Accelerating Food and Drug Administration (FDA) product approval to market based on surrogate markers in the absence of proven efficacy creates a risk of adverse outcomes for affected patients, even in response to a life-threatening condition, such as in this case, Alzheimer’s disease. FDA’s recent unexpected approval of aducanumab, despite the unified opposition of its own highly respected advisory committee after the early termination of two efficacy trials, creates the potential risk of adverse effects and lack of clinical efficacy at very high costs. In view of these concerns, a thorough review of the issues and pressures that led to this decision is worth the careful consideration of the clinical and scientific communities with regard to whether this approval represents a calculated and balanced compassionate decision versus a disturbing precedent.
- Nervous System Diseases
- Evidence-Based Practice
- Drug-Related Side Effects and Adverse Reactions
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Correction notice This article has been corrected since it first published. In the Conclusions section, the following sentence has been corrected from "Using the accelerated approval pathway, and basing their surprise decision on a validated surrogate…" to "…surprise decision on a unvalidated surrogate…".
Contributors All authors contributed.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Provenance and peer review Not commissioned; externally peer reviewed.