As China’s clinical research sector expands, the U.S. is looking to keep pace. The upcoming Prescription Drug User Fee Act reauthorization could give lawmakers an opportunity to streamline domestic trials. The FDA will discuss the proposed agreement at a public meeting Wednesday.
The government has announced measures intended to streamline processes and speed investigational new drug reviews through Operation TrialBlazer, but additional reforms through reauthorization legislation could further shorten the time needed to begin early-stage human trials.
PDUFA, first implemented in 1992, is reauthorized every five years, and the most recent round kicked off last year. The program collects application and annual program fees from drugmakers to help fund the FDA’s review of prescription drugs and biological products, subject to exemptions and waivers.
The reauthorization follows a somewhat unusual process, starting with industry representatives and the FDA hammering out an updated fee agreement. The HHS secretary must submit the plan, including funding levels and performance goals, to congressional committees by Jan. 15, 2027. Congress must then pass legislation renewing the program and can include additional policy reforms.
“In terms of the FDA and industry, I think negotiations have basically concluded,” he said. Public feedback at the scheduled Sept. 16 FDA meeting may lead to modifications.
Despite substantial interest in clinical trial reforms, the negotiated agreement largely leaves that issue to Congress, said David H. Dorsey, a strategic adviser at Brownstein Hyatt Farber Schreck, in an email. One exception is a proposed 50% application-fee reduction for drugs whose applications include data from at least one U.S.-anchored phase 1 trial initiated after Oct. 1, 2027.
“I expect this incentive will be insufficient to address the cost and other advantages of running clinical trials outside the U.S., so it will be important for Congress to make meaningful reforms on this issue,” Dorsey said.
This puts the ball in Congress’s territory, where Rep. Jake Auchincloss, D-Mass., is already working with Rep. Morgan Griffith, R-Va., chair of the Energy and Commerce Committee’s health subcommittee, and the Trump administration to develop the proposal, Auchincloss told BioCentury. The aim is to gather enough support to pass it as part of the reauthorization package before the current user-fee authority expires at the end of September 2027.
The proposed Cures in Care Initiative could integrate clinical research into routine healthcare, turning hospitals and clinics into research sites. The broader proposal would also establish pilot programs modeled on Australia’s approach, allowing third parties to oversee lower-risk phase 1 trials to speed up trial initiations.
The proposal is designed to complement Operation TrialBlazer, which HHS announced in June, to expedite IND timelines and move drugs more quickly into human trials. HHS also said in an earlier email to PharmaVoice that the agency is taking steps to clarify phase 1 expectations and support more flexible early-stage trial designs.
But whether Congress will take steps to reform clinical trials isn’t the only outstanding question related to the PDUFA reauthorization, Dorsey said. Another is whether the FDA is operationally sound after losing a substantial number of staff members, including departures of high-level staff. The proposed agreement would reserve funds for hiring and retaining drug-review staff, although funding alone does not guarantee the FDA can fill those positions.
“I think the challenge will be whether the FDA can follow through on its commitments in the agreement,” he said. “This challenge is most directly related to staffing at the agency.”
The FDA has always had trouble hiring staff to achieve user fee performance goals, such as managing meetings with drug sponsors, Dorsey said. “With current staffing levels, these hurdles will remain high for the agency,” he said.
