The Food and Drug Administration on Friday called for a federal investigation of the process that led to the approval of a new drug for Alzheimer’s disease that has spurred sharp criticism from lawmakers and the medical community.
In a letter to the Department of Health and Human Services’ independent Office of the Inspector General, the F.D.A.’s acting commissioner, Dr. Janet Woodcock, acknowledged the scrutiny the agency has faced about the approval process for the drug, which is known as Aduhelm and has a $56,000 annual price tag. She pointed to interactions between representatives from the drug’s developer, Biogen, and the agency, saying some “may have occurred outside of the formal correspondence process.”
“To the extent these concerns could undermine the public’s confidence in F.D.A.’s decision, I believe it is critical that the events at issue be reviewed by an independent body,” Dr. Woodcock wrote. She noted that the review should look at whether any of the communication between the agency’s staff and Biogen’s representatives violated F.D.A. rules.
Dana Conti, a spokesman for Biogen, said the company “will, of course, cooperate with any inquiry in connection with a possible review of the regulatory process.”
It is unusual for the agency to request an investigation into its own staff’s decision-making process for an individual drug approval. The move is likely to intensify the controversy that has surrounded Aduhelm. The F.D.A. approved it a month ago, overriding the fierce objections of its own independent advisers and many other scientists, who said there was insufficient evidence to know whether the drug was effective.
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- Does New Drug Work?: Patient groups are desperate for new options, while several prominent Alzheimer’s experts and the F.D.A.’s own independent advisory committee objected to its approval, having raised concerns over lack of sufficient evidence of its effectiveness.
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On Thursday, the F.D.A. moved to narrow its recommendation about who should receive the drug. After originally recommending it for all Alzheimer’s patients, the agency’s new guidelines say it should be prescribed only to people with mild cognitive problems.
Dr. Aaron Kesselheim, one of three experts who quit an F.D.A. advisory panel last month in protest of the agency’s decision on Aduhelm, said he welcomed the call for a federal investigation.
“It’s really important that an investigation happen publicly and quickly so that we find answers to some of the many outstanding questions swirling around this decision,” said Dr. Kesselheim, a professor of medicine at Harvard Medical School and Brigham and Women’s Hospital.
A number of Alzheimer’s and public health experts have raised concerns about what they characterized as an unusually close collaborative relationship between Biogen and the F.D.A. while the drug was being reviewed. One example was the agency’s move to jointly present with Biogen its glowing review of the evidence supporting the drug in a meeting of the F.D.A.’s independent advisers in November 2019.
Stat, the medical news organization, first reported that in early May 2019, Dr. Billy Dunn, the head of the agency’s neuroscience division, held an off-the-book meeting with a Biogen executive, Dr. Al Sandrock. While it is not unusual for drug company executives to meet frequently with F.D.A. officials, it is unusual to present data that would be part of an F.D.A. application outside a formal setting.
A few months earlier, Biogen had moved to halt two late-stage studies of Aduhelm after an early analysis found that it would not prove to be effective. But Biogen researchers analyzing the data soon concluded that the decision to halt the studies had been premature and that they had reason to believe it might be effective after all.
The May 2019 meeting between Dr. Dunn and Dr. Sandrock was an initial step in restarting the talks that led to last month’s approval. It led to the first of a series of standard formal meetings between Biogen and agency staff. It also led to a more unusual collaboration: That summer, representatives from the company and the agency worked closely together to sort through Biogen’s complicated data from the studies, communicating nearly every day, Stat reported.
Aduhelm was the first drug approved to treat Alzheimer’s in 18 years — and it was the very first approval for a drug designed to attack the biological underpinnings of the disease, instead of just delaying symptoms.