The EveryLife Foundation for Rare Disease and our rare disease community have been closely engaged in the Prescription Drug User Fee Act (PDUFA) reauthorization for FY 2028–2032. This is a critical next step in the process.
What are User Fees?
Every five years, PDUFA negotiations shape how the FDA reviews new drugs and biologics, allowing the FDA to collect “user fees” from drug manufacturers when they submit an application to the FDA.
The Prescription Drug User Fee Act (PDUFA) was initially passed by Congress in 1992 in an effort to provide supplementary resources to the FDA that could solve for specific bottlenecks in regulatory review and ensure review timelines and regulatory processes reflected the urgency of the patient community. Every five years, a new review cycle occurs in which ‘user fees’ and review-timeline commitments from the agency are renegotiated by the FDA and the regulated industries, with opportunities for input from public stakeholders.
In exchange for the supplemental resource of the user fees, the FDA agrees to the PDUFA pre-negotiated terms aimed at supporting regulatory review including target review timelines and staffing levels commitments related to how the review process works such as new or pilot programs.
Why does this matter for the rare disease community?
- Previous PDUFA reauthorizations have yielded the creation of landmark initiatives that have transformed the rare disease therapy development process, such as the Patient Focused Drug Development initiative, the establishment of the Benefit -Risk Framework, the application of Accelerated Approval to rare disease and much more. Learn more about PDUFA here.
- PDUFA is an important ‘legislative vehicle’ for our community and is considered to be ‘must pass’ legislation. This is an exciting opportunity for our rare disease community to strengthen our regulatory review processes.
What has happened so far?
- To date, the negotiation process has included negotiations between FDA and the regulated trade industry (BIO and PhRMA), and meeting minutes were made publicly available on FDA’s website.)
- Patient organizations were invited to join monthly public stakeholder meetings with FDA throughout the negotiations. Our rare disease community was well represented, including participation from the EveryLife Foundation.
- Over the past 18 months, the EveryLife Foundation has convened an Ad-Hoc Working Group of Community Congress coalition members, a network of rare disease patient advocacy groups, partners, and other key stakeholders, to understand rare disease community priorities for PDUFA VIII. We’ve brought those priorities to the FDA in their monthly stakeholder consultation meetings throughout negotiations to ensure rare patient and caregiver perspectives were a central part of considerations.
- The draft PDUFA Commitment letter reflects the agreement between FDA and the pharmaceutical industry related to performance goals, review timelines, and process or program enhancements yielding from the new user fees cycle.
What happens now?
- Our policy team is reviewing the draft letter in full and will share highlights with the community.
What happens next?
- There will be a public meeting at the FDA to discuss the draft goals letter on September 16, 2026 and an opportunity to submit written comments.
- The letter will be finalized by the FDA and sent to Congress in January.
- Congress will create legislation that includes the details from the final goals letter. The legislation often includes several other regulatory policy priorities related to how the FDA reviews new therapies and engages with the community.
- The EveryLife Foundation will share opportunities for engagement around legislative priorities that have been identified by our community in the coming weeks.
- Check out our early priorities here and stay tuned for more insights on how the draft goal letter is advancing rare disease therapy development.
- If your organization is developing PDUFA legislative priorities, please reach out to Dylan Simon at dsimon@rareadvocates.org from our policy team so we can collaborate.
Stay connected and engaged! This will be an exciting couple of months of momentum and progress!
