After a tumultuous year, experts call for stability while anticipating the first fruits of policies intended to expedite approvals for rare disease drugs.
When the Trump administration assumed power in January, it marked the beginning of a particularly tough transition for the FDA under the leadership of Health Secretary Robert F. Kennedy Jr.
“The most disruptive trend by far was the massive workforce reduction early in the year,” Aparna Thakur, assistant project manager of Forecasting and Analytics at DelveInsight, told BioSpace in an email. By early July, the regulator had lost some 3,500 employees—an approximately 20% reduction in its workforce since the start of the year.
What followed was a chaotic stretch at the agency, marked by unsubstantiated and opaque claims regarding vaccines, regulatory guidance reversals and a string of high-level exits that left remaining staff demoralized and drained the agency’s institutional knowledge.
Speaking to the abrupt retirement of Center for Research and Drug Evaluation (CDER) director Richard Pazdur just three weeks after assuming the position, Chad Landmon said the departure “didn’t give folks in the drug industry a lot of comfort.”
“I think we’re all going to be looking to see who gets in that position and see if there can start being some personnel stability over at FDA,” Landmon, chair of Hatch-Waxman & Biologics at Polsinelli Law Firm, told BioSpace. While Tracy Beth Høeg was named as acting CDER chief on Dec. 3, Landmon wouldn’t hazard a guess as to whether she would be more than a placeholder.
With personnel in flux, there were bright spots this year for the FDA. Thakur pointed to a “burst of innovation” under FDA Commissioner Marty Makary, particularly in terms of expedited reviews, clinical trial monitoring and artificial intelligence. The FDA also published a trove of previously confidential complete response letters, a move Thakur called “revolutionary,” especially as it gave the public—and competitors—insight into the reasons why drugs were rejected.
Heading into the new year, Thakur hopes to see stability at the FDA. “First and foremost,” she said, “I’d love to see the FDA return to full staffing levels and have secure, protected funding.” A regulator that the industry can rely on “helps everyone—especially smaller companies who can’t afford long delays or ambiguity.”
Thakur would also like to see some balance given to the FDA’s innovative streak. She lauded the agency’s efforts to leverage AI and to devise flexible regulatory pathways but said the agency needs to have the right systems in place. “Faster is only better if it’s also trustworthy.”
For this piece, BioSpace spoke with FDA observers and insiders to glean their thoughts on what 2026 could hold for the agency. Experts were largely reluctant to offer definitive predictions, given the current transition period, but educated guesses—based on how 2025 turned out—were fair game.
Will Leadership Instability Continue?
From a personnel perspective, experts agree that 2025 was a catastrophic year for the FDA. “That translated to real-world slowdowns: delayed reviews, missed PDUFA deadlines and fewer guidance documents,” Thakur said.
Some of the staffing difficulties were to be expected with a new administration, but what actually transpired exceeded those expectations, Graig Suvannavejh, managing director of Equity Research at Mizuho Securities, told BioSpace in an email.
“While we totally expected 2025 to be a year of transition at FDA, given a change from the Biden administration to the Trump administration, I don’t think we were really prepared for . . . the mass exodus we saw in staffing at FDA,” he said. Aside from the mass layoffs under Kennedy, he noted that hundreds of employees have also voluntarily left the agency this year.
Suvannavejh also pointed to leadership tumult at the agency, particularly at CDER, which he said “is the single most important agency that exists at FDA.” Including Høeg and Pazdur, the division has been led by a veritable parade of individuals, with George Tidmarsh, Jacqueline Corrigan-Curay and Patrizia Cavazzoni preceding them.
“That’s five people in one year alone. It’s not acceptable,” Suvannavejh said.
There are “certainly a lot more policies being driven out of HHS,” Landmon added. “I think in the past, FDA was really given a lot of autonomy, and certainly that has changed under RFK’s leadership,” he said. “Obviously, he’s driving a lot of this, from vaccine policy to staffing and everything like that, so I think it’ll be interesting to see kind of how that continues, whether the same people continue in those roles.”
Faster Verdicts for Rare Disease Drugs
Despite its organizational struggles, the FDA in 2025 laid the policy groundwork for expedited reviews, particularly for therapies that address rare diseases and conditions with limited treatment options. In 2026, experts say we are likely to see the first therapies approved under these new frameworks.
In November, the agency set in motion its “plausible mechanism” approval pathway to accelerate the development of experimental treatments for diseases affecting an exceedingly small patient population where an underlying biological process has been identified.
Stuti Mahajan, consulting manager at DelveInsight, said she looks forward to seeing this pathway in action. “The FDA will likely double down on [the] new fast-track approval mechanism introduced in 2025,” she told BioSpace via email.
Mahajan also pointed to the Commissioner’s National Priority Voucher (CNPV) program. Launched in June, this scheme seeks to drastically shrink review times from 10–12 months to 1–2 months for drugmakers that meet certain criteria aligned with U.S. national priorities. The FDA has handed out 15 of these priority tickets—which Mahajan expects will see play in 2026. The first approval has already been granted to Augmentin XR, manufactured by USAntibiotics.
“We’ll likely see some of these voucher-holding products hit the market much faster than usual, proving out this concept,” Mahajan said. “The agency may even open additional rounds of vouchers or broaden the criteria if the pilot is deemed successful.”
Another initiative in this vein is the FDA’s move to require just one pivotal trial—as opposed to the usual two—for new drug approvals. This one, however, has been met with caution. “Remember, the #1 mandate for the FDA is oversight of the safety of the American public,” Suvannavejh said.
While he acknowledged that accelerating clinical development and review timelines can lower the barrier to bringing new therapies to patients, he added, “I always worry about unintended consequences—and when it comes to the safety of Americans, I don’t think you want to gamble with that.”
‘Regulatory Carrots’ for Policy Alignment
The CNPV program, according to Mahajan, will be crucial to another emerging trend at the FDA that she sees continuing into 2026: Using the promise of speedy drug reviews to incentivize companies to adhere to certain national priorities of the Trump administration.
In fact, this is one thrust of the CNPV program, she said. In selecting the first batch of awardees in October, the FDA said it considered the potential of a given product “to address a major national priority,” such as meeting an unmet medical need, addressing a public health crisis or bolstering the domestic supply chain. Augmentin XR, for example, was selected for its ability to help boost the “domestic manufacturing of a common antibiotic,” according to the FDA’s announcement.
Another key CNPV priority that Mahajan singled out is drug pricing. While it’s not clear if a priority voucher has been granted because a company has promised to lower the cost of a product, the FDA said when rolling out a pilot run of the program in July that the agency could consider sponsors that bring their prices in line with President Donald Trump’s Most Favored Nation rule.
“If this policy continues, we might see FDA in 2026 effectively integrating pricing policy with regulatory carrots,” Mahajan said, adding that this will result in “a novel situation where the agency’s review speed is used as a bargaining chip for policy goals.”
Beyond pricing, Mahajan noted that Health Secretary Robert F. Kennedy, Jr. exerts an “unorthodox influence on FDA,” particularly given his vaccine skepticism and “aggressive stance” against pharmaceutical advertising. The agency in September sent out around 100 cease-and-desist letters warning companies to remove what it called deceptive ads in a move Kennedy at the time said will help “shut down the pipeline of deception” from the industry.
If Kennedy continues to exert this pressure, “it may so happen that HHS pushes FDA further in directions like [regulating] nutritional health claims, ‘natural’ products oversight, or even revisiting vaccine approvals,” Mahajan said.
More AI Integration Ahead
Also in 2025, the FDA took a leap forward in the use of artificial intelligence in its regulatory processes. Experts agree that this momentum will continue into 2026.
In June, for instance, the regulator introduced Elsa, its agency-wide generative AI assistant designed to help reviewers and investigators improve their efficiency. Elsa allows FDA employees to access troves of internal documents securely, enabling speedier turnaround times, according to its announcement. Agency staffers can also use Elsa to better prioritize their tasks.
Then, in December, the FDA rolled out agentic AI—models that emulate human problem-solving to help with complicated tasks such as pre-market reviews, post-market surveillance and inspections and compliance assessments.
Given staffing cuts at the agency and pressure from Makary to modernize its workflows, “The FDA is betting on technology to fill the gap,” Mahajan said, noting that agency leaders have made it clear that Elsa is just the start of its AI push. “By 2026, they will likely build on that momentum, possibly launching AI models for specific review decisions or expanding Elsa’s capabilities.”
At the same time, Mahajan said the FDA will need to address Elsa’s “teething issues.” Indeed, Elsa’s initial days were rough, prompting criticisms that its implementation had been rushed. Agency staffers told NBC News that Elsa provided incorrect or partially accurate information, adding that some of its core functions needed more work before being put to use.
However, Mahajan posited that once these growing pains are addressed, the FDA should be able to take the benefits of AI one step further, allowing the technology to handle increasing workloads despite lean staffing.
“We can anticipate the FDA to integrate AI into even more of its processes,” she said, including summarizing clinical data, comparing drug labels, triaging adverse event reports and even identifying inspection targets. These capabilities would lead to shorter review timelines, she noted.
Of course, biopharma companies are also increasingly turning to AI to streamline their processes, and Mahajan expects the FDA to respond with new guidance and rules regarding the use of these models, not just for drug development but also for manufacturing.
Guidance will certainly be welcomed by the industry in the coming year, especially after a chaotic 2025—and particularly as the industry enters a crucial period that could set it up for a comeback after years of funding woes.
“Just like with financial markets, the [biopharma] industry needs stability and a steady hand from FDA,” Suvannavejh said. “Taken all together, I think what we’ve seen from FDA this year has overall been a net negative.”