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In one of the worst decisions in its history, the Food and Drug Administration (FDA) on June 7 approved drugmaker Biogen’s monoclonal-antibody drug aducanumab (ADUHELM) for the treatment of Alzheimer’s disease.[1] The FDA took this action despite the nearly unanimous conclusion of an independent panel of experts convened by the agency in November 2020 — and of many other scientists, neurologists and geriatric specialists — that there was inadequate evidence that the drug is effective for...
In one of the worst decisions in its history, the Food and Drug Administration (FDA) on June 7 approved drugmaker Biogen’s monoclonal-antibody drug aducanumab (ADUHELM) for the treatment of Alzheimer’s disease.[1] The FDA took this action despite the nearly unanimous conclusion of an independent panel of experts convened by the agency in November 2020 — and of many other scientists, neurologists and geriatric specialists — that there was inadequate evidence that the drug is effective for slowing cognitive decline in patients with Alzheimer’s disease.[2]
After aducanumab’s two identical phase 3 trials were stopped early because a preliminary analysis found that they were unlikely to show the drug benefitted Alzheimer’s disease patients, the FDA and Biogen worked collaboratively to salvage the drug. They jointly relied on dubious analyses that overemphasized the results of one phase 3 trial suggesting the drug at a high dose may provide minimal benefit on one measure of cognitive function but disregarded data from the other phase 3 trial showing no benefit of the drug at any dose.[3] For the meeting of the FDA’s independent panel of experts, the agency and Biogen co-authored an unprecedented joint briefing document on aducanumab that was heavily biased in favor of the drug.[4]
In December 2020, Public Citizen called for an investigation into the unprecedented and inappropriately close collaboration between Biogen and the FDA during the analysis of data from the key clinical trials of the drug.[5] This close collaboration before and after the submission of the company’s marketing application for aducanumab dangerously compromised the integrity of the FDA’s review.
Using its accelerated approval pathway, the FDA based its approval on findings that aducanumab reduced amyloid plaques (deposits) in the brains of Alzheimer’s disease patients — a so-called surrogate endpoint.[6] The agency claimed that “it is expected that the reduction in amyloid plaque will result in a reduction in clinical decline.”[7] However, other experimental drugs for Alzheimer’s disease that reduced amyloid plaques in the brain of Alzheimer’s disease patients failed to provide clinical benefits. And even in the two phase 3 trials of aducanumab, there was little correlation between the reduction in amyloid plaques in the brain and the decline in cognitive function.[8]
The FDA’s decision showed a stunning disregard for science and eviscerated the agency’s standards for approving new drugs. Because of this reckless action, the agency’s credibility has been irreparably damaged.
Approving aducanumab despite the lack of evidence of effectiveness has raised false hope for millions of Alzheimer’s disease patients and their families and will potentially bankrupt the Medicare program because of the drug’s exorbitant price — $56,000 per year — and impede for years, if not decades, the development of other experimental treatments for the disease.
References
[1] Food and Drug Administration. FDA’s Decision to Approve New Treatment for Alzheimer’s Disease. June 7, 2021. https://www.fda.gov/drugs/news-events-human-drugs/fdas-decision-approve-new-treatment-alzheimers-disease. Accessed June 9, 2021.
[2] Food and Drug Administration. Final summary minutes of the Peripheral and Central Nervous System Drugs Advisory Committee meeting. November 6, 2020. https://www.fda.gov/media/145690/download. Accessed June 9, 2021.
[3] Public Citizen. Letter to the Department of Health and Human Services Office of Inspector General investigation of the Food and Drug Administration’s inappropriate close collaboration with Biogen before and after the submission of the biologics license application for aducanumab for treatment of Alzheimer’s disease. December 9, 2021. https://mkus3lurbh3lbztg254fzode-wpengine.netdna-ssl.com/wp-content/uploads/2660.pdf. Accessed June 9, 2021.
[4] Food and Drug Administration and Biogen. Combined FDA and Applicant briefing document for the November 6, 2020, meeting of the Peripheral and Central Nervous System Drugs Advisory Committee meeting regarding NDA/BLA# 761178, Aducanumab. https://www.fda.gov/media/143502/download. Accessed June 9, 2021.
[5] Public Citizen. Letter to the Department of Health and Human Services Office of Inspector General investigation of the Food and Drug Administration’s inappropriate close collaboration with Biogen before and after the submission of the biologics license application for aducanumab for treatment of Alzheimer’s disease. December 9, 2021. https://mkus3lurbh3lbztg254fzode-wpengine.netdna-ssl.com/wp-content/uploads/2660.pdf. Accessed June 9, 2021.
[6] Food and Drug Administration. FDA’s Decision to Approve New Treatment for Alzheimer’s Disease. June 7, 2021. https://www.fda.gov/drugs/news-events-human-drugs/fdas-decision-approve-new-treatment-alzheimers-disease. Accessed June 9, 2021.
[7] Ibid.
[8] Food and Drug Administration and Biogen. Combined FDA and Applicant briefing document for the November 6, 2020, meeting of the Peripheral and Central Nervous System Drugs Advisory Committee meeting regarding NDA/BLA# 761178, Aducanumab. https://www.fda.gov/media/143502/download. Accessed June 9, 2021.
