The report said the F.D.A.’s approval process for Aduhelm was “rife with irregularities” and criticized Biogen for setting an “unjustifiably high price.”
A close view of a box of the drug Aduhelm, with its name printed in blue letters on white and with Biogen's logo small and in black at the lower left corner of the package's side, resting on a surface with an out-of-focus background.
The investigation found that Biogen, Aduhelm’s manufacturer, initially priced the drug at $56,000 a year because it wanted a “blockbuster” that would “establish Aduhelm as one of the top pharmaceutical launches of all time,” according to documents.Credit...Pool photo by Jessica Rinaldi

Pam Belluck

By Pam Belluck

The Food and Drug Administration’s process for approving the Alzheimer’s drug Aduhelm, despite great uncertainty about whether it worked, was “rife with irregularities,” according to a congressional investigation released on Thursday. The agency’s actions “raise serious concerns about F.D.A.’s lapses in protocol,” the report concluded.

The 18-month investigation, initiated by two congressional committees after the F.D.A. approved the drug, also strongly criticized Biogen, Aduhelm’s manufacturer. Internal documentsshowed the company set “an unjustifiably high price” of $56,000 a year for Aduhelm because it wanted a history-making “blockbuster” to “establish Aduhelm as one of the top pharmaceutical launches of all time,” even though it knew the high price would burden Medicare and patients, the report found.

The investigation said Biogen was prepared to spend up to several billion dollars — more than two-and-a-half times what it spent developing the drug — on aggressive marketing to counter expected “pushback” over whether Aduhelm was worth its price. The report said the campaign planned to target doctors, patients, advocacy groups, insurers, policymakers and communities of color, who were drastically underrepresented in its clinical trials of the drug.

The F.D.A. is now evaluating two other Alzheimer’s drugs for possible approval early next year, including one that Biogen helped develop. The congressional report said the agency “must take swift action to ensure that its processes for reviewing future Alzheimer’s disease treatments do not lead to the same doubts about the integrity of F.D.A.’s review.”

Aduhelm’s approval in June 2021 caused an outcry from many Alzheimer’s experts. Major health systems, including the Cleveland Clinic and the Department of Veterans Affairs, decided not to offer Aduhelm, an intravenous infusion, citing its uncertain benefits and risks of brain swelling and bleeding. After Medicare sharply limited its coverage of Aduhelm, the drug — still expensive even after Biogen halved its annual price to $28,800 — was essentially sidelined from the marketplace.

The report included the F.D.A.’s own internal inquiry into its Aduhelm review process, an inquiry first reported by The New York Times in 2021. The agency’s self-examination, conducted shortly before Aduhelm was approved, was less negative than the congressional findings, but identified similar issues and said its collaboration with Biogen “exceeded the norm in some respects.”

That internal inquiry, which the F.D.A. had not previously disclosed, found that agency officials leading the Aduhelm evaluation did not pay enough attention to dissenting views from the agency’s own statistical team, which said there was inadequate evidence that Aduhelm worked. After expressing those reservations, team members were excluded from some deliberations and given little notice about the preparation of a key document.

That document was a presentation which, in a highly unusual move, the F.D.A. jointly produced with Biogen for a November 2020 meeting of the agency’s independent advisory committee. The congressional investigation said the F.D.A. had done joint presentations only nine times before, all involving cancer drugs, and that in the bullish joint Aduhelm presentation, at least one point attributed to Biogen was actually written by the F.D.A. The advisory committee overwhelmingly voted against approval.

The F.D.A.’s inquiry concluded that “given the internal disagreement” within the agency “and the lack of a unified F.D.A. perspective on the data, the use of the joint briefing document was not an appropriate approach in this instance.”

The F.D.A.’s inquiry and the congressional investigation both found that the agency did not properly document many interactions with Biogen. Beginning in July 2019, in an unusual arrangement called a “collaborative workstream,” it met repeatedly with Biogen to analyze data from one clinical trial that had failed and another trial that seemed slightly successful, helping advise whether the company should seek approval.

Over 12 months, there were at least 52 meetings, and not all of them were documented fully under F.D.A. standards, the congressional report said. In addition, “there was no official memorialization of at least 66 calls or substantive email exchanges,” the report said. Even the F.D.A.’s internal inquiry said its review “did not reveal a clear record of the number and nature of interactions” between the agency and Biogen.