PDUFA letter stresses stability, transparency, maturity, says BIO negotiator

An agreement designed to shape the future of drug approval recommends new efficiencies while leaning into the existing strengths of a system that makes America the world leader in biotech.

The agreement, the Prescription Drug User Fee Act (PDUFA) VIII commitment letter, was released on Aug. 13. It was negotiated by the Food and Drug Administration (FDA) and representatives of the biopharmaceutical industry, including the industry’s association, the Biotechnology Innovation Organization (BIO).

Renewed by Congress every five years since 1992, PDUFA allows the FDA to collect user fees from biopharmaceutical companies to support the review of new therapies. These resources fund FDA’s scientific staff, modernize review systems, and strengthen drug safety monitoring.

The commitment letter is intended to set goals for the new PDUFA legislation, which will guide FDA’s review process between 2028-32.

“This commitment letter achieves a focus on stability, a focus on transparency, and a focus on maturity for the FDA,” said Steve Berman, BIO VP for Science and Regulatory Affairs Strategy, who was involved in negotiating the letter. “All three of those themes are reflected in the details of the document.”

Stability

“On the stability side, we address all of the things that industry relies on from the FDA: Can we get our meetings on time? Can we get our reviews done in the time that we expected?” Berman said. “All of that is maintained. Despite all the changes of 2025, for the future of 2028-2032, that stability remains in place.”

Stability is also emphasized in the goals set for financing, according to Berman. While the commitment letter recognizes that FDA needs to rebuild its staff, it does not call for a costly expansion that would require a much higher industry contribution.

“We’ve set FDA up on a path towards making sure that it has every dollar that it needs for all of the staff that it needs, but not $1 more,” he said. “The growth of the program would be constrained in a way that it hasn’t been in the past, so the fees are more stable than they have been in the past.”

Transparency

Improved communication between FDA and applicants can help to clear questions efficiently and keep new drug or biologic applications moving more quickly. To this end, the commitment letter encourages changes to refine the use of written response only (WRO) communication. It seeks to increase opportunities for in-person meetings and to enable multi-division meetings for more efficient discussions about multi-indication products.

“One thing that we’ve noticed over time is that there are many applications that are ultimately approved but aren’t approved on the first cycle,” Berman said. “That tells us that there are drugs that the FDA determines have a positive benefit-risk ratio—a good safety and efficacy profile—but for whatever reason, FDA doesn’t approve them on the first cycle, which means patients are delayed in accessing those innovations.”

The commitment letter calls for having a third party investigate what is happening during first-cycle review, to reduce unnecessary delays during this stage of the process.

Many of the questions coming from FDA during first-cycle review involve the Chemistry, Manufacturing, and Controls (CMC) segment of the application, Berman said.

The commitment letter recommends two new meeting types to address this challenge.

“The first of those is an opportunity to meet with FDA, even before you submit your New Drug Application (NDA) or Biologics License Application (BLA), to discuss your manufacturing with them,” Berman explained. “And then the second one of those is while FDA is reviewing the application: If they go and inspect a facility and find something there that might otherwise lead to a first cycle Complete Response Letter (CRL), there’s an opportunity to discuss and to potentially mitigate any of those findings and get those drugs to patients sooner.”

Maturity, and next steps

Using the latest advances in regulatory science, the FDA has developed many innovative methods for improving the speed and accuracy of its review processes. Several of these approaches have been tested as pilot projects and now have matured to where they are ready to become standard practice, according to Berman.

“We’re able take a look at all of those successes from past cycles and say, ‘It’s great that these pilots have been successful, but they’ve been sort of limited in size and scope,’ ” he said. “It’s time for us to move beyond pilots and into regular regulatory review practice, and that’s what PDUFA VIII does.”

The next step in PDUFA VIII approval is discussion in a hybrid public meeting on Sept. 16. Following any adjustments to the commitment letter based on the public discussion, Congress will review the recommendations and weigh reauthorization of PDUFA for fiscal years 2028-32. A vote on PDUFA VIII is expected next year.

Read more:

• The PDUFA commitment letter
• BIO’s statement on the commitment letter
• BIO’s PDUFA resources

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