A controversial plan by the FDA commissioner has sparked alarm and confusion within the agency, raising concerns about the potential compromise of long-standing legal, ethical, and scientific standards for drug approval.
The program, led by Marty Makary, has caused anxiety among FDA staff, who are already reeling from layoffs, buyouts, and leadership changes. According to seven current or recently departed staffers, the program has created a sense of uncertainty and confusion.
At the heart of the matter is the Commissioner's National Priority Voucher program, which promises to fast-track drug approvals within a month for medicines that support U.S. national interests. This approach challenges the traditional process, where approval decisions are typically made by FDA review scientists and their supervisors, not political appointees.
Drug reviewers have expressed concerns about the lack of information regarding the new program's operations. Some staffers working on an anti-obesity pill were even instructed to skip certain regulatory steps to meet aggressive deadlines set by top officials.
Outside experts highlight that FDA drug reviews, which typically take 6 to 10 months, are already the fastest in the world. Dr. Aaron Kesselheim, a professor at Harvard Medical School, emphasizes that a review in one to two months lacks scientific precedent and that the FDA lacks the resources for such a detailed analysis in such a short timeframe.
Reuters reported on Thursday that FDA officials have delayed the review of two drugs in the program due to safety concerns, including the death of a patient taking one of the medications.
The voucher program, which aims to deliver "gold standard scientific review" and "effective treatments and cures," remains popular at the White House. Republican President Trump has repeatedly announced pricing concessions alongside FDA vouchers for drugmakers who agree to reduce their prices.
For instance, when the White House announced that Eli Lilly and Novo Nordisk would lower their obesity drug prices, FDA staffers had to rush to vet new vouchers for both companies in time for Trump's news conference. This has sparked concerns about political interference in the traditionally objective FDA drug review process.
"It's extraordinary to have such an opaque application process, one that is obviously susceptible to politicization," said Paul Kim, a former FDA attorney now working with pharmaceutical clients.
Top FDA officials have declined to sign off on expedited approvals, citing concerns about the program's lack of federal rules and regulations. The FDA already has several programs to speed up or streamline reviews for promising drugs, all approved by Congress with agency staff-written regulations. In contrast, the voucher program's information is mostly confined to an agency website, with drugmakers applying by submitting a brief "statement of interest."
Agency leaders, like Dr. Vinay Prasad, the FDA's top medical officer and vaccine center director, have been directly contacting drugmakers about awarding vouchers, creating quandaries for FDA staffers on basic questions like how to formally award a voucher to a company that didn't request one.
Questions about the program's legality led the FDA's then-drug director, Dr. George Tidmarsh, to decline to sign off on approvals under the pathway. After his resignation, Sara Brenner, the FDA's principal deputy commissioner, was initially set to decide, but she also declined after considering the legal implications. Currently, the responsibility has fallen to Dr. Mallika Mundkur, the agency's deputy chief medical officer, who works under Prasad.
Giving final approval to a drug carries significant legal risks, essentially certifying that the medicine meets FDA safety and effectiveness standards. If unexpected safety issues arise later, both the agency and individual staffers could face investigations or lawsuits.
Traditionally, approval comes from FDA drug office directors, made in consultation with a team of reviewers. Under the voucher program, approval is granted through a committee vote by senior agency leaders led by Prasad, with staff reviewers not having a say.
"It is a complete reversal from the normal review process, which is traditionally led by the scientists who are the ones immersed in the data," said Kesselheim, who is both a lawyer and a medical researcher.
Not everyone sees problems with the program. Dan Troy, the FDA's top lawyer under President George W. Bush, says federal law gives the commissioner broad discretion to reorganize drug review handling. However, he notes that the voucher program, like many of Makary's initiatives, may be short-lived because it isn't codified.
"If you live by the press release, you die by the press release," Troy said. "Anything they're doing now could be wiped out in a moment by the next administration."
The voucher program has expanded significantly after outreach by FDA officials. Initially framed as a pilot program for no more than five drugs, it has grown to 18 vouchers awarded, with more under consideration. This puts extra pressure on the agency's drug center, where 20% of the staff has left through retirements, buyouts, or resignations over the past year.
When Makary unveiled the program in October, immediate concerns arose about the unprecedented power he would have in deciding which companies benefit. Makary stated that nominations for drugs would come from career staffers, and indeed, some early drugs were recommended by FDA reviewers, who deliberately selected drugs that could be vetted quickly. However, selection decisions are increasingly led by Prasad or other senior officials, sometimes unbeknownst to FDA staff.
Access to Makary is limited because he does not use a government email account for business, breaking with longstanding precedent.
Under pressure from drugmakers, some FDA reviewers were told they could skip steps in the review process. Two people involved in the ongoing review of Eli Lilly's anti-obesity pill said company executives initially expected the drug to be approved within two months, alarming FDA reviewers due to the exclusion of the agency's standard 60-day prefiling period. Lilly pushed for a quicker turnaround, and eventually, the agency and the company agreed on a two-week period.
Staffers were urged to keep the application moving forward, even though key data about the drug's chemistry appeared to be missing. When reviewers raised concerns about these gaps during an internal meeting, they were told by a senior official, "If the science is sound, then you can overlook the regulations."
Former reviewers and outside experts argue that this approach contradicts how FDA reviews should function, where following regulations scientifically confirms drug safety and effectiveness. Skipping review steps could also pose risks for drugmakers if future FDA leaders decide a drug wasn't properly vetted.
Kesselheim and other experts believe the program may not outlast the current administration.
"They are fundamentally changing the application of the standards, but the underlying law remains what it is," Kesselheim said. "The hope is that one day we will return to these scientifically sound, legally sound principles."
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