Andrew Kish, MSPPM
Director, Office of Program and Strategic Analysis
U.S. Food and Drug Administration
Center for Drug Evaluation and Research
10903 New Hampshire Avenue
Silver Spring, MD 20993
April 17, 2026
Dear Mr. Kish and the PDUFA Reauthorization Team,
Thank you for the opportunity to participate in the PDUFA Stakeholders meetings. As representatives of patient, consumer, health, and public interest organizations that do not receive pharmaceutical funding, we are providing this letter in advance of our final meeting to once again highlight our mutual priorities.
We appreciated the many presentations by FDA staff but were disappointed with the limited time allotted to ask clarifying questions and to discuss our priorities and concerns. The minutes of FDA’s meetings with industry were not detailed and generally did not reflect our concerns, particularly about our strong recommendations that performance goals include metrics that reflect safety and efficacy goals or the use of user fees for post-market staff and other resources. We request that you represent these stakeholder concerns in your final meetings with industry so they will be addressed in the commitment letter.
For example, FDA staff presented substantial information about pilot studies initiated under previous PDUFA commitments and plans to continue and expand them, but we were not asked what new pilot studies we’d like to see in PDUFA based on our experiences and expertise as stakeholders.
Broader representation in Patient Focused Drug Development
We also want to emphasize that we expressed concern that most of the patient groups that are involved in pilot programs, such as Patient Focused Drug Development, are financially supported by industry. Most of us were not invited to participate, and we know that patient groups who are not supported by industry funding often lack sufficient staff to participate in frequent meetings. Unfortunately, if only the industry-funded patient or consumer groups can afford to participate in pilot programs intended to incorporate patient viewpoints, the FDA needs to find ways to ensure that all types of patient and consumer groups are included to achieve representative engagement. A first step would be for the FDA to disclose industry funding for patient groups involved in these pilot programs, and for user fees to help support participation by nonprofit organizations that don’t get industry funding.
The following is a short list of our mutually shared priority issues that we raised, most of which were not mentioned as having been discussed in the FDA-Industry meeting minutes that the FDA shared online.
Public health indicators/measures
At least some performance measures should evaluate the public health impact of the PDUFA program and should therefore be included in the Commitment letter. User fees should support staff that improve the quality of FDA reviews, not just the speed. For example, we urge that these should include:
- How many of the recently considered drug approvals and rejections were based on the gold standard of at least 2 independent, randomized, blinded, controlled trials;
- How many trials included a sufficient number of representative demographic subgroups (and statistical power) to evaluate whether benefits outweigh the risks for males and females; racial and ethnic minorities; and age groups that include young adults (>18) and patients over 65;
- How many fast-track drug approvals resulted in recalls, warnings, withdrawals or other key safety issues within the following 5-10 years after approval?
- If confirmatory trials do not meet required outcome measures, how many of these drugs are still on the market and for how long? Were labels changed as a result?
Other performance measures that should be considered
- What proportion of drugs that the FDA approved or rejected were reviewed by an FDA Advisory Committee? The FDA always publicly releases FDA’s memoranda reviews of data for drugs publicly discussed by Advisory Committees, but even when there is no Advisory Committee, the same FDA reviews of data should be publicly available on FDA.gov.
- How many (number and proportion) of the approved drugs and biologics were reviewed via accelerated approval, priority vouchers, plausible mechanism pathway, and other fast track pathways, and what was the impact on submissions reviewed through the standard pathways? The FDA should include information about patient populations that would most benefit from these fast-tracked drugs.
- Clarify the distribution of FDA staff FTEs assigned to pre-market and post-market safety and efficacy work, including subject matter experts. How many staff are assigned to ensure that post-market confirmatory trials are done correctly, on time, and publicly disclosed?
Transparency
At Stakeholder meetings, we repeatedly requested but failed to receive specific information about financial/staffing resources needed by CDER and CBER to achieve their mission. We also asked how much of those resources have been from user fees and from appropriations. We were not given information about which activities the Centers consider the highest priorities, so were unable to share our views of those priorities. For example:
- How many staff supported by user fees vs. appropriation funding conduct facility inspections and what proportion of inspections are done as frequently as required?
- How many staff paid by user fees vs. appropriations review Direct-to-Consumer ads?
- Are user fees supporting staff to redact complete response letters and respond to nonprofit FOIA requests in a timely manner? Those are important priorities for us.
Other Issues
- Stakeholders raised the issue of clinical representation and lack of age-specific post-market data of underrepresented age populations, including young adults (>18) and patients over 65. FDA should ensure data collected from clinical trials and post-market surveillance is properly disaggregated by age subgroups and publicly reported (including on the Adverse Event Monitoring System (AEMS).
- Public stakeholders should be involved in the queries that Sentinel seeks to answer, and how the data are used to answer those queries. That information should be made public; if industry researchers are afforded data use opportunities, independent researchers should as well.
- We support the idea of reinstating user fees for previously submitted orphan waivers that now have non-orphan indications tacked on through supplement add-ons or expansion indications. FDA should also remove the previously granted market exclusivity provided under the initial waiver approval.
As you know, we have repeatedly requested that Stakeholders be included in the FDA-Industry PDUFA meetings or at least be able to watch the meetings remotely in real time or videos of those meetings. Stakeholder organizations should then have the opportunity to directly share their views in meetings with the FDA and industry. Instead, FDA staff have told us that Stakeholder meetings with the FDA are intended to provide us with sufficient input. Unfortunately, despite hours spent sharing our views at this meeting, there was limited evidence of the extent to which our priorities were discussed at the FDA-Industry negotiations. This letter is an additional effort to seek meaningful input prior to the commitment letter being finalized.
The undersigned organizations agree with the recommendations in this letter and will individually contact you with additional recommendations and concerns. Feel free to contact us at the emails above.
Sincerely,
Doctors for America
Generation Patient
National Center for Health Research
U.S. PIRG