Makary suggests lower PDUFA fees as reauthorization process begins

The US Food and Drug Administration (FDA) kicked off the reauthorization process for the eighth iteration the Prescription Drug User Fee Act (PDUFA VIII) on Monday, with FDA Commissioner Marty Makary saying he’d like to see lower fees paid by industry this time around.

“I’d like to see lower user fees. It’d be a reduced barrier for small companies and individual inventors and people in academics that may be trying to understand this process, including the capital requirement,” Makary said, though he contended that, “First and foremost, before that goal, a program that works very well,” is needed.

For FY 2025, the PDUFA fee for an application requiring clinical data is $4.3 million, while the annual program fee is just over $400,000. About half of FDA’s annual budget comes from its various user fee programs, most of which must be renewed before 30 September 2027.

Monday’s meeting was the first step of a lengthy negotiations process with industry to secure a commitment letter for the next PDUFA program. The commitment letter will provide the necessary information for Congress to pass legislation to reauthorize the program for another five years.

At the meeting, FDA officials stressed that the user fee program is a priority for the agency. “User fees are critically important, and I want to just briefly emphasize our commitment to user fees,” Makary said. He added that “we want your input on how to make it better.”

Andrew Kish, director of the Office of Program and Strategic Analysis within the Office of Strategic Programs at the agency’s drug center, noted that, “Before PDUFA, things were not ideal. We had backlogs of overdue applications; we had long review times – we just didn’t have the staff to do the work.”

Kish said that user fees from industry are critical to the success of PDUFA. In 1993, PDUFA user fees funded 7% of the program while in 2024, user fee funded 78% of the program. It grew from a $135 million program in 1992 to a $1.7 billion program in 2024.

Since 2020, Kish noted, over half of filed (new drug applications (NDAs) and biologics license applications (BLAs) have had at least one special designation such as priority review, orphan, fast track, breakthrough therapy, or accelerated approval.

Industry backing

The Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Innovation Organization (BIO) – the two lobby groups that will represent industry in the negotiations – praised the success of the user fee program.

Annetta Beauregard, senior vice president for science and regulatory affairs at BIO, said that BIO “recognizes and appreciates Commissioner Makary's commitment to upholding the gold standard of trusted science, increasing transparency, and applying common-sense decision making."

She added that BIO would like to see further improvements to communication and transparency in PDUFA VIII. “How FDA communicates the basis for its decisions will enable greater insights into how America's biotechnology leaders are using the latest science to improve public health,” she said.

Kristy Lupejkis, vice president and chief of staff for regulatory science and advocacy at PhRMA, said PDUFA “supports more timely patient access to safe and effective new medicines.”

Under PDUFA VIII, Lupejkis said PhRMA hopes to see enhancements to “help ensure US drug development and review processes are consistent, predictable, transparent, and rooted in sound scientific principles.”

Concerns about FDA’s approval standards

One patient organization representative expressed the concern about FDA’s accelerated approval program, which was codified under previous PDUFA reauthorization cycles and was updated in the most recent user fee package.

Kaylin Bower, founder of On a Mission for Multiple Sclerosis an MS advocacy organization, discussed the recent deaths of two patients that appear to be linked to Sarepta’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl), which is used to treat non-ambulatory pediatric male patients with Duchenne muscular dystrophy (DMD).

“There is concern in the patient advocacy community that some provisions connected to PDUFA, such as accelerated approval, appear to have been possibly lowered the bar with the substantial evidence of effectiveness requirements,” she said.

PDUFA VII, which spans FY 2023-2027, includes goals for improving the consistency and predictability of post-marketing requirements (PMRs) for drugs approved under the accelerated approval pathway.

While members of the review team wanted to reject the therapy, the decision was reportedly overruled by former Center for Biologics Evaluation and Research (CBER) Director Peter Marks.

“Will future FDA accelerated approval decisions be like Elevidys, and could this possibly have the same outcomes?” she asked.

FDA

Makary suggests lower PDUFA fees as reauthorization process begins

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