Alarm inside FDA over expedited voucher pathway

The Food and Drug Administration is facing legal, scientific and ethics concerns over a new expedited drug review program that aims to speed up approvals tied to President Donald Trump’s administration, according to current and former agency staff members. The staff described anxiety and confusion inside the agency over how the pathway operates and who has authority to sign off on approvals.

The concerns, described to The Associated Press by seven current or recently departed staffers speaking anonymously because they were not authorized to discuss confidential agency matters, focus on whether the program complies with standards long used to vet drugs for safety and effectiveness. Staffers also said they have had little information about how the program works, and some described being pushed to meet aggressive deadlines even when reviewers raised regulatory or data concerns.

“National Priority Voucher” and the one-month promise

Central to the internal questions is the FDA commissioner’s National Priority Voucher program, which promises approval in as little as one month for medicines tied to “U.S. national interests.” Staffers told AP that they do not have clarity at the highest levels about which officials have the legal authority to sign off on drugs cleared under that pathway.

Review scientists and their immediate supervisors have traditionally handled approval decisions, according to the AP report. But reviewers and staffers described a shift in practice, with questions about whether political appointees and senior leaders are taking a more central role than in past FDA review processes.

Outside experts cited constraints on how quickly FDA can conduct a detailed scientific review. Dr. Aaron Kesselheim, a professor at Harvard Medical School, said, “The concept of doing a review in one to two months just does not have scientific precedent,” and he added that “FDA cannot do the same detailed review that it does of a regular application in one to two months, and it doesn’t have the resources to do it.”

Questions over information flow and skipping steps

Staffers told AP that drug reviewers had received little information about how the new program operates. Some also said that staff working on a highly anticipated anti-obesity pill were told they could skip certain regulatory steps to meet top officials’ deadlines.

The report said Reuters had separately reported that FDA officials delayed reviews of two drugs in the voucher program, citing safety concerns that included the death of a patient taking one of the medications. The AP account also described staff concerns that the expedited pathway has not been laid out in federal rules and regulations in the way other FDA programs have been.

What the administration says

Health and Human Services spokesman Andrew Nixon defended the voucher approach, saying the program prioritizes “gold standard scientific review” and aims to deliver “meaningful and effective treatments and cures.” Nixon also said voucher submissions are evaluated by “a senior, multidisciplinary review committee,” led by Vinay Prasad.

According to the AP report, the program has remained popular at the White House, where pricing concessions have been paired with FDA vouchers for drugmakers that agree to cut prices. The AP account said that when the White House announced Eli Lilly and Novo Nordisk would reduce prices on popular obesity drugs, FDA staffers had to scramble to vet new vouchers for both companies ahead of Trump’s news conference.

Paul Kim, a former FDA attorney who now works with pharmaceutical clients, argued in the AP report that the voucher pathway appears prone to political influence. He said, “It’s extraordinary to have such an opaque application process, one that is obviously susceptible to politicization.”

How approvals are being handled

The AP report said staffers’ concerns about legality and process also relate to how final approvals are reached. It said FDA drug director Dr. George Tidmarsh declined to sign off on approvals under the pathway, and that Tidmarsh resigned from the agency in November. After Tidmarsh’s departure, the report said Dr. Sara Brenner was set to have the power to decide but declined after looking further into legal implications.

AP reported that Dr. Mallika Mundkur, the deputy chief medical officer, is taking on responsibility for the pathway’s approvals. The report described a shift from the usual role of FDA review scientists: under the voucher program, it said, approval comes through a committee vote by senior agency leaders led by Prasad, and staff reviewers do not get a vote.

Kesselheim, who was quoted in the AP report, characterized that change as a reversal. The AP account said Kesselheim, who is also a lawyer and medical researcher, described “a complete reversal from the normal review process,” which is traditionally led by scientists immersed in the data.

Expansion beyond an initial pilot

AP said the voucher program started framed as a pilot with no more than five drugs, but has expanded to 18 vouchers awarded, with more under consideration. The report described added pressure inside the FDA’s drug center, where 20% of staff left through retirements, buyouts or resignations over the past year.

The AP account said Makary unveiled the program in October and that nominations for drugs were framed as coming from career staffers, with some early drugs recommended by FDA reviewers. But the report also described a growing pattern in which selection decisions are led by Prasad or other senior officials, sometimes unbeknownst to FDA staff, and in one instance, reviewers learned from GlaxoSmithKline representatives that Prasad had contacted the company about a voucher.

Lilly, timelines, and data gaps

The AP report described confusion and anxiety linked to differing interpretations of review timelines. Two people involved in the ongoing review of Eli Lilly’s anti-obesity pill told AP that company executives initially told the FDA they expected approval within two months.

Staffers said that timeline alarmed reviewers because it did not include the agency’s standard 60-day prefiling period, which has been in place for more than 30 years. AP said Lilly pushed for a faster filing turnaround, demanding one week, and that the agency and company eventually agreed to a two-week period.

The AP report also said Lilly’s CEO, David Ricks, told attendees at a health care conference on Tuesday that the company expects FDA approval of its pill in the second quarter of the year. Nixon declined to comment on Lilly’s review specifics but said FDA reviewers can “adjust timelines as needed.”

A person involved in the process told AP that staffers were pushed to keep the application moving forward even as key chemistry data appeared to be missing, and that when reviewers raised concerns, they were told it was OK to overlook regulations if the science is sound. Former reviewers and outside experts told AP that following regulations is how FDA staff scientifically confirm safety and effectiveness.

The AP report said not all experts see the voucher pathway as problematic. Dan Troy, who served as the FDA’s top lawyer under President George W. Bush, told AP that federal law gives the commissioner broad discretion to reorganize drug review handling.

Troy also said the voucher program, like other Makary initiatives, may not last because it is not codified. In the AP account, Troy said: “If you live by the press release then you die by the press release,” adding that the effort could be wiped out by the next administration.

Kesselheim told AP the program is fundamentally changing how standards are applied while the underlying law remains what it is. He said, “The hope is that one day we will return to these scientifically sound, legally sound principles.”