F.D.A. Seeks Investigation of Its Own Alzheimer’s Drug Approval

The Food and Drug Administration on Friday called for a federal investigation into the process that led to the approval of a new drug for Alzheimer’s disease that has sparked harsh criticism from lawmakers and the medical community.

In a letter to the independent office of the Inspector General of the Department of Health, Acting Commissioner of the FDA, Dr. Janet Woodcock, the testing the agency has been undergoing approval for the drug known as Aduhelm and has an annual price tag of $ 56,000. She pointed to interactions between representatives of the drug’s developer, Biogen, and the agency, saying that some “may have occurred outside of the formal correspondence process”.

“To the extent that these concerns could undermine public confidence in the FDA’s decision, I believe it is critical that the disputed events be verified by an independent body,” wrote Dr. Woodcock. She noted that the review should investigate whether communications between agency staff and Biogen representatives violated FDA rules.

Dana Conti, a spokeswoman for Biogen, said the company would “naturally cooperate with any request related to a possible regulatory review.”

It is unusual for the agency to request an investigation into the decision-making process of its own staff for an individual drug approval. The move is likely to add to the controversy surrounding Aduhelm. The FDA approved it a month ago, overcoming stout objections from its own independent advisors and many other scientists who said there wasn’t enough evidence to know if the drug was effective.

    • New drug approved: The FDA approved the first new Alzheimer’s treatment in 18 years and the first to attack the disease process.
    • Is a new drug working?: Patient populations are desperate for new options, while several prominent Alzheimer’s experts and the FDA’s independent advisory committee have objected to the approval because of concerns about the lack of adequate evidence of efficacy.
    • Understanding Alzheimer’s Disease: Get answers to frequently asked questions about the disease that affects approximately 30 million people worldwide.
    • A face of Alzheimer’s: This profile of a woman in the early stages of the disease shows what it can be like to face the onset of symptoms and look to the future.

On Thursday, the FDA narrowed its recommendations on who should receive the drug. Originally recommended for all Alzheimer’s patients, the agency’s new guidelines state that it should only be prescribed to people with mild cognitive problems.

Dr. Aaron Kesselheim, one of three experts who stepped down from an FDA advisory panel last month in protest of the agency’s decision on Aduhelm, said he welcomed calls for a state investigation.

“It is really important that an inquiry be made publicly and quickly so that we can find answers to some of the many open questions surrounding this decision,” said Dr. Kesselheim, 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 an unusually close collaboration between Biogen and the FDA while the drug was being reviewed. One example was the agency’s move with Biogen to present its compelling review of the evidence in support of the drug at a meeting of FDA independent advisors in November 2019.

Stat, the medical news organization, first reported that Dr. Billy Dunn, the agency’s head of neuroscience, held an off-the-book meeting with a Biogen manager, Dr. Al Sandrock. While it is not uncommon for drug company executives to meet frequently with FDA officials, it is uncommon to present data that would be part of an FDA filing outside of a formal framework.

A few months earlier, Biogen had discontinued two late-stage studies of Aduhelm after early analysis found it would not prove effective. However, the Biogen researchers who analyzed the data soon concluded that the decision to stop the studies was premature and that they had reason to believe that it might still be effective.

The meeting between Dr. Dunn and Dr. Sandrock in May 2019 was a first step in resuming talks that led to approval last month. It resulted in the first in a series of standard formal meetings between Biogen and agency staff. It also resulted in a more unusual collaboration: this summer, representatives from the company and the agency worked closely to sort Biogen’s complicated data from the studies and communicate it almost daily, 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 target the biological basis of the disease, rather than just delay symptoms.

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