Bouquets and Brickbats for FDA OK of Alzheimer's Drug Aduhelm

The Alzheimer's Association praises the approval but not the price tag. A Harvard Medical School professor who resigned from an FDA advisory committee says the OK "was probably the worst approval decision in recent U.S. history" in his resignation letter.

Aducanumab (Aduhelm, Biogen)was controversial before the FDA announced it was approving the Alzheimer's drug, and it is proving to be perhaps even more controversial after.

Three members of the advisory committee of outside experts that voted against approving the drug in November 2020 resigned last week to protest the agency's approval. The Institute for Clinical and Economic Review in Boston issued a statement that said agency had "failed in its responsibility to protect patients and families from unproven treatments with known harms" by approving Aduhelm.

The Alzheimer's Association, a major patient advocacy organization, split its verdict. The association lauded the approval but criticized Biogen's pricing. The company set the wholesale acquisition cost so the annual price tag for a maintenance dose will be about $56,000, according to Biogen. The association called that price "simply unacceptable."

“For many, this price will pose an insurmountable barrier to access, it complicates and jeopardizes sustainable access to this treatment, and may further deepen issues of health equity,” the organization said. “We call on Biogen to change this price.”

Biogen says it has entered into value-based agreements with payers and insurers. The price reflects the “overall value this treatment brings to patients, caregivers and society — and one that will enable continuous innovation,” Biogen CEO Michael Vounatsos said in a prepared statement.

Related: FDA greenlights controversial aducanumab, Biogen’s Alzheimer’s treatment

Biogen is committed to not raising Aduhelm’s price for the next four years, Vounatos said.

The three committee members who resigned are Aaron Kesselheim, M.D., J.D., M.P.H., a Harvard Medical School professor and director of program on drug law and regulations at Harvard-affiliated Brigham and Women's Hospital in Boston; David Knopman, M.D., a neurologist at the Mayo Clinic and an expert on Alzheimer's and frontotemporal degeneration; and Joel Perlmutter, M.D., a neurologist at the Washington University in St. Louis.

In his resignation letter, Kesselheim said the OK of Aduhelm "was probably the worst approval decision in recent U.S. history." He also criticized the agency's 2016 approval of eteplirsen (Exondys 51, Sarepta Therapeutics), a drug for Duchenne muscular dystrophy.

Alzheimer’s Association defended the Aduhelm approval: "As the global nonprofit leader in Alzheimer's research and science we have extensively reviewed the clinical trial data for Aduhelm (aducanumab). Based on the trial data, the treatment demonstrated an efficacious result which has also been confirmed by leading scientists in the Alzheimer's and dementia scientific community and the FDA,” the organization said.

The association said it is committed to working with the Centers for Medicare and Medicaid Services along with the private payer community to “expedite access for all of those who would likely benefit based on the successful clinical trial.”

The FDA also “appropriately took into account the vast unmet need of the Alzheimer's community,” Alzheimer’s Association said.

“Aduhelm is certainly not a cure, but at long last it provides many with Alzheimer's disease and their families an effective treatment. The confirmatory trial should begin promptly," the association's statement continued.

Aduhelm was approved under the FDA's Accelerated Approval program. Although critics of the agency and the program say enforcement of the requirement is lax, drugs approved under the program are supposed to be assessed further in clinical trials and removed from the market if the findings on which they were approved don't hold up.

In his resignation letter, Kesselheim said the FDA switched Aduhelm to the Accelerated Approval program at the last minute.

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