FDA Official Criticizes Advisory Committee Experts
An FDA official discussing an ongoing dispute this week with UniQure over its gene therapy for Huntington’s disease (see story) also raised concerns with outside experts who sit on agency advisory committees. The official, who spoke on condition of anonymity, criticized the advisory committees, suggesting some outside experts “do not do their homework” and may have financial conflicts of interest. The remarks came after the official was asked to respond to comments from prominent researchers and former regulators questioning the agency’s stance on the therapy. Among the critics is former CDER director Janet Woodcock, who called the decision “truly evil” in a recent interview with The New York Times..
The unusually blunt FDA media briefing reflects rising tension between the agency, drug developers and outside experts over several recent regulatory decisions that companies say represent shifting expectations for drug approval. In recent months, several companies have argued that the agency has altered expectations late in the development process. Last month, the agency faced criticism for initially refusing to review an application from Moderna for an mRNA influenza vaccine before ultimately agreeing to review the filing after revisions.
Drug developers including Atara Biotherapeutics, Pierre Fabre Pharmaceuticals and Regenxbio have also raised concerns about regulatory decisions affecting rare disease therapies. The agency has defended its actions, saying rigorous evidence — particularly randomized clinical trials — remains essential for determining whether new treatments are safe and effective.
“The FDA actions are not backing up the positive rhetoric about accelerating development of cell and gene therapies,” Alliance for Regenerative Medicine vice president Stephen Majors told the Times. “And over the past few months, surprising regulatory U-turns on promising medicines for rare diseases are frustrating patients and confusing the companies trying to bring them to the market.” He called for the agency to hold more public meetings of advisory committees on proposed drugs “to transparently review the evidence.”
Last month, a new analysis published in JAMA Health Forum examined FDA’s quiet but significant shift in how it seeks outside scientific advice, raising legal and credibility concerns about the agency’s decision-making process. In the article, C. Joseph Ross Daval and Aaron S. Kesselheim contend that FDA has scaled back its use of longstanding advisory committees while convening a new series of “expert panels” that they say lack the procedural safeguards and independence historically associated with formal advisory bodies. Daval and Kesselheim are both part of Harvard Medical School’s Program On Regulation, Therapeutics, and Law.
Over the past year, FDA has convened fewer traditional advisory committee meetings than at any recent point in its history. Instead, the agency has held five expert panels addressing topics including antidepressant use in pregnancy, hormone therapy and menopause, talc additives, testosterone therapy in men, and infant formula.
According to the authors, these panels differ in key structural and procedural respects from standing advisory committees. Unlike advisory committees — whose members typically serve ongoing terms, deliberate collectively, and vote on specific regulatory questions — the expert panels consist of individuals invited for a single session. Panelists offer short, sequential presentations and are not asked to deliberate as a group or issue formal recommendations.
The authors raise the possibility that the new panels may implicate the Federal Advisory Committee Act (FACA), which governs how federal agencies obtain advice from external groups. Potential plaintiffs could challenge panel composition or argue that FDA has unlawfully curtailed required advisory committee meetings, particularly in areas such as new drug approvals where statutes mandate regular consultation, they contend.
Beyond legal risks, the article warns of reputational consequences. Advisory committees have long served not only as a source of scientific expertise but also as a public demonstration of the FDA’s commitment to independent, evidence-based decision-making.
The article concludes that the agency’s evolving approach signals a broader tension between scientific process and political influence, raising questions about whether FDA is prioritizing constituency signaling over the procedural tools traditionally used to safeguard regulatory integrity.