The prominent consumer advocacy group Public Citizen is calling for the resignations of three top officials at the Food and Drug Administration, following the agency's decision to approve a controversial new medicine for Alzheimer's disease.
The medicine, now known as Aduhelm, is meant to slow the decline in brain function that Alzheimer's causes. But whether it does remains unclear. Despite shaky evidence, questionable data analysis, and warnings from external advisers and its own statisticians, the FDA last week chose to clear Aduhelm for market, thereby making the drug available to roughly 6 million people in the U.S. living with Alzheimer's.
Aduhelm's approval has been exceptionally divisive. For patients and their caregivers, who until now only had treatments for Alzheimer's symptoms, rather than the disease itself, the drug offers a much-needed source of hope.
Yet, many experts aren't convinced Aduhelm actually helps patients, especially for those with more advanced disease, who weren't allowed to participate in the clinical trials of the drug.
Aduhelm's developer, Biogen, also set its initial average price at $56,000 a year, which is expected to put an enormous strain on healthcare budgets — particularly Medicare, the federal health insurance program for people 65 and older.
Against this backdrop, Public Citizen is asking Xavier Becerra, the new head of the Department of Health and Human Services, to request the resignations of Janet Woodcock, acting FDA commissioner, Patrizia Cavazzoni, director of the FDA's Center for Drug Evaluation and Research, and Billy Dunn, director of the agency's neuroscience office.
"The FDA's decision to approve aducanumab for anyone with Alzheimer's disease, regardless of severity, showed a stunning disregard for science, eviscerated the agency's standards for approving new drugs, and ranks as one of the most irresponsible and egregious decisions in the history of the agency," Michael Carome, director of Public Citzen's health research group, wrote in a June 16 letter to Becerra.
"The primary beneficiaries of the agency's action are Biogen and its shareholders," Carome added, "who undoubtedly are ecstatic about their soon-to-be-reaped windfall profits from sales of the company's exorbitantly priced but ineffective drug."
While Public Citizen's call isn't likely to be heeded by the Biden administration, it's a notable rebuke of the agency's leadership amid mounting blowback over their decision. In an email to BioPharma Dive, Carome noted that Public Citizen has never before made "such a formal request urging the resignations or removal of FDA officials over a specific action," nor has the group objected so strongly to an FDA drug approval decision.
Public Citizen isn't alone in its criticism of Aduhelm's approval and price tag. Ron Wyden, the Democratic senator from Oregon who heads the powerful Senate Finance Committee, recently wrote on Twitter that it's "unconscionable to ask seniors and taxpayers to pay $56,000 a year for a drug that has yet to be proven effective."
Another Democratic congressman, Peter Welch of Vermont, wrote a letter to Biogen CEO Michel Vounatsos demanding information on how the company set Aduhelm's price.
According to Welch, if just one in six Alzheimer's patients gets Aduhelm, it would cost the Medicare Part B drug program $56 billion a year — far more than the $37 billion spent for all Part B drugs in 2019.
"Biogen has abused the pricing power that it holds, making the promise of this drug unreachable for many of the 6 million people living with Alzheimer's, while pushing Medicare closer to financial disaster," Welch wrote.
Outside of Congress, three members on the committee that advises the FDA about new brain drugs have resigned within the past week in protest of the decision.
That committee, which is mostly comprised of experts in neurology and medicine, voted almost unanimously against Aduhelm during a November meeting convened by the FDA. The experts were critical not only of the limited data supporting the drug, but also of the ways Biogen and the FDA interpreted it.
The FDA often follows the recommendations of its advisers. But with Aduhelm, it deviated — a decision made all the more unusual by just how strongly the committee opposed the drug's approval. That deviation has led some advisers to take the uncommon step of removing themselves from what many consider to be prestigious roles.
"[I]t is clear to me that FDA is not presently capable of adequately integrating the Committee's scientific recommendations into its approval decisions," Aaron Kesselheim, a professor of medicine at Harvard Medical School, wrote in his letter resigning from the committee. During his time as an adviser, Kesselheim evaluated Aduhelm as well as another controversial drug, Exondys 51, which secured approval in 2016.
In addition to Kesselheim, Joel Perlmutter, a professor of neurology at Washington University in St. Louis, and David Knopman, a professor of neurology at Mayo Clinic, have also decided to leave the advisory committee.
Ned Pagliarulo contributed reporting.