The FDA gathers Wednesday to hear what industry stakeholders and the public want included in the latest iteration of the Prescription Drug User Fee Act that sets the agency’s priorities for fiscal years 2028–2032.
The FDA will meet Wednesday with patient groups and other stakeholders to discuss recommendations for reauthorizing the Prescription Drug User Fee Act. The agency is homing in on increasing regulatory transparency and proactive monitoring of chemistry, manufacturing and controls risk, while the public remains laser focused on rare disease.
By Sept. 30, 2027, the U.S. Congress is expected to authorize the eighth iteration of PDUFA, which will set the FDA’s performance objectives, fee structures and program enhancements for fiscal years 2028-2032. Implemented by Congress in 1992, PDUFA allows the agency to collect fees from drug manufacturers to fund the review of new drug applications and must be reauthorized every five years.
The user fees prescripted by PDUFA enable the FDA to complete drug review work but they by no means guarantee a favorable decision, Steven Grossman, president of policy and regulatory consulting firm HPS Group, told BioSpace. “It buys timely reviews and more FDA reviewers. It does not buy yes,” he said.
Wednesday’s public meeting is expected to focus on proposed enhancements to pre-market drug review, post-market safety and chemistry, manufacturing and controls (CMC) risk, according to the public draft agenda—all hot button issues that continue to draw consternation among biopharma companies and patients alike.
Pre-market drug review processes have tripped up rare disease biotechs in particular, with the FDA in the past two years reversing guidance given under previous leadership. After the departure of former Commissioner Marty Makary and Center for Biologics Evaluation and Research (CBER) director Vinay Prasad this spring, some recommendations changed again.
Regulating investigational therapies for rare diseases—which the agency under Makary singled out as a priority—is complex, with often miniscule patient numbers making traditional clinical trial designs impossible. The International Pemphigus & Pemphigoid Foundation (IPPF)—which represents people living with rare autoimmune blistering diseases—alluded to this in a public comment submitted to the FDA prior to Wednesday’s forum.
“We encourage FDA to use PDUFA VIII to advance a more patient-centered, scientifically rigorous, and fit-for-purpose rare-disease development ecosystem,” IPPF wrote.
In another comment, Christopher Freeman “encourage[d] FDA to consider one additional enhancement: establishing a sustained, cross-functional Rare Disease Regulatory Science and Review capability within the existing FDA structure.”
Freeman, a self-described caregiver of a child with a progressive rare genetic disease, notes that PDUFA VIII “already proposes important investments in these areas,” through initiatives such as the Rare Disease Endpoint Advancement, Complex Innovative Trial Design and Model-Informed Drug Development. “I encourage FDA to connect these efforts through a durable capability that can provide specialized expertise early in development and during regulatory review,” Freeman added.
Indeed, in an updated memo regarding Heidi Overton’s nomination as FDA commissioner, health policy consulting firm Aviva Strategies wrote on Friday that “FDA’s Rare Disease Innovation Hub is also working to improve alignment between CDER [Center for Drug Evaluation and Research] and CBER, a longstanding issue for developers.
“For rare disease stakeholders, the key watchpoint is therefore continuity: whether Overton preserves the current movement toward flexible evidence standards, individualized therapies, stronger CDER-CBER coordination, and greater predictability in reviews,” the firm added.
The FDA in its PDUFA VIII commitment letter also touches on rare disease drug development.
“To ensure that new and innovative products are developed and available to patients in a timely manner, FDA will continue to ensure the sustained success of the breakthrough therapy program, continue early consultations between FDA and sponsors on the use of new surrogate endpoints as the primary basis for product approval, advance rare disease drug development, and advance the development of combination products,” according to the document, which was published on August 14.
More key items: US trials, CMC
Another item that has drawn attention prior to the meeting is an FDA proposal that would offer a 50% reduction in the new drug application (NDA) fee for investigational products with early-stage trials conducted in the U.S—a roughly $2.3 million in savings at current rates, according to an opinion article written by Ovid Therapeutics CEO Jeremy Levin and published last week in BioSpace.
Levin, however, argued that this proposal is unlikely to achieve the intended result.
“I support the objective but question the means,” Levin wrote. “This incentive is being offered near the end of development—at the time of an approval filing—to influence a decision that was made years earlier about where to conduct the first clinical trial.”
This proposal dovetails with Operation TrialBlazer, an HHS-wide initiative rolled out in June aiming to expedite early clinical development timelines to make the U.S. more competitive as an ideal location to run these trials.
Another key theme for the FDA under Health Secretary Robert F. Kennedy Jr. is transparency, which features in the FDA’s commitment letter.
With a new program aimed at enhancing review transparency and communication for NDAs and biologics license applications (BLAs), the FDA intends to “promote the efficiency and effectiveness of the first cycle review process and minimize the number of review cycles necessary for approval, ensuring that patients have timely access to safe, effective, and high quality new drugs and biologics,” according to the document.
Grossman emphasized the importance of transparency in regulatory reviews.
“FDA is the world’s most credible drug regulatory agency because it prides itself on showing that its product reviews are scientifically thorough and that all viewpoints have been considered,” Grossman said. “Understandably, industry values this and it is always a theme in PDUFA agreements.”
The FDA has also recently prioritized a crackdown on CMC risk.
Inspection access is not “an administrative footnote,” leaders of key FDA divisions, including CDER and CBER, wrote in an editorial earlier this month vowing to expand oversight of foreign clinical trial sites.
Noting that manufacturing facility deficiencies can lead to complete response letters, FDA in its PDUFA VIII commitment letter furthers this initiative with a proposed “risk-based life cycle approach,” in which the agency and industry “will focus on enhancing CMC communications during drug development and application review.”
Wednesday’s meeting is one of the last steps before FDA finalizes the PDUFA VIII work plans and sends them, along with legislation language, to Congress on Jan. 15, 2027, Grossman said.
“Because PDUFA is viewed as must-pass legislation, there will be the core user fee renewal legislation and potentially dozens of unrelated FDA provisions intended to make the agency better,” he told BioSpace. “Ideally—and unlike in 2022—those can be resolved quickly and not force FDA and industry to wait until September 30, 2027, to be assured PDUFA will be renewed.”
