FDA May Drop Expert Reviews, Impacting Drug Approval

FDA leaders under President Donald Trump are moving to abandon a decades-old policy of asking outside experts to review drug applications, a move critics say would shield the agency's decisions from public scrutiny.

The agency "would like to get away" from assembling panels of experts to examine and vote on individual drugs, because "I don't think they're needed," said George Tidmarsh, head of the FDA's Center for Drug Evaluation and Research. He relayed the message Tuesday at a meeting of health care product makers and Wednesday to an FDA advocacy group.

In addition to being redundant, Tidmarsh said, advisory meetings on specific drugs were "a tremendous amount of work for the company and for the FDA. We want to use that work and our time to focus on the big questions."

The FDA's advisory committees were created in their current form by a 1972 law aimed at expanding and regulating the government's use of experts in technical decisions. They're periodically summoned for advice, including to review evidence and vote on whether the FDA should approve drugs, vaccines, and medical devices, often when FDA officials face a difficult decision.

FDA actions have traditionally aligned with committee votes. A departure can provoke controversy and public debate, as was the case with the split 2021 decision on whether to approve the Biogen drug Aduhelm to treat Alzheimer's disease.

The FDA approved the drug despite a "no" vote from its advisory committee, whose members felt the medicine did little to treat the disease. The conflict over Aduhelm laid bare the FDA's struggle to reconcile pressure from industry and desperate patients with its rigorous evaluation of drug risks and benefits.

Tidmarsh said the committees would still be consulted on general issues like how to regulate different classes of drugs. But meetings on specific drugs, in which experts plow through piles of studies and hours of testimony from FDA and company officials, were mainly useful, he said, because they allowed the public to see how the FDA worked.

This month, the FDA began publishing the "complete response letters" it sends to companies when it declines to approve their products. Releasing the letters, which previously required filing requests under the federal Freedom of Information Act, promotes a level of transparency akin to the advisory meetings," Tidmarsh said.

Advisory committee meetings on individual drugs "are redundant when you have the complete review letters," he told KFF Health News in a brief interview after appearing at the health care products conference.

Former FDA officials and academics who study the agency disagree. The meetings help FDA scientists make decisions and increase public understanding of drug regulation, and abandoning them doesn't make sense, they said.

Tidmarsh's reasoning is "hard to follow," former FDA Commissioner Robert Califf told KFF Health News. "It's extremely useful for people inside the FDA to find out what other experts think before they make their final decisions. And it's important to do that in a way that enables the public to understand the points of view."

"Experts might ask questions of the company or FDA that neither of them thought of on their own," said Holly Fernandez Lynch, an associate professor of bioethics and law at the University of Pennsylvania. "The public has few other opportunities to comment about FDA decisions."

Spokespeople for FDA and the Health and Human Services Department did not respond to repeated requests for elaboration on Tidmarsh's comments.

Califf at times disagreed with advisory committees as commissioner of the agency and once floated the idea that it might be better if they deliberated but did not vote on products. Still, while "maybe someone can come up with a better one, I always thought it was an amazing system," he said.

The FDA is not obliged to ask outside experts to review drugs and usually hasn't. It calls on them mainly for important new types of medications or when a decision is especially tricky because of high demand for a product that may have limited value, Aduhelm being a classic example.

The advisory committees are "an important resource" for the FDA, said Sarah Ryan, a spokesperson for the Pharmaceutical Research and Manufacturers of America. "They can play an important part of the rigorous human drug review process we have in the U.S."

The committees are often asked to help settle disagreements within the FDA about how to move forward on a regulatory decision, said Reshma Ramachandran, a health services researcher and clinician at the Yale School of Medicine.

She and other researchers and former FDA officials praised FDA Commissioner Marty Makary's decision to publish the complete response letters.

But the letters don't obviate the need for committee meetings, said Peter Lurie, a former associate FDA commissioner who heads the Center for Science in the Public Interest.

"A disclosed complete response letter tells the public that a company's application was rejected and why," Lurie said. "An advisory committee meeting says to outside experts and the public, "Here's what we're thinking of doing and we'd love your input before we decide." Plainly, those are not equivalent."