpace this week said the impact of this initiative would be minimal
This is partly because of the words “statutorily possible.” A provision in the Food and Drug Administration Modernization Act of 1997 “has been interpreted to mean that newly created advisory committees must include an industry representative,” Kanter told BioSpace in an email
Kanter suspects that RFK and Makary will only be able to remove industry representatives from adcomms established prior to this law
Statement: Makary said President Donald Trump and HHS Secretary Robert F
are the two most popular politicians in America right now
According to multiple outlets, Trump’s approval ratings are on the decline. A Pew survey conducted between April 7 and 13 revealed that 59% of 3,589 respondents disapprove of Trump’s job performance
Citing the same survey, Forbes reported last week that amid ever-changing tariff policies
Trump has “the worst economic reviews in years,” at 37% favorable to 51% unfavorable
Meanwhile, an informal poll conducted by BioSpace in the wake of Kennedy’s massive job cuts at HHS showed that 82% of 804 respondents believed he himself should get the axe
BioSpace has reached out to HHS to obtain the polling data mentioned by Makary
Statement: When asked by Kelly about reports that some people walked out of Kennedy’s recent speech to FDA employees
It was during this speech that Kennedy accused FDA staffers of being a “sock puppet” to the pharma industry. According to reporting by Politico
“several FDA staffers had walked out of the rooms where the speech was being broadcast at the agency’s headquarters in White Oak
an FDA insider told BioSpace that they went to the ceremony for “about 15 minutes” before leaving
Statement: When asked by Kelly about the causes of autism
I think it is the cumulative burden of all of these exposures
Kennedy has said that he will find a cause for autism by September, tapping discredited scientist David Geier to lead the research effort. The Secretary has long touted a link between autism and vaccines—a connection that has repeatedly been debunked
“More than 100 genes on different chromosomes may be involved in causing ASD, to different degrees,” according to the National Institutes of Health
If someone is susceptible to ASD because of genetic mutations
“an infection or contact with chemicals in the environment could cause autism in someone who is susceptible because of genetic mutations,” the agency states
someone who is genetically susceptible might not get an ASD even if he or she has the same experiences.”
The FDA plans to consolidate some administrative functions
but won’t embark on a wholesale reorganization of the review centers that oversee drugs
biologics and medical …
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A leaked document revealed details about changes that could impact how the agency operates
but recent comments from FDA Commissioner Dr
Job cuts were just the first disruption at health agencies
questions remain about how the FDA will be reshaped after commissioner Dr
Marty Makary “rejected” a proposed restructuring that was leaked this month
The proposal would have upended the agency’s organizational structure and created five new shared services offices rather than current product-based offices
But Makary shot down some of those rumors about the scale of the reorganization
“There will not be a reorganization,” Makary said yesterday in an interview with MedPage Today. “Yes, we are consolidating travel offices
IT and some other things that make sense for efficiency
A proposal for a reorganization came from some staff
I said that we will work within the existing structure.”
But without any official reordering plan from the administration
Makary’s latest comments add to the uncertainty of an already frayed situation
The health agencies are already hurting after a massive workforce reduction announced in April slashed 10,000 jobs across HHS, including 3,500 at the FDA, and has already impacted the flow of agency work. The overhaul was part of HHS’ stated effort to save $1.8 billion per year and “streamline” agency divisions from 28 to 15
Some pharma and biotech companies have experienced delays in the aftermath
with the FDA pushing back decisions on clinical trials
The Trump administration was targeting fiscal year 2026
to implement the proposed FDA restructuring plan
And while Makary’s latest comments may have quelled some fears
the leaked plan piled onto mounting concerns about other top-down changes
the reorganization of the agency is likely to have a long-term impact
“This would be a radical and profound change,” Laura DiAngelo, senior director of research at AgencyIQ, said during a recent webinar on the restructuring
“It takes a long time to understand where everyone sits
what everyone's workstreams look like when under a new reporting system.”
One of the biggest changes in the proposed structure brought all product review divisions into one. The FDA underwent the “single largest reorganization in the agency’s recent history” last year
the FDA brought reviews and inspections under one office
regulatory policy and intelligence at AgencyIQ
The new plan aimed to once again separate those offices, potentially undoing any efficiencies gained when the FDA made changes last year based on the recommendations from an independent expert panel
“The entire reorganization of the human foods program … was done purely because they [said] we need inspectors and reviewers in the same office so that they can talk to each other
because it doesn’t make sense to split that up,” Gaffney said
Congress could object to the restructuring efforts
“It would not surprise me if Congress takes a look at this and goes
we actually know a lot about why this would not be a good idea because we just reorganized stuff based on the fact that this approach … that’s being raised here is a bad idea
specifically with reviews and inspections being in separate offices,” Gaffney said
The proposal to create five offices also took some heat from former FDA officials, including Janet Woodcock, a long-time FDA leader, who called the idea “a mindless approach” and slammed the attempt at centralization
But the biggest impact to the FDA’s operations may be the current workforce reductions combined with a draft budget proposal that would slash nearly 20% of the FDA’s budget as part of a plan to cut one-third of HHS’ coffers overall
What eventually gets approved by Congress for the 2026 fiscal year could look substantially different from the leaked proposal
Makary’s comments come after swift backlash to the healthcare agency changes
Meanwhile, some legal experts have argued that the workforce reduction and how it was executed might be illegal
Others are sounding the alarm about the lack of HHS communication, despite Kennedy’s stated commitment to “radical transparency.”. Three former FDA leaders, including Lurie, took to The Washington Post to voice worries that Kennedy’s actions are making the agency’s moves more opaque — a shift that started with a freeze on HHS communications after President Donald Trump took office
Staff from the communications departments at the FDA
“With so much obfuscation and opacity at HHS
it has become crystal clear that ‘radical transparency’ is not about openness and engagement,” they wrote
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WASHINGTON — The commissioner of the Food and Drug Administration welcomed three new leaders to the agency this week
Following massive layoffs and cuts at the FDA
Makary’s plan for the agency is yet to fully unfold
But he has hinted at what his approach might look like
A month into his new role as commissioner of the Food and Drug Administration (FDA)
Marty Makary’s approach to the agency is becoming a bit clearer
While Makary has hinted at staying true to scientific standards in his first comments as commissioner
the agency is still reeling from mass layoffs and structural changes that could jeopardize how it has always conducted its regulatory work
The FDA has historically been a global leader in approving new drugs and defining regulatory standards. It also approves more new drugs than other countries’ agencies, according to Eunjoo Pacifici
chair and associate professor at the Department of Regulatory and Quality Sciences at USC Mann
The 20% reduction in staff size and changes in recent weeks could lead to an FDA that is slower at pushing new drugs into the market
then we will have to follow — and who would we follow?” Pacifici said
“We might end up relinquishing our role as the leader of the regulatory community that everybody looks to — and that impacts the entire innovation cycle.”
Other experts were cautious to identify clear-cut impacts
and instead emphasized expectations of how the FDA operates should be fluid
“There’s no doubt that this is a situation that will constantly evolve,” noted Matthew Weinberg
president of regulatory sciences at consultancy ProPharma Group
“One of the things we know from Trump’s first presidency is that things happen constantly
so we’re going to live in a world of constant change.”
But there are ways for pharma companies to manage the tumult occurring both on the regulatory front and in looming tariffs
who has been in the business for 40 years and has seen 22 different FDA commissioners during that time
believes a commitment to “the pursuit of good science” will be the key to staying afloat amid choppy waters
“That should be a theme throughout all of this for companies to stick to,” Weinberg said
Since Makary stepped into his role as FDA commissioner
he announced three new hires to the agency — Lowell Zeta as deputy commissioner for strategic initiatives
Craig Taylor as acting chief information officer
and Timothy Schell as acting director for the Center for Veterinary Medicine
The slew of hires comes after numerous FDA officials departed the agency this month in the wake of the administration changes
including former director of the Center for Biologics Evaluation and Research (CBER) Dr
Makary’s plan for the agency — and the extent to which he’ll be influenced by HHS Secretary Robert F
But he did hint at some of his plans in an interview with former Fox News journalist Megyn Kelly last week
Makary proposed rolling out a new pathway for rare disease approvals that could get drugs into the hands of patients faster
If there’s a new treatment for a rare disease that makes sense physiologically — meaning the mechanism is scientifically plausible — the proposed pathway would allow patients to get on the treatment if they wanted to
even in the absence of a randomized controlled trial
“If there’s a rare condition or a condition that’s incurable that affects a small number of people
we might be approving drugs based on a plausible mechanism,” Makary said
“We would allow that and at the same time monitor everyone who gets it
so we can make inferences as soon as [there’s] a signal in the data.”
The proposal for a “plausible mechanism” pathway would require even less pre-approval data than the FDA’s current accelerated approval pathway
It would also require the agency to walk a fine line between speeding up potentially life-saving drug approvals for rare disease patients — while protecting those patients from unsafe or ineffective drugs
Makary also touched on the burgeoning anti-vaccine efforts taking shape under Kennedy’s helm
noting that “there’s been an epidemic of distrust [in the FDA] and part of it is warranted.”
In response to a question posed by Kelly about negative side effects from the COVID-19 vaccine
Makary claimed the FDA will conduct “intense
comprehensive research” on the safety and efficacy of vaccines and emphasized the use of real-world data in doing so
“If we have massive electronic health record data — which we now have from the Health Information Exchange — we can have researchers go in there and look at real-world complication rates,” Makary said
we’re not relying on self-reported data
from which you can make no inferences about rates
That’s a basic scientific methodological principle.”
He also said that Marks’ former position as the agency’s top vaccine regulator will be filled
and that a lot of “talented people” were applying for the job
Finally, Makary announced last week that the FDA would implement a new policy that would prevent individuals employed at pharma companies from serving as official members of FDA advisory committees
all in the name of boosting transparency and reducing conflicts of interest
“While the FDA should be partnering with industry to ensure a user-friendly review process
the scientific evaluation of new products should be independent,” Makary said in a statement
Taken altogether, Makary’s moves appeared to be a mixed bag but still somewhat reassuring to the pharma industry, with The Wall Street Journal calling them a “bullish signal to biotech.”
Biotech stocks rose on Monday following his interview with Kelly
with analysts at RBC Capital Markets noting that Makary’s comments generally appeared to be “scientifically grounded and level-headed.”
Makary’s emphasis on the use of real-world data could also potentially placate political concerns from Kennedy and Trump around vaccines without upending the FDA’s scientific processes
we believe he exhibited an adeptness at packaging them to align with administration values without appearing inclined to tear down and remake the agency,” RBC Capital Markets analyst Brian Abrahams wrote
much will depend on who is chosen to replace Marks as head of the CBER
“We can only hope (not something that we say frequently) Dr
Makary appoints a science-driven professional to lead CBER
and thus the review and approval of biological products (including the ever critical vaccines),” he wrote
other therapeutic areas and FDA functions could see changes or a slowdown of regulations and guidance
Scott Gottlieb pointed to cell and gene therapy approvals — as well as generic drugs — as being most at risk given the massive funding cuts and layoffs at the HHS
the Division of Policy Development — which sits within the larger Office of Generic Drug Policy (OGDP) and develops guidance for generic drug approvals — was eliminated
That means FDA approvals of generics could decrease
resulting in higher drug costs for consumers
“That [generics division] has been cut pretty close to the bone,” Weinberg said
“I think it’s fair to say the guidance that the generic industry used to get — which would give them some idea of how to develop their products — will be of a vastly lower degree than it used to be
unless the Secretary [decides] to add some things back.”
Gottlieb also emphasized the importance of OGDP
noting that during his time as commissioner
it was responsible for bringing the first generic EpiPen to market
OGDP staff were tasked with finding ways to genericize a drug
device or delivery system when there were patent protections around it — and then issued good review management practices
“Without that [policy formulation work]
you can’t make generic approvals,” Gottlieb noted
“That is going to have a tangible effect on both new technology but also novel generic approvals
Trying to genericize a complex formulation where there’s no precedent — that’s where they’re going to have their hands tied now.”
Plenty of other therapeutic areas — spanning from cancer to neurodegenerative diseases like Alzheimer’s — could see a slowdown in getting new therapies approved with fewer staff
She also pointed to the growing implementation of AI and machine learning (ML) in drugs and medical devices — something that the FDA had just begun figuring out how to regulate
“We’re at an inflection point where technology and scientific advancements could change the face of therapeutics,” Pacifici said
“This is not the time to take your foot off the pedal.”
pharma experts expressed concerns around communications and transparency at the agency
with many guidance documents removed from the site
Public-facing activities at the FDA are important
because they provide transparency and predictability into a process in which pharma companies are spending upwards of hundreds of millions of dollars to develop a product
While Makary’s initial comments inspired some positive sentiment among industry players and analysts
a large degree of uncertainty will likely remain for the foreseeable future
Weinberg noted many of his pharma clients have expressed concerns about all of the uncertainty
“They want to move life-saving therapies through the process as fast as possible — and they want to find out if they can monetize those as necessary,” Weinberg said
Pacifici encouraged pharma companies to invest in their public policy engagement activities in order to ensure policymakers “hear them and understand the implications of the regulatory reduction in force.”
But there’s also plenty pharma companies can do internally to manage tumult
is to avoid knee-jerk reactions to the daily policy changes — from layoffs to tariffs — and instead take a step back to figure out a plan that’s adaptable
“Think of yourselves on a ship in the middle of [a storm] or on an airplane during turbulence,” Weinberg said
“You want to go with a captain who’s [calm and says]
Let’s just take a breath and we can navigate our way through this.’ I think the companies that do that will persevere.”
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a Johns Hopkins surgeon who is poised to become the next commissioner of the Food and Drug Administration
told senators Thursday that he will stick to the science
Makary said he wanted to help restore America's trust in the FDA and the products it regulates
"If we have the cure for pancreatic cancer
but only 40% of the public is going to come in and take it because the rest don't trust us
then that medication is only 40% effective," he said
"So I think we fight bad ideas with more ideas
I don't … believe in censorship of scientific opinions
When pressed on several controversial topics
such as access to the abortion pill and firings at the FDA
he didn't answer directly and said instead he would "do an assessment" after becoming FDA commissioner
Here are five things that came up during the confirmation hearing
Multiple senators asked Makary whether he would maintain current access to mifepristone
a pill used in medication abortions and miscarriage management up to 10 weeks gestation
the Louisiana Republican who chairs the HELP committee
asked whether Makary would end telemedicine access to the drug
a research organization that supports access to abortion
"I have no preconceived plans on mifepristone policy except to take a solid
hard look at the data and to meet with the professional career scientists who have reviewed the data at the FDA
and to build an expert coalition to review the ongoing data which is required to be collected as a part of the REMS program," Makary said
REMS stands for "Risk Evaluation and Mitigation Strategy." The FDA adds REMS programs to some drug approvals
and they can limit how drugs are prescribed and distributed
Makary said he would look at the data before making any decisions
He wouldn't commit either way to limiting or maintaining access to mifepristone
I wish you were hedging a little bit less today," said Sen
Senators also asked Makary what he would do about the recent cancellation of a vaccine advisory committee meeting that was scheduled to consider next season's flu shot
FDA advisory committees are made up of experts outside the agency
They publicly deliberate and vote on various questions of interest to the agency
Ultimately the FDA can go along with those recommendations or ignore them
This committee, called the Vaccines and Related Biological Products Advisory Committee
was set to evaluate which strains of flu to include in next year's flu vaccine
Makary said he had nothing to do with canceling the meeting
"You have my commitment to review what the committees are doing
I was critical when that committee was not convened at all during one of the COVID booster guidance decisions by the FDA."
there's a "huge difference" between cancelling one of these meetings for the flu shot
in which he says members just "rubber stamp" what international organizations already recommend
and having a transparent meeting over whether "requiring every 12-year-old girl in America to take an eighth COVID booster shot."
He also answered questions about conflicts of interest for the members of the committees
"We need to review the ethics policy because people see things that appear to be a cozy relationship between industry and the regulators that are supposed to be regulating the products," he said
But we need to call balls and strikes and then keep that independent scientific review process free of any conflicts."
To have the experience needed to serve on these committees
members often have histories working with companies doing the kind of work they are helping to evaluate
asked if Makary would reinstate the vaccine panel's meetings
that "vaccines save lives." Makary added later
"And I do believe that any child who dies of a vaccine-preventable illness is a tragedy," in a response to a question about the recent measles outbreak and HHS Secretary Kennedy's stance on vaccines
Makary said he would take a look at the recent firings at the FDA, which included people working on factory inspections, medical device evaluations and veterinary drug reviews
you have my commitment that I will do an assessment of the staffing and personnel at the agency," he said
"I have not been involved in any of the decisions regarding any of the personnel changes recently
you have my commitment that I will do an assessment."
Collins pressed him on whether he would be the one doing the assessment
High prescription drug prices came up a few times during the hearing
which is something Makary said he thinks he can help solve
He suggested that if more drugs were sold without a prescription
"It would force the companies to put a price on the shelf," he said
It will have an effect on the entire marketplace."
He also said he wanted to work with Congress to rein in abuse of the patent system, which has allowed companies making brand name drugs to stave off competition from generics and biosimilars
Makary brought up the effect of food on health: "Childhood obesity is not a willpower problem
and the rise of early onset Alzheimer's is not a genetic cause
and we will be addressing food as it impacts our health."
he would instruct the FDA to look into chemicals in food that cause inflammation
even though they're currently recognized as safe
"Half of our nation's children are sick and nobody has really been doing anything meaningful on this front until we have gotten new momentum and enthusiasm from Secretary Kennedy and President Trump to finally address the root causes of these diseases," he said
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The FDA is eyeing a “new pathway” for rare disease drugs, potentially allowing for their approval even without data from a randomized, controlled clinical trial, recently confirmed FDA Commissioner Marty Makary said in an interview on Friday
Speaking on The Megyn Kelly Show in his first major interview since taking the reins at the FDA
Makary said the agency will open a new regulatory pathway based on what he called a “plausible mechanism,” focusing mainly on rare or incurable diseases that affect “a small number of people.”
The mechanism is scientifically plausible that this treatment would help these individuals.” The FDA could approve that therapy on “a conditional basis
even though we don’t have a randomized controlled trial because it’s not feasible.”
was quick to caution that “no one is forcing these medications [on patients].”
This new mechanism-driven pathway would also include a patient surveillance system
which would “monitor everybody who gets [the treatment]
so that we can make inferences as soon as the data speaks,” Makary added
The interview comes after Peter Marks, former head of the Center for Biologics Evaluation and Research, left the agency late last month
Marks was a supporter of regulatory flexibility for rare diseases
In an interview with BioSpace in July 2024
Marks emphasized the importance of accelerated approvals for rare disease therapies
noting that timely treatment for these patients could lead to dramatic improvements in quality of life
Makary also addressed what he called the “epidemic of distrust” toward the FDA—and health authorities in the U.S
more broadly—fostered primarily by the agency’s alleged downplaying of side effects of the COVID-19 vaccines
Makary also touched on vaccines, seeming to partly back up claims made by Health and Human Services Secretary Robert F. Kennedy Jr. connecting vaccines to autism despite overwhelming evidence to the contrary
While noting the possibility that certain environmental factors may indeed play a role in the developmental disorder
Of the supposed distrust against health authorities
Makary said that “part of it is warranted.”
“When you don’t want to look at complications
the complication rate looks lower than it really is
It makes products look safer than they really are.” At the center of this
is the FDA’s Vaccine Adverse Event Reporting System (VAERS)
which is intended to monitor side effects associated with immunization
meaning it relies on patients and physicians to file reports of side effects
“You can make no inferences about rates” using self-reported data
“That’s just a basic scientific methodological principle.”
the FDA will now employ a more comprehensive method
tapping into what Makary called the “Health Information Exchange,” a “massive” repository of electronic health record data
which will allow the FDA to look at real-world complication data
Last week, Kennedy also took aim at VAERS
“it’s outrageous that we don’t have a surveillance system that functions.” Kennedy is eyeing certain improvements to the VAERS
including an automated adverse event monitoring system
which he said could help find the “contribution” of vaccines to a yet unclear outcome
By Lizzy Lawrence
WASHINGTON — When Marty Makary was tapped to lead the Food and Drug Administration, public health experts hoped the Johns Hopkins physician would shield the agency from the vaccine criticism of his boss
Makary seems just as willing to use his power and position to more harshly scrutinize vaccines and to shift vaccination policy
By Ed Silverman
Ed Silverman
Ed Silverman, a senior writer and Pharmalot columnist at STAT, has been covering the pharmaceutical industry for nearly three decades. He is also the author of the morning Pharmalittle newsletter and the afternoon Pharmalot newsletter
especially since the middle of the week is upon us
we have made it this far so we are determined to hang on for another couple of days
The alternatives — at least those we can identify — are not particularly appealing
So what better way to make the time fly than to keep busy
So grab that cup of stimulation and get started
Here are a few items of interest to help you get started
And we will once again remind you that we changed our settings to accept postcards and telegrams
who said “there will not be a reorganization,” when asked about restructuring the agency’s centers and divisions
he said he rejected a reorganization proposal that was offered by some staff
and the agency now only plans to consolidate travel offices and information technology
Department of Health and Human Services executed massive layoffs April 1 across federal health agencies
aiming to drastically reduce the size of its workforce
The cuts targeted 3,500 employees at the FDA and broadly hit staff working in program management
Drugmakers spent a record $31 million to lobby in Washington last year, and about $13 million in the first quarter of 2025, according to public filings. Millions more went to donations to political groups and ads, many of which blamed pharmacy benefit managers for the high price of drugs, The Wall Street Journal tells us
executives at the PhRMA drug industry trade association told the group’s lobbyists that taking on pharmacy benefit managers was a top priority in 2025
Drug executives spent much of their time at a million-dollar-dinner with Trump urging him to focus his criticism on benefit managers
They have repeatedly talked to Trump and his top aides in recent months
and some are slated to be in the Oval Office on Wednesday
“Pharmaceutical companies have been pretty successful in shifting a disproportionate amount of blame to the PBMs,” said Steve Knievel
a health-policy expert with Public Citizen
It’s been a little more than two weeks since Marty Makary took over as FDA commissioner and already he’s announcing plans for a new
Marty Makary is the new commissioner of the FDA under President Trump, an agency that he has a history of criticizing and which is now facing sweeping layoffs. Health secretary Robert F. Kennedy Jr. announced that the FDA would fire around 3,500 employees
Makary became a go-to commentator during Covid for his critiques of vaccine mandates
and the CDC’s transparency during the pandemic
He’s written books about challenging the medical status quo and the problem of overtreatment
His position as a Covid contrarian and anti-establishment thinker brought him closer to Kennedy and the Make American Healthy Again movement
FDA Commissioner Marty Makary gets off to a bruising start as agency is wracked by layoffs
RFK Jr. brings FDA under tighter control with HHS workforce cuts
Marty Makary and Jay Bhattacharya confirmed by Senate as FDA commissioner and NIH director
Truth-teller or reflexive contrarian: Which Marty Makary would run the FDA?
Trump’s FDA director pick has a history of criticizing the agency. Here are seven examples
Office of Speaker of the House Mike Johnson
Fifty influential people shaping the future of health and life sciences across biotech
iStock, M_Light_Zone†
“There literally should be nothing political about the FDA,” Marty Makary, the agency’s newly minted commissioner, declared during an interview earlier this month with former Fox News journalist Megyn Kelly
Makary has not shied away from political commentary
and there have been questions regarding possible political interference in FDA decisions—particularly those regarding vaccines—since Makary’s boss
Health and Human Services Secretary Robert F
On April 1, the FDA missed its PDUFA deadline for Novavax’s COVID-19 shot after reports that principal deputy commissioner Sara Brenner
who was acting commissioner prior to Makary taking office
The Novavax PDUFA was the first action item on Steele’s desk
But rather than signing off on the approval
who then “bumped it up” to Kennedy’s office
In Marks’ resignation letter
the former biologics boss wrote that Kennedy “wishes subservient confirmation of his misinformation and lies.” Truist Securities in a March 31 note to investors said this statement insinuates that the secretary may have plans for an anti-vax propaganda campaign
The HHS secretary and FDA commissioner are appointed by the president and confirmed by the Senate
while all employees in the review centers hold nonpolitical positions
“One of the commissioner’s most important jobs is to shield the agency professionals from political interference,” he said
The fact that Novavax’s application ended up in front of Kennedy is a clear sign that the agency is not shielded from the administration as it should be
While attempts have previously been made to draw the FDA into ideological battles
University of Minnesota professor Richard Painter
who served as chief ethics officer under President George W
said he has never heard of the FDA itself being a “political hot button.”
But the FDA rejected the application after a memorandum from Sebelius overruled the recommendation—backed up by President Barack Obama himself
Judge Edward Korman overturned Sebelius’ decision to place limits on Plan B
saying the ruling was made in “bad faith and improper political influence.”
Hamburg told Sebelius that if she overrode the FDA’s decision on this matter
In a recent post on his website
policy expert Steven Grossman called on Makary to make a similar stand against politicization of the FDA
“The integrity of FDA and of FDA decisionmaking are at risk,” he wrote
Makary to either be the bulwark against politicization
or to defer to his boss and the prevailing ethos of the new Administration.”
who served as associate commissioner for external relations at the FDA from 2003 to 2004
was responsible for briefing then–HHS Secretary Tommy Thompson on these types of “hot button issues” that came in front of the FDA
The secretary had a lot of very strongly held beliefs
to change the agency’s action,” Pitts said
He expressed a similar confidence in Makary: “Marty Makary is an honest guy
and he’s not there to rubber stamp the secretary’s decisions full stop.”
BMO Capital Markets analyst Evan Seigerman told BioSpace that Makary is less extreme than Kennedy in his views on vaccines but cautioned that the commissioner may be a bit under the secretary’s thumb
“Clearly he knows who his boss is,” Seigerman said
Novavax and other vaccine-focused companies have already felt the reverberations of potential political influence or concern over such. Shares of key vaccine makers, including Pfizer, Eli Lilly and Novavax, fell in November 2024 after Trump announced his intention to nominate Kennedy
“A more politicized FDA is negative for the biopharmaceutical industry, in our view,” Leerink Partners wrote in an April 25 note to investors in response to a recently published article by Pink Sheet
who said that any company “working with a drug that is not ideologically favored” could be at risk
immunology and reproductive health but said “there could be others as well
depending on the whims of the secretary of Health and Human Services.”
Cuts to NIH grants could also have long-term implications for biopharma pipelines. In an interview with BioSpace earlier this month
founder of the Center for Breakthrough Medicines
called NIH funding “the lifeblood of early-stage discovery” in cell and gene therapy
the biggest impact to biopharma of a “political FDA” would be on Prescription Drug User Fee Act (PDUFA) dates
“The most important thing to sponsors isn’t speed
and PDUFA provides predictability via user fee dates,” he said
“So when user fee dates become less predictable because of potential interference from outside the FDA review team
the regulatory relationship between sponsor and agency suffers
the appetite for investing in riskier development programs suffers
Pitts noted that the Novavax PDUFA delay occurred before Makary was sworn in as FDA commissioner
As for whether the biopharma industry should be concerned about it setting a precedent
because the Health and Human Services is run by Kennedy
“We may have increasing politicization over the FDA
WASHINGTON, April 23, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. ("Vanda") (Nasdaq: VNDA) today announced that FDA bureaucrats have committed to delay Vanda's request for a hearing on the approvability of tradipitant for gastroparesis
FDA bureaucrats identify a new scapegoat: newly appointed Commissioner
FDA last night represented to a federal court that the April 1 reduction in force is partially to blame for the Center for Drug and Evaluation and Research's delay of at least 6 more months to provide its recommendation on whether the Commissioner should hold a hearing
Commissioner Makary said that any cuts were not to scientists or reviewers
the very individuals who supposedly need more time
has requested the same or longer delay to provide hearing recommendations: CDER took six months to submit a proposed order on the Hetlioz jet lag hearing request and likewise requested six months for the Hetlioz insomnia application
It is unfair for CDER and its lawyers to blame the recent reductions in force for their habitual institutional delays on hearing requests
These statements also conceal the extraordinary fact that FDA has denied every hearing request on new drug approvability for at least the past decade
FDA bureaucrats have created policies to avoid scrutiny of their decision-making by habitually denying hearings
FDA's office of chief counsel and DOJ have repeatedly defended FDA's right to act unlawfully when confronted by federal judges
When considering FDA's delay in resolving Vanda's hearing request in a prior instance
a federal judge pointedly asked FDA whether it was "conceding that the [HHS] Secretary is presently not complying with the statute," and a DOJ lawyer replied
We urge Commissioner Makary to step in and restore adherence to the law at FDA; targeted RIFs are not to blame for FDA's culture of delay and closemindedness
Commissioner Makary and Secretary Kennedy have been clear that "radical transparency and common sense" should be the operating culture of the Agency
We also urge Attorney General Bondi to stop DOJ lawyers from defending unlawful Agency actions
"Vanda has fought for 'transparency and common sense' for years because rational innovation can only thrive in a democracy and not in a bureaucracy
It is time for FDA and DOJ to stop fighting innovators like Vanda
focus on what is broken and listen to ideas of how it can be fixed
and usher in a new era of rational and transparent decision making," said Dr
Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit www.vandapharma.com and follow us on X @vandapharma
Corporate Contact:Kevin MoranSenior Vice President, Chief Financial Officer and Treasurer Vanda Pharmaceuticals Inc.202-734-3400[email protected]
Jim Golden/Jack Keleher/Dan MooreCollected Strategies[email protected]
(Vanda) (Nasdaq: VNDA) today announced participation at the American Academy of Neurology (AAN) Annual Meeting
(Vanda) (NASDAQ: VNDA) today announced that a New Drug Application (NDA) was submitted to the U.S
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By Elizabeth Cooney
Elizabeth Cooney
Elizabeth Cooney is a cardiovascular disease reporter at STAT
Get your daily dose of health and medicine every weekday with STAT’s free newsletter Morning Rounds. Sign up here
Liz Cooney here taking a turn with the newsletter while Theresa’s out exploring the world
potentially hobbling efforts to protect people from evolving disease
Looking ahead to the next four years, 44% of Americans say leadership changes in the federal government have eroded their trust in public health recommendations. That’s down from the 77% of adults who previously had faith in the CDC’s guidance after the Covid-19 pandemic, a poll released today by the Harvard T.H
Chan School of Public Health and the de Beaumont Foundation reports
There is a divide along political party lines
with Democrats more doubtful (76%) and Republicans more confident (57%) about health directives going forward
Among the adults concerned about the next four years
most fear that the CDC’s recommendations will be skewed by politics (76%)
and that it will trim programs too much (75%)
downplay infectious disease outbreaks (72%)
and reduce public access to important health information on such topics as about vaccines (70%)
or in lower-income countries where rates are even lower
The end of compounded versions of powerful and popular obesity drugs leaves people adrift after depending for two years on copies of medications such as Wegovy
Because they are no longer deemed by regulators to be in shortage
compounding pharmacies and the telehealth companies they work with must stop offering copies of the treatments
It’s a struggle to afford the brand-name medications that can cost as much as $1,000 a month
The pharma companies Novo Nordisk and Eli Lilly now offer their drugs at around $350 to $700 a month
but that’s still unaffordable to many patients used to paying about $200 a month
“This is kind of like a lose-lose situation,” said Yale’s Reshma Ramachandran
a physician who treats many patients taking compounded GLP-1s because they can’t afford the branded drugs
These are more than pocketbook concerns. When patients suddenly stop these medications, their weight and all the other health conditions they were trying to address, like high blood pressure, cholesterol, and blood sugar, can rebound. STAT’s Katie Palmer and Elaine Chen explain how compounding and telehealth companies are responding
including with offers of “personalized” doses
Life expectancy in many southern U.S. states — especially for females — has risen little over the past century, according to a new analysis of mortality from 1900-2000 published Monday in JAMA Network Open
Despite life expectancy increasing nationally by 10 years in that period
life expectancy for females increased by three years or less in several southern states
life expectancy increased between 1900 and 1950
Areas with the highest gains in life expectancy included Washington D.C.
which saw an increase from 61.1 to 72.8 years of age
as well as the western and northeastern U.S
The researchers attribute these state level differences to factors such as cigarette smoking
such as the tobacco-control policies adopted decades ago by some states
could have a marked impact on life expectancy and suggested state level analysis of mortality should help guide policy choices and public health resource interventions
The Trump administration is set to release a sweeping report about transgender health for children in the United States, according to a new executive order published Monday
The order says the report has been developed by “eight distinguished scholars.” The document also summarizes how the federal government has targeted the trans community and trans health care since the president’s inauguration in January — including ending 215 health grants totalling over $477 million
pushing medical professionals to stop using internationally recognized standards of care
and ending federal health benefits coverage for the “mutilation of the children.”
Many experts expect the review to resemble the so-called “Cass Review,” a lengthy overview of health care for trans youth in England that has been decried by researchers and advocates for failing to provide an adequate picture of gender-affirming care
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Marty Makary, a surgical oncologist and health policy researcher who has spent more than two decades at Johns Hopkins, was confirmed Tuesday night by the U.S. Senate as the next commissioner of the U.S. Food and Drug Administration (FDA) in a bipartisan vote
The appointment positions Makary atop the federal agency charged with ensuring the safety
and security of human and veterinary drugs
and medical devices; ensuring the safety of the nation's food supply and cosmetics; and regulating the manufacturing
and distribution of tobacco products to protect the public health
which oversees approximately one-fifth of the U.S
is part of the Department of Health and Human Services
"I am pleased to congratulate Dr. Makary on his new role as FDA commissioner. We are fortunate to have one of our senior faculty members at the School of Medicine undertake this critical service to the nation," JHU President Ron Daniels said
"An accomplished clinician and widely published health policy researcher
Makary has had a storied career at Johns Hopkins that reflects the research and clinical acumen and openness to different ideas and approaches undergirded by academic freedom that are the hallmark of our great institution."
health care practices throughout his career
advocating for efforts to lower the cost of treatment and reduce unnecessary procedures and medical errors
he questioned elements of the nation's response—he was an early advocate for universal masking and vaccines
but later questioned vaccine mandates as well as prolonged school closures
Makary founded the Johns Hopkins Center for Surgical Trials and Outcomes Research and co-developed a surgical safety checklist that is used in operating rooms around the world today
He was the first to perform novel surgical procedures
including his first-in-the-world series of laparoscopic pancreas islet transplant operations
He received emeritus status at the university's Advisory Board of the Medical Faculty upon his departure
Makary's most recent research focused on the underlying causes of disease
but over the course of career he has also conducted research related to cancer
frailty and physiologic reserve in older patients
His development of novel quality metrics in health care has had a meaningful impact on health safety and health outcomes
A member of the prestigious National Academy of Medicine
Makary has published more than 300 peer-reviewed articles and is also the author of three New York Times bestselling books on health care
including Unaccountable (2013) and The Price We Pay: What Broke American Health Care—and How to Fix It (2019)
Blind Spots: When Medicine Gets it Wrong and What it Means for Our Health
"Dr. Makary's 22-year tenure at Hopkins has been marked by great collegiality, partnership, and clinical and research excellence, and his profound love and admiration for Johns Hopkins and of his colleagues shines through in any conversation, public or private, one has with him," Theodore DeWeese
dean of the medical faculty and CEO of Johns Hopkins Medicine
wrote in a message to the Hopkins Medicine community today
Makary for his years of service to our institution and wishing him well on bringing the excellence
and care that defines Johns Hopkins to his new role at the FDA."
Posted in Health, University News
Tagged marty makary
Commissioner Martin Makary said the agency would restrict drug company employees from participating as industry representatives on the advisory committees it sometimes consults
The Food and Drug Administration will aim to limit the participation of industry experts in the advisory committees that the agency consults for some regulatory decisions, Martin Makary, the FDA’s new commissioner, announced Thursday
Advisory committees, which the FDA typically convenes for additional input on high-profile reviews or thorny clinical and regulatory issues, regularly include an industry representative alongside a dozen or so independent experts
These representatives don’t vote on questions put to the committee
They are meant to share the perspective of their industry broadly
rather than of the specific company that employs them
There are also usually patient or consumer representatives on the panels
the FDA will restrict industry representatives from taking part as a committee member
“Industry employees are welcome to attend FDA advisory committee meetings
along with the rest of the American public
but having industry employees serve as official members of FDA advisory committee members represents a cozy relationship that is concerning to many Americans,” said Makary
the FDA has a history of being influenced unduly by corporate interests.”
The FDA’s statement said its new policy won’t prevent company employees from attending or presenting their views at advisory committee meetings
and noted that exceptions could be made in “rare circumstances.”
While advisory committee meetings offer a public window into the FDA’s regulatory processes
The FDA can go against their recommendations
although in practice the agency and its committees often align
the FDA shapes advisory committee discussions by posing specific questions to the panel and guiding the conversation
If the committee is asked for input on a specific drug review
the developer of that drug will usually give an overview of its data
followed by presentations from an FDA review team that scrutinized those results
Committee members ask follow-up questions and then later vote
industry representatives don’t typically drive the conversation
although they share their thoughts alongside other panelists
Makary announced the new policy alongside his first interview as FDA commissioner, conducted at the agency’s offices with former Fox News host Megyn Kelly and posted to the social media site X
“I was shocked when I learned that employees of big pharma companies sit on FDA advisory committees as members,” Makary told Kelly
“We’re going to be replacing them whenever statutorily possible with patients and family caregivers.”
It’s not clear whether Makary would try to add an additional patient representative in the many instances where existing advisory committees already have one
It’s also unclear which advisory committees could be stripped of their industry representative
The Food and Drug Administration Modernization Act of 1997 amended existing law to direct the Health and Human Services Secretary
to establish expert panels that include “a representative of interests of the drug manufacturing industry not directly affected by the matter to be brought before the panel.”
Makary has made common cause with his boss
in targeting perceived industry influence over regulators like the FDA
challenging medical consensus on several issues
including the need for children to receive COVID-19 boosters
He was sworn in as FDA commissioner on April 1 and has kept a relatively low profile as Kennedy has ordered a drastic downsizing of the federal health workforce
including layoffs for several thousand FDA employees
Editor’s note: This story has been updated with additional detail on a 1997 law governing advisory committee formation
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Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade
Just two acquisitions in the first quarter
a notable decrease from the six seen during the same period last year
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The Johns Hopkins surgeon will helm the agency as layoffs loom and important decisions on treatments for rare and infectious diseases await
The U.S. Senate on Tuesday confirmed Johns Hopkins University surgeon Martin Makary to be commissioner of the Food and Drug Administration
giving him authority over an agency that regulates a wide swathe of the U.S
Three Democrats joined 53 Republicans to confirm Makary in a 56-44 vote
the senior minority member of the Senate Health
saying in a statement that his “medical background and strong vision make him more than qualified to lead the FDA.”
Makary will take the helm of an agency employing some 18,000 people under likely orders from President Donald Trump to cut the workforce while reviewing a growing number of drugs
About half of the FDA’s workforce is funded through user fees such as those authorized under the Prescription Drug User Fee Act
In his confirmation hearing before the HELP committee, Makary said he wasn’t involved in the decision to lay off hundreds of FDA employees
and indicated his evaluation of any future reductions would be independent and ensure that remaining agency workers would have all the necessary resources to do their jobs well
John Crowley, head of the Biotechnology Innovation Organization, a powerful trade group, cautioned in an op-ed in Stat against “aggressive and often indiscriminate personnel cuts.”
Many conservatives want the FDA to impose tighter restrictions on mifepristone and misoprostol
The medication accounted for 63 percent of U.S
Amna Nawaz spoke with the new FDA commissioner
Notice: Transcripts are machine and human generated and lightly edited for accuracy
Now to the new Food and Drug Administration commissioner
and a wide-ranging conversation I had with them earlier today as part of Semafor's World Economy Summit
mifepristone and what plans the FDA may have in store for medication abortion
do you expect to take action further restricting the pill
I have no plans to take action on mifepristone
Could that be changed over the course of your tenure
given the pressure that I know has come from a lot of the political forces at play on this
there is an ongoing set of data that is coming into FDA on mifepristone
So if the data suggests something or tells us that there's a real signal
then I — we can't promise we're not going to act on that data that we have not yet seen
But if that new data contradicts the decades of data that said that it's overwhelmingly safe and effective
rather than the precedent of decades of data
because you're talking about the strength of the data
So it's very hypothetical what you're asking
Commissioner Makary also seemed to agree today that the federal government should reverse itself and no longer recommend the COVID booster for children
Previous scientific panels have found it safe and beneficial to prevent kids from getting seriously ill
but Makary told me today he believes that evidence is insufficient
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More than 20 of the agency’s roughly 60 travel staff will be reinstated, according to two FDA staffers notified of the plan this week, who spoke on condition of anonymity to discuss confidential agency matters.
Food scientists who test samples for bacteria and study potentially harmful chemicals also have been told they will get their jobs back, but have yet to receive any official confirmation.
The same uncertainty hangs over employees who process agency records for release to lawyers, companies and journalists under the Freedom of Information Act. About 100 of those staffers were recently eliminated, according to an agency official with direct knowledge of the situation.
But in recent days the FDA has missed multiple court-ordered deadlines to produce documents, something that could result in hefty fines. That’s prompted plans to bring back a significant number of those staffers.
The apparent reversals are the latest examples of the haphazard approach to agency cuts that have shrunk FDA’s workforce by an estimated 20%, or about 3,500 jobs, in addition to an unspecified number of retirements, voluntary buyouts and resignations.
In February, the FDA laid off about 700 provisional employees, including food and medical device reviewers, only to rehire many of them within days after pushback from industry officials, members of Congress and others.
The Department of Health and Human Services hasn’t detailed exactly which positions or programs were cut in the mass layoffs.
FDA Commissioner Marty Makary has repeatedly said that no FDA scientists were fired as part of the reductions. But at least two dozen food scientists who worked in a San Francisco testing laboratory and a Chicago research center were let go in March.
An HHS spokesperson suggested the apparent mix-up was due to “the fractured, outdated HR infrastructure we inherited from the Biden administration and are now actively overhauling.” The spokesperson did not respond to specific questions about which employees are being reinstated but said the administration will “streamline operations and fix the broken systems left to us.”
“I hope Commissioner Makary continues to assess these ill-informed cuts and works to bring back impacted employees expeditiously,” said Susan Mayne of Yale University, the FDA’s former food director. “His legacy as commissioner is on the line.”
With more than 15,000 employees remaining across various U.S. and foreign offices, the FDA’s core responsibilities are reviewing new drugs, medical products and food ingredients as well as inspecting thousands of factories.
Makary has said no inspectors or medical reviewers were fired as part of the recent reductions. But current and former FDA officials note that those frontline employees are often supported by teams of administrative staff.
FDA inspectors, for example, have long relied on travel bookers to coordinate trips to India and other countries that often involve visa permissions, security measures, ground transportation, tech support, translation services and other logistics. Inspectors can spend up to half the year traveling, a grueling workload that makes recruiting and retaining staff a challenge.
For a brief period last month, inspectors were told they would be booking their own travel. The FDA set up a hotline to assist with making the arrangements. Then, agency leaders developed a plan to hire an outside contractor to perform the work.
On Monday, staffers were informed that about a third of the fired staff who performed the work would be returning.
Perrone writes for the Associated Press. AP reporter JoNel Aleccia contributed to this report.
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The US Senate voted Tuesday to confirm Martin Makary
to lead the US Food and Drug Administration and the National Institutes of Health (NIH)
who is a member of the National Academy of Medicine and chief of islet transplant surgery at Johns Hopkins University in Baltimore
and previously served as the editor-in-chief of MedPage Today
picking up support from three Democratic senators
a health economist and emeritus professor of health policy at Stanford University in Palo Alto
was voted in as director of the NIH along strict party lines 53-to-47
Aligned with US Health and Human Services (HHS) Secretary Robert F
Kennedy Jr.’s agenda to “make America healthy again,” Makary and Bhattacharya made promises during their respective hearings to improve the lives of people with chronic disease
who served as FDA commissioner from 2016 to 2017 and again from 2022 to January 2025
said he’s hoping for the best even though both Makary and Bhattacharya “are coming into jobs leading a demoralized workforce that has been decimated by reductions in force and demeaning depictions of the work they do by the incoming administration.”
“there is tremendous uncertainty in the 20% of the economy regulated by FDA and the vast external research community that has been the envy of the world because of NIH’s tradition,” he said in an email
“I cannot tell from their previous statements exactly what they plan on doing
AdvaMed, a lobbying group for the medical device industry, issued remarks from its president and CEO
“As a renowned surgeon and health policy expert
Makary understands the importance of improving patient access to transformative medtech,” he said
Bhattacharya was congratulated by Stanford University, which said in statement that “as an institution
we are staunch supporters of the NIH's mission
which continues to advance the frontiers of medical knowledge and unlock new possibilities for health improvement.”
Yael L. Maxwell is Senior Medical Journalist for TCTMD and Section Editor of TCTMD's Fellows Forum
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The Senate late Tuesday voted 56-44 to confirm Martin Makary to lead the Food and Drug Administration.
a former Johns Hopkins University surgeon and author
has been widely seen as a more middle-of-the-road pick compared to Trump’s other choices to lead the Department of Health and Human Services’ subagencies.
He will take over leadership of the agency as the FDA has been targeted by sweeping cuts by Elon Musk’s so-called Department of Government Efficiency. Makary said during his confirmation hearing that he will review recent cuts to the FDA’s workforce
“I have not been involved in any of the decisions regarding any of the personnel changes recently
you have my commitment that I’ll do an assessment,” he said during his March 6 appearance before the Senate
The HELP committee advanced Makary’s nomination on a 14-9 vote March 13
John Hickenlooper of Colorado and Maggie Hassan of New Hampshire voting to advance his nomination
Hickenlooper voted against his confirmation Tuesday night.
Makary picked up a trio of Democratic votes — Hassan
Makary has criticized school closures during the COVID-19 pandemic and was skeptical of the need for COVID-19 boosters in kids.
an area that has become controversial with HHS Secretary Robert F
Kennedy Jr.’s history of vaccine skepticism
and I do believe that any child who dies on a vaccine-preventable illness is a tragedy in the modern era,” Makary said
Makary will join the agency as the future of the FDA’s advisory committee system appears to be in flux
The agency last month abruptly canceled the meeting of the Vaccines and Related Biological Products Advisory Committee during which they were expected to make a recommendation on which flu strain to target with the fall and winter vaccine rollout.
Makary’s medical background and strong vision make him more than qualified to lead the FDA,” he said
make sure to check out this week’s edition of BioPharm Executive where we take a deep dive into another sizzling space—transthyretin amyloid cardiomyopathy (ATTR-CM)—and find out which pharma CEO has the biggest golden parachute
Using his “guiding principles” of common sense and gold-standard science
he plans to focus on reforms including improved pathways for medical AI and food safety as well as addressing employee morale
WASHINGTON — Marty Makary’s first address to staff as FDA commissioner on Wednesday afternoon sounded a bit like an introduction to one of his books
It included sweeping declarations about the need to challenge scientific norms
and to identify the root causes of chronic disease
Makary sprinkled in anecdotes intended to stir emotion
recounting the time when his father gave a cancer patient a hug at the grocery store
But he said little about specific plans for his agency and about the Health and Human Services department layoffs that roiled staff on Tuesday
according to a recording of the town hall obtained by STAT
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In his first sit-down interview since beginning his role as FDA commissioner 17 days earlier, Marty Makary, a former Johns Hopkins surgeon and the only Trump pick for HHS whose confirmation received Democratic support, said he would speed up approvals for rare-disease treatments by reducing reliance on animal testing and shifting towards organoids and computational models.
In her own words: Rathmell reflects on her priorities and values—and encourages everyone to do the same
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WASHINGTON, D.C.—The nonprofit Physicians Committee for Responsible Medicine, which promotes the use of human-relevant test methods to replace animal use in drug testing and development, enthusiastically supports the U.S. Food and Drug Administration’s historic decision to phase out animal use in favor of human-relevant methods
to test monoclonal antibody therapies and other drugs
“FDA Commissioner Marty Makary’s historic announcement that the FDA will phase out animal testing ushers in a new era for drug testing that will save human and animal lives by integrating better science to make better decisions for health,” says Elizabeth Baker
director of research policy for the Physicians Committee for Responsible Medicine
“The announcement reflects that innovation
it’s been known that animal testing for drug development is not good science
The costs to develop drugs are staggering—at $2.6 billion per drug
Companies lose $500,000 to $1 million in revenue each day a drug is delayed from entering the market
and more than 90% will fail in clinical trials
largely due to being unsafe or ineffective for humans
safe and effective medications may be screened out by unpredictive animal tests
and other animals are used in painful and often lethal procedures
Human-based technologies—such as advanced in vitro models and sophisticated computer modeling
and more predictive of human outcomes than traditional animal tests
makes them more biologically relevant to humans
avoiding translational issues that come with cross-species extrapolation
“As more of these predictive methods are integrated into decision-making
companies can make more informed decisions about which compounds to advance or leave behind
leading to safer and more effective compounds advancing to clinical trials at less cost,” says Baker
Support for Commissioner Makary’s action has never been stronger. In a recent poll
86% of Americans agreed that “animal experimentation should be phased out in favor of more modern research methods.”
Congress supports this from both sides of the aisle
Congress sent a letter to the FDA requesting the agency update its regulations and establish a path for acceptance of nonanimal methods
Congress passed Appropriations report language directing FDA to modify regulations to clearly reflect the ability to accept valid
the bipartisan FDA Modernization Act 2.0 clarified that FDA can accept nonanimal methods
A 2023 letter from Congress asked the FDA to update its regulations to remove requirements for animal testing
Congress also provided $5 million for the agency to reduce animal testing through nonanimal methods
The recently introduced FDA Modernization Act 3.0 seeks to change FDA regulations to ensure clarity that nonanimal methods are accepted
the Science Board to the FDA provided key recommendations for integrating modern methods
Recommendations include creating a central office to drive progress
tracking metrics to measure impact and progress
building an accessible agency-wide framework for accepting new methods
providing transparent and rigorous review for applications that involve these methods
and establishing a central database for clarity on which new methods are in use
Historically, the agency has engaged in many activities related to nonanimal methods, including the 2017 Predictive Toxicology Roadmap, the 2019 Alternative Methods workgroup, the 2020 Innovative Science and Technology for Advancing New Drugs (ISTAND) pilot program, and the 2023 New Alternative Methods Program, but until now the agency lacked strong leadership to drive innovation. This commitment from FDA leadership was sorely needed to drive culture change.
asking him to commit to reducing and replacing animal testing with modern human-relevant methods; to change policies that currently require or recommend animal experiments; and to implement recent recommendations of the Science Board to the FDA to embrace modern approaches
changing policies to clearly reflect acceptance of nonanimal methods will be key—this includes regulations and guidance
Other important activities: training agency staff to build competence in new methods and create internal knowledge base
and leveraging human data for evaluation of new methods
the Physicians Committee for Responsible Medicine is a nonprofit organization that promotes preventive medicine
and encourages higher standards for ethics and effectiveness in education and research
Food for Life classes teach you how to improve your health with a plant-based diet
The Make America Healthy Again (MAHA) movement proved to be more than just a campaign talking point for Donald Trump
is now heading up the Department of Health and Human Services (HHS)
was tapped to lead the Food and Drug Administration (FDA)
Makary breezed through the Senate confirmation process (even getting the support of a few Democrats along the way)
His time thus far has been spent listening and learning to get a better sense of the state of the agency and what needs to be done to restore the public trust that eroded during the COVID pandemic
Megyn traveled to FDA headquarters to sit down with Dr
Makary for his first interview as commissioner and learn more about steps he is already taking to limit conflicts of interest with Big Pharma
Makary was a highly respected surgical oncologist at Johns Hopkins and health policy expert
who emerged as a leading voice of reason during COVID
He said he “had a lot of emotions” leaving the operating room for the final time before his confirmation hearings began and is now adjusting to the “entirely different world” he finds himself in
was the opportunity to address the public health problems in the U.S
“We are not on a good path as a country in terms of the health outcomes,” Dr
“We do great with sophisticated operations and amazing drugs that can treat certain kinds of lymphoma and other types of cures
but when it comes to the health of the population right now we have had this massive… rise of all these chronic diseases.”
He believes the country needs to “start talking about our problems” rather than “just keep throwing medications at them,” and that requires a blend of established orthodoxy and new thinking
“We need the old guard to ensure that we hold to rigorous scientific methodology
new ideas at the same time,” he said
“We’re trying to bring all of that together now.”
Makary acknowledged that many Americans have lost faith in public health entities like the FDA
Part of that has to do with the fact that there often appears to be a revolving door between these agencies and major pharmaceutical companies
The movie Dopesick and other projects about the opioid crisis often reference Curtis Wright
the medical review officer who approved OxyContin while at the FDA and then went on to work for the drug’s maker Purdue Pharma.
who was FDA commissioner from 2017 through 2019 during the first Trump administration
is a member of Pfizer’s board of directors
a lot of people in the United States feel that the system is rigged
A lot of people feel that the relationship is too cozy between pharma and regulators,” Dr
“This is an agency that belongs to the American people
ensure that the scientific evaluation process is totally independent.”
That is why one of his first acts as FDA commissioner is aimed at limiting conflicts of interest
“Today we are announcing that we are removing industry members – pharma members – from FDA advisory committees,” Dr
“I was shocked when I learned that employees of Big Pharma companies sit on FDA advisory committees as members of those committees
with patients and family caregivers.”
He said the dynamic between the pharmaceutical industry and government needs to change
“We have to partner with industry and pharma to facilitate the process to make it user-friendly and expeditious… We want American pharma companies to do well and companies that do business in the United States to do well,” he added
“But the scientific evaluation needs to be independent.”
“The pharma and device members on those advisory committees will say that they are ‘non-voting members,’ but there is a sort of a close club of individuals that has a running dialogue… [and] we need the scientific evaluation and the voting to be totally independent,” he continued
we are pro-pharma but our evaluation has to be independent and we cannot have any more indications for chronic pain written for a drug based on a 14-day study where the regulator then goes immediately works for the company.”
that is “the kind of thing that breeds distrust” and is “why people perceive that this agency
As it relates to senior leaders ferrying back and forth between government and drug manufacturers
Makary said the FDA needs a culture change because the country should not “tolerate” the behavior
“We cannot have people who leave as regulators go to the industry
and we’ve thought about an ethics pledge
We’ve thought about all kinds of things,” he said
“It’s non-binding because we live in a free country… but what we can do is create a culture here where people want to stay
We can ensure that people who leave don’t have undue influence.”
The “influence” piece is something he has already experienced firsthand
“When I got this nomination for the job
‘the swamp’ reached out to me,” Dr
I was in the operating room and the next day I discovered what the swamp was.”
He said the overtures came in the form of things like “we want to help you with your confirmation,” or “we want to write a letter on behalf of our company to the senators on your committee,” or “we know these senators
we’re going to talk to them if it’s okay with you.”
“You know what I said: Don’t talk to the senators
Those are obligations that then you feel indebted to return once you’re in office
and I’d rather not get confirmed into this job and have those obligations,” he shared
Makary is currently on “a listening tour” at the FDA to speak with the career scientists and various departments that he said have become “siloed” with their own “communications [team]
and IT departments” that do not talk to one another.
this set up gets back to the ‘culture’ issue
“We are trying to make sure they have all the resources they need to do their job well,” Dr
“And we are trying to change the culture here… to a culture of teamwork
If agencies like the FDA are to learn anything from the MAHA movement
“There should be nothing political about the FDA
We’re talking about values,” Dr
and independent moms came out and voted for President Trump and they believed in Secretary Kennedy’s MAHA mission.”
“There is literally nothing political about looking at the influence of food colors and ingredients
and getting infant formula without seed oil and added sugar
which we’re doing right now,” he concluded
“These are the most apolitical things in society.”
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The FDA doesn’t plan to execute a reorganization of the agency but will consolidate offices that handle travel
an HHS spokesperson confirmed with Bloomberg Law
The Food and Drug Administration’s plan was first reported Tuesday by MedPageToday in an interview with Commissioner Marty Makary
The former Johns Hopkins surgeon said “there will not be a reorganization,” when asked about restructuring the agency’s centers and divisions
Makary in the interview said he rejected a reorganization proposal that was offered by some staff
Makary’s comments come after the US Department of Health and Human Services executed massive layoffs April 1 across federal health agencies
Makary said in the interview that the FDA’s staff has increased over the years
which resulted in an “increased regulatory creep.”
Employees left at the agency have expressed concerns about completing drug
and food reviews after the FDA culled administrative and support staff that would have helped them carry out their responsibilities
said that center leaders are confident they will reach targets under the Prescription Drug User Fee Act (PDUFA)
That act allows the FDA to collect sizable fees from industry so that the agency can complete reviews in a timely manner
Stealth BioTherapeutics Inc., a biotech company, said Tuesday that the FDA communicated that it would not meet the April 29 PDUFA date for its New Drug Application for elamipretide
The FDA has delayed this PDUFA date before
A spokesperson for HHS said the FDA can’t comment on any pending application to preserve the integrity of the review process
“Any delays in drug approvals are the result of the FDA’s rigorous review process
which is designed to ensure that all medications are safe and effective for the American people
These delays are not connected to the recent reorganization,” the spokesperson said in a statement
To contact the reporter on this story: Nyah Phengsitthy in Washington at nphengsitthy@bloombergindustry.com
To contact the editors responsible for this story: Zachary Sherwood at zsherwood@bloombergindustry.com; Brent Bierman at bbierman@bloomberglaw.com
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