‘A slow-moving catastrophe’: What we heard at the Biopharma Congress
At this week’s Biopharma Congress in Washington, former FDA officials and industry leaders gathered to discuss the agency’s current turmoil, touching on workforce cuts and potential reorganizations. A rare bright spot: The first in a new series of rare disease workshops is expected to be announced in the coming months.

At the Biopharma Congress this week, stakeholders weighed in on the state of the FDA and what to expect moving forward

• The in-person  Biopharma Congress meeting took place April 7, 2025, in Washington. The meeting was hosted by Prevision Policy and Friends of Cancer Research, with support from Merck and Amgen. Originally scheduled for Feb. 24, the meeting was moved in late January “due to the federal freeze on communication and event participation.”
• Only one current regulator took part: AMY COMSTOCK RICK, the head of the Rare Disease Innovation Hub. While FDA AI Policy Director TALA FAKHOURI was included in the agenda, she ultimately did not attend. This left a gap in the event’s session on using artificial intelligence in drug development. Still, industry representatives emphasized the need for clear guidance from the FDA to ensure regulatory certainty and trust these models across a variety of applications. This comes just a few weeks after a Food and Drug Law Institute event convened life sciences regulatory experts to review what’s next for AI policy at the FDA. [ Read full AgencyIQ analysis here.]

Reactions and impressions from former FDA executive leadership

• An all-star cast: The event’s first panel featured former FDA Acting Commissioner JANET WOODCOCK, former Center for Devices and Radiological Health Director JEFF SHUREN and former FDA regulatory counsel FRANK SASINOWSKI. A later reforms-focused discussion spotlighted former FDA Principal Deputy Commissioner JOSHUA SHARFSTEIN.
• Staffing reductions: As AgencyIQ has  discussed extensively, the second Trump administration is effectuating an overhaul of the federal workforce, implementing policies that reduce the overall size of the federal governmental workforce. [ See AgencyIQ’s running list of which FDA staff affected by RIFs and layoffs here.] Woodcock described the decision to spare reviewers and inspectors from the reductions-in-force as a “naive conception,” likening it to downsizing a hospital by keeping doctors but firing nurses, lab techs, phlebotomists, and others. From Shuren’s perspective, attrition was a significant issue at the agency prior to these actions. Sharfstein later weighed in, saying that previous staff cuts have been the result of budget cuts. This time, he said, “There was no budget cut, right? And so, there’s literally no external reason why this needs to happen.”
• Downstream reprioritization: With reduced capacity, the leaders agreed that the FDA will need to prioritize application reviews and safety assessments. However, activities such as developing guidance and providing advice to sponsors will be lower on the list. Woodcock said the impact will not be immediate but, rather, “a slow-moving catastrophe.” The removal of key leadership will pose further challenges because of the role they play in settling disagreements between different types of review groups. Absence of this layer of technical management will likely lead to bottlenecks, she said.
• Reorganizations: In early April,  Endpoints News first reported that HHS had distributed a memo to certain lawmakers’ offices that seemed to reflect the content of Phase 2 Agency RIF and Restructuring Plans, with new organization charts for agencies focused on consolidation. In short, the memo indicates that FDA’s six head offices and 15 center divisions could be collapsed into five “shared services” offices. [ Read AgencyIQ analysis here.] The former FDA officials reacted to this proposal, emphasizing the importance of highly specialized expertise. Sharfstein said different products have different science and laws, asking, “What could possibly go wrong?”
• New policy priorities: New FDA Commissioner MARTIN MAKARY has offered very limited information on concrete policy issues he plans to address during his tenure. The former FDA leaders acknowledged this peculiarity. Sasinowski mentioned potential changes to the agency’s positions on evidence development, specifically referencing views of new special assistant to the commissioner, TRACY BETH HØEG. As  POLITICO reported, Høeg is a sports and physical medicine doctor who has expressed significant concerns about vaccine policy in the U.S. She previously served as an advisor to the JOSEPH LADAPO, Florida’s surgeon general.
• Sasinowski flagged that Høeg may have “came out with a statement opposed to the use of accelerated approval the way it’s been used for cancers.” Based on AgencyIQ’s research, this may refer to Høeg’s 2023 blog post on “FDA shortcomings” that criticizes use of surrogate “endpoints that may or may not translate into true health benefits.” Concerns regarding the conversion of products that received accelerated approval to traditional approval have surfaced in recent years. In addition, the FDA received multiple new authorities in 2022 via the  Food and Drug Omnibus Reform Act to modernize accelerated approval and quickly withdraw products that do not ultimately confirm benefit. Key questions remain: Will Høeg look to reform the agency’s accelerated approval paradigm? If so, will this involve stricter approval standards upstream, stricter confirmatory study enforcement downstream, or both?
• User fees: The leaders briefly touched on discussions regarding user fee program reauthorizations, including the notion that industry should push to “flatline these for the next couple of years until the climate gets a little bit more friendly.” AgencyIQ has  previously flagged that new leadership may look to reform – or even terminate – the paradigm given its stated general concerns about undue “industry influence.” On April 3, 2025, AgencyIQ wrote about how recent layoffs place the FDA at risk of violating statutory triggers that could cause the agency’s user fee programs to collapse in the coming months. [ See our extensive analysis here.] At the Biopharma Congress, Woodcock concurred that these triggers could pose an existential threat. Further, she commented that the finance professionals that could address this issue may have been affected by the RIFs.

A dispatch from FDA’s Rare Disease Innovation Hub

• Catching up: Launched in July 2024, the RDIH intends to have a coordinating role in leveraging rare disease expertise across the agency’s product centers. In addition, the hub will serve as a “single point of connection and engagement with the rare disease community,” the agency said in a July announcement. In November, the agency  hired Amy Comstock Rick as the hub’s director of strategic coalitions, and RDIH  published a strategic agenda for 2025 in mid-January, in the waning days of the Biden administration.  The agenda lists “Patient and Patient Organization Engagement in the Development and Review of Drugs for Rare Diseases” as an action item under its regulatory science goals. The text describes Patient-Focused Drug Development meetings and acknowledges “strong interest among patient and disease organizations and many drug developers for deeper and more substantive involvement of patients.”
• Rick sees longevity for RDIH amid the turmoil at FDA. From her perspective, the hub’s stated goals of “efficiency, centralization, coordination” align with the immediate priorities of the new administration. She emphasized that rare disease development is “not a partisan issue.” She said, “So my job right now is to make sure the hub stays in place, doing kind of some background prep work, so that when things normalize and settle a little bit, the hub is already down the path of coordination.” Rick did not address how the hub would function should the agency be reorganized into shared services offices as proposed.
• At the Biopharma Congress, Rick offered some points of clarification and updates. For example, she made clear that the hub does not get involved in matters related to any individual product or product application and instead focuses on big-picture processes. This is a key difference from the agency’s similarly situated Oncology Center of Excellence.
• Workshop coming soon: The RDIH strategic agenda published early this year previewed the new Rare disease Innovation, Science, and Exploration, or RISE, workshop series. These public events intend to address “cross-cutting or common issues and will not be focused on any specific product under review by the Agency.” The intention is to provide a forum for stakeholder interaction, including community members, industry, academia and regulators. According to Rick, plans for the first RISE workshop are in the works, in collaboration with the Duke-Margolis Center for Health Policy. While Rick said it would be “irresponsible to make an absolute commitment right now of what would happen,” she informed the group that the event is slated to be announced in May and take place in June. The announcement will be accompanied by a Federal Register docket to source proposals for future workshops.

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