When Biogen's (BIIB) Aduhelm won accelerated approval last year, the U.S. Food and Drug Administration said it represented "a first-of-its-kind treatment approved for Alzheimer’s disease."
The treatment was described as the first new treatment approved for Alzheimer’s since 2003 and the first therapy that targeted the fundamental pathophysiology of the disease.
The road to approval had not been easy. In November 2020, an FDA advisory panel voted against approving Aduhelm.
Three members resigned from the panel the following year, with one of them declaring "this might be the worst approval decision that the FDA has made that I can remember."
The FDA's approval process for Aduhelm is the subject of a scathing congressional report released on Dec. 29.
The 18-month investigation was initiated by two congressional committees and included the FDA.’s own internal inquiry into its Aduhelm review process.
Among other charges, the report found the FDA's approval process was "rife with irregularities" that included "atypical collaboration" between the agency and Biogen.
Biogen Knew of Financial Burden on Medicare
An FDA-Biogen working group engaged in at least 115 meetings, calls and substantive e-mail discussions over the course of a year beginning in July 2019, the report found.
The report said the FDA's internal review found that not all interactions between the agency and Biogen were properly documented or archived, as per FDA protocol.
The FDA "accelerated approval of Aduhelm without having put the question of the appropriateness of accelerated approval before any external advisory body or internal expected group,” the report said.
The report also charged that Biogen set an "unjustifiably high price" for Aduhelm to “make history” for the company despite the impact on patients and the Medicare program.
"Documents obtained by the committee showed that Biogen viewed Aduhelm as an unprecedented financial opportunity--estimating a potential peak revenue of $18 billion per year--and developed aggressive launch and marketing plans to maximize revenue throughout the drugs lifecycle," the report said.
In addition, the report said that the documents showed that Biogen initially set Aduhelm’s price at $56,000 per year, "despite a lack of demonstrated clinical benefit in a broad patient population and the anticipated financial impact on patients and the Medicare program."
The documents also show that Biogen was aware that the financial burden of its high price for Aduhelm "would fall primarily on Medicare."
The report made several recommendations, including ensuring that all substantive FDA interactions with drug sponsors are properly documented.
FDA Continues to Review Findings
In a statement, the FDA said it had "fully cooperated with the Committees’ evaluation and we continue to review their findings and recommendations."
"It is the agency’s job to frequently interact with companies in order to ensure that we have adequate information to inform our regulatory decision-making," the FDA statement said. "We will continue to do so, as it is in the best interest of patients."
The agency said it has already started making changes consistent with the Committee’s recommendations, including examining the use of a joint briefing document and plans to update its “Guidance for Industry Regarding Development and Review of New Alzheimer’s Drugs.”
Biogen said it “stands by the integrity of the actions we have taken.”
As stated in the congressional report, an FDA review concluded that, “There is no evidence that the interactions advance of filing “were anything but appropriate in this situation."
Alzheimer's disease is a progressive brain disorder that affects more than 50 million people around the world. To date, no drug has been found to address the disease, which can accelerate into dementia and other more serious cognitive conditions.
