Can we learn lessons from the FDA's approval of aducanumab?
Open accessAbstract
On 7 June 2021, aducanumab was granted accelerated approval for the treatment of Alzheimer disease (AD) by the FDA on the basis of amyloid-lowering effects considered reasonably likely to confer clinical benefit. This decision makes aducanumab the first new drug to be approved for the treatment of AD since 2003 and the first drug to ever be approved for modification of the course of AD. Many have questioned how scientific evidence, expert advice and the best interests of patients and families were considered in the approval decision. In this article, we argue that prior to approval, the FDA and Biogen's shared interpretation of clinical trial data - that high-dose aducanumab was substantially clinically effective - avoided conventional scientific scrutiny, was prominently advanced by patient representative groups who had been major recipients of Biogen funds, and raised concerns that safeguards were insufficient to mitigate regulatory capture within the FDA. Here, we reflect on events leading to the FDA's decision on 7 June 2021 and consider whether any lessons can be learned for the field.
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Aducanumab received FDA accelerated approval in June 2021, with the post-marketing requirement to perform a phase 4 trial, though one such attempt has been abandoned due to low uptake.
“Aducanumab received FDA accelerated approval in June 2021, (499) with the post-marketing requirement to perform a phase 4 trial, though one such attempt has been abandoned due to low uptake.”
From Dementia prevention, intervention, and care: 2024 report of the Lancet standing Commission (2024) · cites this paper as [499]
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- Authors
- Liu, K. Y., & Howard, R.
- Affiliations
- University College London (GB)
- Corresponding author
- Liu, Kathy Y.
- Venue
- Nature Reviews Neurology
- Published
- 2021-09-17
- Volume
- 17
- Issue
- 11
- Pages
- 715-722
- ISSN
- 1759-4758 (ISSN-L)
- 1759-4766
- OpenAlex
- W3200817894
- Cited by
- 104
- References
- 13
- Citations by year
- 2026: 10 · 2025: 15 · 2024: 16 · 2023: 20 · 2022: 36 · 2021: 7
- FWCI
- 2.63
- Type
- journal article
- Language
- en
- Open access
- green
- discovery.ucl.ac.uk
- Locations
- Nature Reviews Neurology (publishedVersion)
- PubMed (publishedVersion)
- UCL Discovery (University College London) (submittedVersion)
- Funders
- Medical Research Council (MR/S021418/1)
- Topics
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmacology › Pharmaceutical industry and healthcare
- Health Sciences › Medicine › Physiology › Biomedical Ethics and Regulation
- Keywords
- aducanumab, Alzheimer's disease, accelerated FDA approval, regulatory capture, Biogen, clinical trial interpretation, conflicts of interest
- MeSH terms
- Aged, Aged, 80 and over, Alzheimer Disease, Humans, Middle Aged, United States, United States Food and Drug Administration, Randomized Controlled Trials as Topic, Treatment Outcome, Drug Approval, Clinical Trials, Phase III as Topic, Antibodies, Monoclonal, Humanized
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