PDUFA VIII: Negotiations touch on patient experience data, reserve funding, fee waivers, and more

Last month, the US Food and Drug Administration (FDA) and the pharmaceutical industry concluded a series of subgroup meetings toward the reauthorizing the Prescription Drug User Fee Program (PDUFA VII).

The meetings explored reserve funding under the PDUFA model, the rare-disease development pilot, patient experience data (PED), and whether to provide financial incentives to firms that conduct early-stage trials in the US.

The PDUFA minutes indicate areas of agreement and disagreement with the agency. The current version of the PDUFA program is set to expire in September 2027. Negotiations for reauthorization began in late 2025.

Progress reported on revising PED language

During a meeting on 17 February, the FDA and industry premarket subgroup discussed the creation of draft commitment letter language regarding PED.

“Industry proposed revisions to the Enhancing Transparency and Consistency Related to PED draft commitment letter language,” according to the meeting minutes. “FDA asked for clarification on Industry’s proposed revisions, which Industry addressed. FDA agreed to review Industry’s proposed revisions and propose revised draft commitment letter language in a future meeting.”

Industry’s goal is to increase the use of PED for regulatory decision making and in labeling. Patient experience data is collected by patients, caregivers, advocacy groups and manufacturers, with the goal of providing information about patients’ experiences with a disease or condition.

FDA asked to elaborate on ORA TRR model

The finance subgroup also met on 17 February, during which, industry representatives requested that FDA provide more details about its funding reserves incorporated in the Operating Adjustment Tracking Reserving Reporting (ORA TRR) processes in the PDUFA VIII commitment language. This mechanism is designed to monitor any underspending on personnel compensation and benefits, ensuring that funds are specifically allocated for reviewer staffing.

According to the minutes, “Industry requested FDA provide an overview of the mechanics of the ORA TRR model to ensure knowledge across all parties. FDA detailed how payroll funds would be captured in the operating reserve, then held in a special set-aside for reviewer staffing. FDA explained how it proposed to derive the relevant payroll figure (to compare against the baseline FY 2024 PDUFA payroll).”

The industry sought confirmation that the set-aside reserve could only be used for payroll expenses, which the FDA confirmed. The subgroup acknowledged that these reserved funds could be utilized for payroll during a shutdown to maintain operations. Yet FDA clarified that once an appropriation was made to the Agency, the reserve would be replenished. The FDA reiterated its preference for a payroll-based approach to measuring restaffing targets, stating that this method would be much easier to plan, monitor, track, and interpret.

In other areas, the industry expressed concerns about the FY 2025 PDUFA financial report submitted to Congress. They requested clarification on the reported increases in PDUFA payroll from FY 2024 to FY 2025 in light of the reductions in force (RIFs) and the “significant” number of voluntary departures the FDA experienced during FY 2025.

FDA said the goals for the next meeting on 19 February “are to provide further clarifications on the FY 2025 PDUFA Financial Report to Congress and to address outstanding Industry questions on the potential financial framework package.”

Progress reported on RDEA program

On 12 February, the FDA presented its response to industry feedback during the premarket subgroup meeting regarding the Rare Disease Endpoint Advancement (RDEA) pilot program.

“FDA presented an updated counterproposal for the RDEA pilot program. FDA stated it is aligned with the goal of moving RDEA towards Type C, RDEA-focused meetings starting in Fiscal Year (FY) 2030 without a need for a formal assessment to help inform the transition. FDA and Industry confirmed that once Type C, RDEA-focused meetings were implemented, sponsors would specify that they are interested in an RDEA-focused meeting in their Type C meeting request cover letter. After further discussion, Industry agreed to respond to FDA’s counterproposal in detail at a future meeting,” the minutes stated.

Industry expresses concern about ‘America First’ initiative

During the meeting of the steering committee subgroup on 12 February, the agency presented a revised fee incentive proposal that would adjust fees for drug developers that conduct their Phase 1 clinical development in the US. This proposal is known as the “America First” fee incentive.

In response, industry reiterated its concerns that “PDUFA may not be the most appropriate mechanism for incentivizing clinical development in the U.S.”

Industry also expressed concern about the feasibility and “potential unintended consequences” of the proposal. Industry then had questions about how FDA would confirm eligibility, the amount of the fee differential and how the fee amounts would be determined, and whether statutory changes would be needed to implement the fee differential.

Meeting minutes

PDUFA VIII: Negotiations touch on patient experience data, reserve funding, fee waivers, and more

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