In July 2025, the US Food and Drug Administration (FDA) announced a pilot program intended to expedite reviews for drug applications that met one of five national priorities, including products deemed “innovative breakthrough therapies” or therapies that target a “large unmet need.” The program, the Commissioner’s National Priority Voucher (CNPV), aims to decrease the timeline of reviews, which are currently from six to eight months, to between one and two months. So far, the FDA has granted 17 vouchers for products ranging from cancer therapies to cholesterol medication; one review was rejected and three others received approval.
More recently, the FDA’s commissioner, Marty Makary, wrote in an article that the FDA would begin requiring only “one pivotal trial” for drug applications as opposed to the traditional two. The standard for two trials was set in the 1960s, but in many instances—such as rare diseases where numbers of patients can be low—the FDA has made exceptions to permit reviews with a single trial.
According to Makary and other FDA officials, these changes aim to “reduce inefficiencies” and expedite the drug approval process without impacting the FDA’s rigorous standards for safety and efficacy. Many individuals across biopharmaceutical companies, patient advocates, and scientists commended the moves. Others, including former FDA personnel and academic researchers, raised concerns about the necessity for and implication of these changes.
A New Expedited Program for Priority Products
One of the ways that the CNPV intends to reduce the review time for selected applications is by sending these materials to the relevant FDA experts. These teams will meet to collectively discuss and share their recommendations to the relevant FDA Center director and a new CMPV review council made up of chief medical officers, senior advisers, and other relevant subject matter experts. According to Makary in a press release, “Using a common-sense approach, the national priority review program will allow companies to submit the lion’s share of the drug application before a clinical trial is complete so that we can reduce inefficiencies. The ultimate goal is to bring more cures and meaningful treatments to the American public.”
Sheila Mathias, the chief scientific officer from Virpax Pharmaceuticals, said that the program was interesting. She added that the expedited timeline could be particularly helpful for small biotechnology companies looking for partners, since they would have less time to wait before having a product in hand.
In opposition of the CNPV program, physician Aaron Kesselheim at Brigham and Women’s Hospital and Harvard Medical School, said, “The FDA is already the fastest drug regulator in the world.”
He added, “Reviewing drugs within one to two months is…far beyond the bounds of the normal time that a review takes.” He said that reviewers typically analyze the provided material from scratch so as not to be persuaded by how sponsors may present findings. “It’s just not clear how the FDA has the resources to reduce that to one to two months and what kind of corners it will be cutting to do that,” he added.
Kesselheim said that the need for the program was unclear, since the FDA has other expedited pathways for specific products and circumstances. Based on this, he raised concerns that the CNPV program was an attempt to elevate political goals, which he said should not be a focus of the FDA.
Mathias admitted that she was not sure how the FDA was reviewing the applications on a shorter timeline but said that newly introduced AI tools will likely be used to help some processes. The press release and announcement indicated that the FDA and review committee reserved the right to extend the review timeline as needed.
Andrew Nixon, the deputy assistant secretary for media relations at the FDA, said that the FDA is in the process of hiring more than 1,000 reviewers, inspectors, and investigators in its goal to modernize the agency, and that the agency has the “expertise and operational capacity necessary to carry out the CNPV pilot program.”
New Expert Panels Raises Researcher Concerns
Another shift in the FDA drug approval process in the past year has been the introduction of expert panels. Since the 1970s, the FDA has used advisory committees comprised of independent, external members of the scientific community with relevant expertise to review application material and provide advice on certain agency questions related to an FDA product.
“[Expert panels are] very different from advisory committees, and…they’re very concerning,” said Kesselheim, who previously served on the peripheral and central nervous system advisory committee from 2015 to 2021.
Per the Federal Advisory Committee Act, these members must be thoroughly vetted for possible conflicts of interest so that the decisions that they make are based only on the best available evidence. Although expert panel participants still come from outside of the FDA, they don’t undergo this same extensive review for conflicts.
“The issue of the conflict of interest is what concerns me the most,” said Juan Hincapie-Castillo, a pharmacoepidemiologist at the University of Pittsburgh who was a consultant to an advisory committee meeting in 2020. Even for his singular participation, he said that he was still screened for affiliations that could lend bias to his opinion.
The fact that the participants of expert panels are not vetted for these conflicts, Hincapie-Castillo added, “raises concerns of who is actually invited to make these decisions.” Kesselheim agreed.
Hincapie-Castillo and Kesselheim both voiced concern about the use of these panels, though, since the agency already has advisory committees available. Yet while the FDA has convened five of these newly created expert panels—on decisions ranging from updating warning labels on hormone therapy to a review of nutrient requirements in infant formula—meetings of advisory committees have starkly dropped; between 2024 and 2025, the number of committee meetings fell by 65 percent, concerning researchers.
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Nixon explained in an emailed response that the purpose of these panels is to collect information and insights from individuals with particular expertise, but not necessarily for them to vote on a recommendation. So far, the FDA has not used these expert panels during the approval process for new drug applications.
A Shift to One Pivotal Trial
In February, Makary and Vinay Prasad, the former director for the Center for Biologics Evaluation and Research, published an article in the New England Journal of Medicine announcing a new policy that drug applications would only require a single pivotal trial, along with supporting data, to be eligible for review. Again, the leaders said that this would decrease the amount of time needed to bring new therapies to market, and they added that the new policy would reduce costs for sponsors.
“When I read that, my impression was that it was very focused on efficiency, where the conversation around drug safety [and] drug approvals has to always be balanced by statute and by federal law on efficacy and safety,” Hincapie-Castillo said.
Mathias said that this change offers a lot of promise for biotechnology and pharmaceutical companies to reduce their costs in the application process. But, she added, “Your trial design has to be spot-on.” This could include ensuring companies select the best endpoint criteria and statistical analyses to demonstrate their product’s efficacy. While she said most companies already strive to design robust trials, “I just feel there’s going to be a little more pressure because it’s one shot that you’re trying to get through.”
Yet Hincapie-Castillo said that, especially since the statement came in a journal as opposed to a formal announcement with regulatory or statutory language, he’s concerned that companies are getting mixed signals about expectations. Indeed, the announcement said that the FDA could still request additional trials if the reviewers thought one was merited.
Hincapie-Castillo and Kesselheim also pointed out that the FDA already had the ability to permit approvals based on single trials. “It doesn’t make any sense to me for the FDA to just sort of voluntarily take power away from itself to make decisions about what kind of testing is necessary in what circumstances,” Kesselheim said.
Although an application has yet to be submitted under this new recommendation, researchers also expressed concerns about the proposal. “As a drug safety researcher, I worry about the loss of numbers of participants in the studies to evaluate some of these adverse reactions,” Hincapie-Castillo said.
While he does not anticipate that this change will result in unsafe products going to market, since the regulatory benchmarks are still in place, Hincapie-Castillo said, “Now what ends up happening is that we’re shifting a lot of that confirmatory evidence and a lot of studies into the post marketing.”
Mathias agreed that, if companies were only using one pivotal trial, they would have a greater responsibility to watch for safety signals after the drug was approved. However, she added that this “phase four” of drug approval, in which the company and the FDA agree to surveillance terms after the product enters the market, is already a regular part of the drug approval process, so it would not greatly impact pharmaceutical companies’ processes.
The Impacts of New Changes Are Yet to Be Seen
The ultimate impact of these changes and new programs will take time to play out. Hincapie-Castillo and Kesselheim expressed that their primary concern is that these updates will strain the FDA or introduce conflicts that prevent it from achieving its primary mission to ensure safe and efficacious medical products come to market.
“It would be a real pity to undermine the great work that is done [at the FDA] with these kinds of overly politicized programs, with cuts to the FDA staff, and all the top scientists and people working at the FDA who’ve been forced out over the last year. I think that really undermines the essential work that the FDA needs to do,” Kesselheim said.
Mathias, though, was more optimistic. “Overall I’m pretty excited. I think there’s the opportunity for more interaction and communication with the FDA which can lead to clear drug development,” she said. “I think that they’re moving towards more emphasis on still having safe and efficacious drugs but trying to streamline barriers.”
Shelby Bradford is an Associate Editor at The Scientist. She earned her PhD in immunology and microbial pathogenesis from West Virginia University, where she studied neonatal responses to vaccination. Find Shelby on X @TheGeekyGoth
A version of this article was originally posted at The Scientist and is reposted here with permission. Any reposting should credit both the GLP and original article. Find The Scientist on X @TheScientistLLC
