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Third member of FDA advisory panel resigns over Alzheimer's drug approval

A third member of a Food and Drug Administration (FDA) expert advisory panel has resigned over the agency's controversial approval of an Alzheimer's drug this week.

In a resignation letter sent to acting FDA Commissioner Janet Woodcock on Thursday and shared with The Hill, Aaron Kesselheim, a professor of medicine at Harvard Medical School, said the approval of Biogen's Alzehimer's drug "was probably the worst drug approval decision in recent U.S. history."

FDA approved the drug, called Aduhelm, over the objections of its 11-member outside advisory panel, which voted nearly unanimously last November not to recommend approval, citing a lack of evidence that the drug is effective. Ten members voted no, one voted uncertain.

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The agency is not required to follow the group's advice, but the overwhelmingly negative vote made the agency's endorsement a surprise. The decision set off a firestorm of criticism, especially when Biogen announced the price tag would be $56,000 for the treatment course.

An analysis from the nonpartisan Kaiser Family Foundation released Thursday found the drug could cost seniors on Medicare $11,500 in annual copays. If even a fraction of Alzheimer's patients on Medicare are prescribed Aduhelm, it would cost the program nearly $29 billion a year.

Aduhelm is the first new Alzheimer's treatment approved in nearly 20 years, but it is not a cure. The treatment, which is delivered intravenously, is intended to reduce the amount of plaque in the brains of Alzheimer's patients. 

Alzheimer's groups and patient advocates cheered the move, arguing that the possibility of improvement was important, because it could give patients more time. 

But the FDA acknowledged the drug had not demonstrated a clear clinical benefit in terms of actually slowing the progression of the disease. Instead, officials argued that by removing the plaques, Aduhelm “is reasonably likely" to benefit patients, and could potentially slow cognitive decline.  

The agency decided to approve the drug under its accelerated approval pathway, and gave Biogen nine years to complete its clinical studies.

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In his letter, Kesselheim said the accelerated approval possibility was not mentioned during the advisory panel meeting.

"At the last minute, the agency switched its review to the Accelerated Approval pathway based on the debatable premise that the drug’s effect on brain amyloid was likely to help patients with Alzheimer’s disease. But this pivotal question was not discussed at the Advisory Committee meeting, and its premise was specifically excluded from discussion," he wrote.

In his letter, Kesselheim also cited the FDA’s 2016 decision to approve eteplirsen to treat Duchenne muscular dystrophy, which also went against the advisory committee's recommendation.

"The decisions by FDA administrators to ignore the Advisory Committee’s clear recommendations led to their approval of two highly problematic drugs that offered little evidence that they would meaningfully benefit patients suffering from these devastating conditions. This will undermine the care of these patients, public trust in the FDA, the pursuit of useful therapeutic innovation, and the affordability of the health care system," Kesselheim wrote.

Kesselheim’s resignation follows those of neurologist David Knopman of the Mayo Clinic and neurologist Joel Perlmutter of Washington University in St. Louis.