Yale experts urge reforming – not gutting – PDUFA

The US Food and Drug Administration (FDA)’s industry user fee is critical and could be reformed to boost public engagement and restore trust, Yale University health policy experts wrote in a 16 August perspective piece published in the New England Journal of Medicine.

Industry-paid user fees accounted for almost half of FDA’s $47 billion budget in fiscal year 2024, 69% of the agency’s budget for small molecule drugs, and 53% of the budget for biologics, noted Therese Ziaks, postgraduate associate, and colleagues at the Yale Collaboration for Regulatory Rigor, Integrity, and Transparency (CRRIT).

“User fees remain critical for supporting the FDA’s key regulatory operations,” Ziaks and colleagues wrote. “Reforms could be considered as part of the ongoing reauthorization process to enhance public participation.”

Discussions about the next iteration of the user fee program (PDUFA VIII) are underway, but there are big hurdles ahead. Health and Human Services Secretary Robert F. Kennedy has spoken out against user fees, and he’s far from alone in the critique.

“Amid bipartisan calls for program reform, PDUFA’s future remains unclear,” Ziaks and colleagues wrote. “Implementing strategic reforms could allow the agency to advance its mission of enabling access to safe and effective drugs while also supporting patient-centered regulation and increasing public trust.”

In mid-July, FDA Commissioner Marty Makary said he favored lowering user fees, which could hamper the agency’s ability to approve products quickly, especially after large staff cuts (RELATED: Makary suggests lower PDUFA fees as reauthorization process begins, Regulatory Focus 16 August 2025).

“With large-scale layoffs at the FDA and budget proposals calling for substantial cuts in appropriations that would lead to further staff reductions, user fees are necessary to ensure sufficient capacity for the agency’s regulatory activities,” Ziaks and colleagues wrote.

The CRRIT researchers noted that pre-PDUFA, the average review time for a new drug application (NDA) was 29 months, and, consequently, treatment for life-threatening diseases like HIV/AIDS languished under review for years.

“The FDA faced significant backlogs in reviewing NDAs, creating uncertainty about when drugs would reach patients,” the authors noted.

Under PDUFA, review times are 12 months for standard and six months for priority applications. The Yale researchers see room to improve the existing program through changes to the way reauthorization discussions are conducted. The current PDUFA regime expires at the end of September 2027, and the FDA has begun soliciting feedback to inform PDUFA VIII. The NEJM article highlighted practical ways the process could be changed to enhance public participation and transparency.

“Although recent PDUFA statutes, including the current legislation, call for monthly meetings with public stakeholders, only six such meetings were held over the more than 1-year negotiation period for PDUFA VII,” Ziaks and colleagues wrote.

Typically, FDA negotiates in private with industry representatives, but it doesn’t have to be that way. Ziaks and colleagues suggested that the FDA could include public stakeholders in these talks instead of engaging them separately and publicly post responses to comments on draft commitment letters.

“Increased transparency could cause delays in negotiations, but delays have also occurred in closed-door negotiation formats,” the authors wrote.

The authors also advised greater scrutiny and transparency about drugs approved through accelerated review programs, more attention to outcomes that matter to patients, such as improved function rather than surrogate measures, and greater stringency on the completion of postmarketing studies.

Ziaks, et al.

Yale experts urge reforming – not gutting – PDUFA

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