美女免费一级视频在线观看

A month into his new role as commissioner of the Food and Drug Administration (FDA), Dr. 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.

“If we’re not the first, 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. Weinberg, 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.

A rundown of Makary’s first moves

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. Peter Marks.

Makary’s plan for the agency — and the extent to which he’ll be influenced by HHS Secretary Robert F. Kennedy, Jr. — has yet to fully unfold. But he did hint at some of his plans in an interview with former Fox News journalist Megyn Kelly last week.

For one, 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, however, 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. “[That way], 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, analysts said.

“Importantly, 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.

Still, much will depend on who is chosen to replace Marks as head of the CBER, according to BMO analyst Evan Seigerman.

“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.

What could be impacted by FDA layoffs?

Beyond vaccine policy, other therapeutic areas and FDA functions could see changes or a slowdown of regulations and guidance, experts said.

In an April interview with POLITICO, former FDA commissioner Dr. 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.

Amid the cuts, 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, according to Pacifici.

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.”

Finally, 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, Pacifici stressed, 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.

How can the pharma industry prepare?

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. “When those roots are uncertain, it creates stress.”

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. The first step, Weinberg stressed, 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], ‘You know, I know what to do. Let’s just take a breath and we can navigate our way through this.’ I think the companies that do that will persevere.”