At a time when there is a significant void at the top of the US Food and Drug Administration (FDA), experts have said that the trenches remain stable.
While Arena International’s Outsourcing in Clinical Trials (OCT) East Coast conference was taking place in New Brunswick on 12 and 13 May, Marty Makary resigned as FDA commissioner.
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
Since then, three other prominent leadership roles have opened up after Tracy Beth Høeg, who headed the FDA’s drug evaluation centre, Katherine Szarama, acting head of vaccines and Jim Traficant, former chief of staff, were all ousted from the agency shortly after Makary’s resignation.
This also follows Vinay Prasad’s departure as head of the Center for Biologics Regulation and Evaluation (CBER), a role he took during his one-year sabbatical from academia. Prasad had previously left the FDA during his tenure amid backlash on his conservative approach to cell and gene therapy (CGT) approvals – particularly around his regulatory approach to Sarepta’s Duchenne muscular dystrophy (DMD) gene therapy, Elevidys (delandistrogene moxeparvovec). He was then reinstated as CBER head just two weeks later at the request of the FDA.
The leadership upheaval may cause concerns among pharma and biotech companies about instability at the FDA, but former agency staff have said that career FDA employees remain mission-driven, patient-focused, and responsive. Overall, PDUFA timelines and approvals are still being met.
FDA overall doesn’t align with politics
Speaking at OCT East Coast, Rik Mehta, co-Founder and CEO of biotech firm Lactiga and a former FDA staffer, said: “We would like to think that FDA operates agnostic to politics, and generally it does. I would say that the intersection happens during periods of uncertainty.”
Jamie Gamerman, head of US regulatory policy at UCB and former FDA staff, agreed that from the perspective of an FDA employee, the science always comes first. “Politics just isn’t even a consideration, but it’s hard. It’s a tough balance,” she said.
Gamerman said that while there is unrest at the top, the staff are still putting the needs of the agency and patients at the forefront of their work.
“The career people put patients first and politics second. And at UCB, we’re still seeing approvals. All deadlines are being met, and the FDA is still being responsive. The right people are still in the right places, no matter what happens at the top,” she explained.
Mehta said while this is true, new management could lead to regulatory changes which could make it difficult for companies, especially small biotech companies with limited resources, to navigate.
Approvals and refusals can be centred by political opinion
Of course, the FDA holds the authority to approve drugs and can withdraw medicines when it sees fit. This has been seen with the abortion pill mifepristone, which has been very much on the political agenda. In addition, CGTs have recently faced significant backlash, such as Sarepta’s Elevidys being the central reason for why Prasad first left the FDA.
“These are the areas where, without leadership at the agency, we’re going to continue to see struggles with the review division on making controversial decisions,” Mehta admitted.
Management changes can alter policy
According to Meht, the current regulatory landscape is highly unpredictable, particularly for early-stage biotech companies operating with limited resources.
“We are hearing about a lot of different, really exciting programmes coming out of the FDA. Every day I’m sitting and thinking, who’s going to be the next FDA commissioner? Who is going to be the head of CBER or CDAR? How is that going to influence my studies?” he said.
Mehta added that companies are increasingly questioning “what level of data” they will need to present to the FDA, what confirmatory studies may be required, and which endpoints will ultimately be accepted, adding that “there’s so much variability now.”
This has already been seen with Moderna’s mRNA flu vaccine, which was initially rejected by the FDA due to their concerns over the control arm used in the trial – despite the agency previously accepting the same approach when the study was submitted.
Although the FDA later reversed its decision and is now evaluating the vaccine, the episode highlights how leadership changes at the agency can create uncertainty for companies, even when they believe they are following the established protocols.
As a result, panellists agreed that alignment and collaboration with the FDA is vital. This can be as simple as explaining your target product profile (TPP), which outlines the desired ‘profile’ or characteristics of a target product that is aimed at a particular disease or diseases.
Anka Ehrhardt, director of Analytical Research & Development for MSD (known as Merck in the US) highlighted how it is important for parties to understand that the FDA may not have the answer to your question, especially when investigating drugs with novel mechanisms of action (MOA).
“It is understandable that the FDA doesn’t have all the answers. We don’t have all the answers, nor does the FDA, which does bring some insecurity. Learning together with the FDA and understanding what they consider scientific rigour if important – the more we learn together, the easier it will get,” Ehrhardt shared.
As well as open and clear discussions, keeping information easily digestible will assist the FDA in better understanding requests and submissions, said Mehta, and ultimately speeding up the whole process.
“Have your programmes flushed out before you meet with the agency. The less thinking the FDA has to do, the better your engagement will be, and the closer you’ll get to aligning what outcomes you want,” he said.
“The best strategy starts with flushing out your programme and explaining it very clearly, almost at a sixth‑grade level to the agency, so that they fully understand and appreciate where you’re coming from, what you’re trying to do and what you’re trying to develop.”
Staff continue to hold foundations
Despite all the unrest, Gamerman again stressed that the staff dealing with approvals and reviews remain dedicated to their jobs, and that the sector will work through this difficult period. “Times have changed a bit, but the people haven’t. They’re still there to review the products and protect the public, and they will be there tomorrow, no matter what the news headlines say today. Your inspections will continue. They’ll be thorough, but they will be fair, and we’re all going to be okay.”
The consensus is that the coming months are likely to be challenging as new leadership settles into place. While this transition may bring some disruption, industry experts believe the FDA’s core staff will continue working to main stability for sponsors and CROs, ensuring patients continue to receive high quality care.
A detailed agenda for 17th Annual Outsourcing in Clinical Trials East Coast 2026 conference can be accessed here. The conference is hosted by Arena International Events Group, a B2B events company owned by GlobalData, the parent company of Clinical Trials Arena and Pharmaceutical Technology.
Interested in attending or sponsoring OCT East Coast? Complete the form below and the Arena team will be in touch.
