WASHINGTON: The FDA failed to follow its own guidance and internal practices during the approval process for Biogen's (BIIB.O) Alzheimer's drug Aduhelm, which was "rife with irregularities," according to a congressional report released on Thursday.
According to a staff report on the findings of an 18-month investigation conducted by two House of Representatives committees into the drug's regulatory review, approval, pricing, and marketing, the FDA's interactions with Biogen were "atypical" and did not follow the agency's documentation protocol.
The FDA approved Aduhelm in June 2021 under an accelerated approval pathway over the objections of its panel of outside advisers, who did not believe data definitively proved the drug`s benefit to patients. It was authorized based on evidence that it could reduce brain plaques, a likely contributor to Alzheimer's, rather than proof that it slowed the progression of the lethal mind-wasting disease. The Medicare program restricted its coverage, which has led to severely limited use of the Biogen drug.
Biogen set an "unjustifiably high" price by initially setting Aduhelm`s price at $56,000 per year despite a lack of demonstrated clinical benefit in a broad patient population, the report said, adding that the company's own internal projections showed it expected the drug to be a burden to Medicare and costly to patients.
"The findings in this report raise serious concerns about FDA's lapses in protocol and Biogen's disregard of efficacy and access in the approval process for Aduhelm," the report, prepared by the staffs of the House Committee on Oversight and Reform and House Committee on Energy and Commerce, concluded.
The agency should ensure that all substantive interactions with drug sponsors are properly memorialized, establish a protocol for joint briefing documents with drug sponsors, and update its industry guidance on the developments and review of new Alzheimer`s Drugs, the report recommended.
Biogen and other drugmakers should communicate to the FDA any concerns over safety and efficacy to the FDA as well as take a value and patient access into consideration when setting prices, the report said.
An FDA spokesperson said the FDA`s decision to approve Aduhelm was based on a scientific evaluation of the data contained in the application. He pointed to the FDA's internal review finding its staff's interactions with Biogen appropriate.
"As stated in the congressional report, an (FDA) review concluded that, `There is no evidence that these interactions with the sponsor in advance of filing were anything but appropriate in this situation,'" Biogen said.
Documents obtained by the committees show that FDA staff and Biogen held at least 115 meetings, calls, and email exchanges over a 12-month period starting in July 2019. The total number of meetings is unknown because the FDA failed to keep a clear record of informal meetings and interactions between its staff and Biogen representatives.
For example, in an exchange of the draft briefing document on October 9, 2020, FDA staff asked Biogen to move a paragraph drafted by the agency into Biogen's section of the memorandum—a change reflected when the document was finalized," the report made public to media organizations said.
According to the report, it did so on a significantly shortened timeline, approving it after three weeks of review, and for a broad label indication of "people with Alzheimer's disease" that was unsupported by clinical data.
Internal documents obtained by the investigation revealed that Biogen accepted the indication despite its own reservations about Aduhelm's ability to help patients at different stages of disease outside of clinical trials.