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Key questions for the evaluation of anti-amyloid immunotherapies for Alzheimer’s disease

Liu, Kathy Y; Villain, Nicolas; Ayton, Scott; Ackley, Sarah F; Planche, Vincent; Howard, Robert; Thambisetty, Madhav; (2023) Key questions for the evaluation of anti-amyloid immunotherapies for Alzheimer’s disease. Brain Communications , 5 (3) , Article fcad175. 10.1093/braincomms/fcad175. Green open access

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Abstract

The clinical benefit associated with anti-amyloid immunotherapies, a new class of drugs for the treatment of Alzheimer’s disease, is predicated on their ability to modify disease course by lowering brain amyloid levels. At the time of writing, two amyloid-lowering antibodies, aducanumab and lecanemab, have obtained United States Food and Drug Administration accelerated approval, with further agents of this class in the Alzheimer’s disease treatment pipeline. Based on limited published clinical trial data to date, regulators, payors and physicians will need to assess their efficacy, clinical effectiveness and safety, as well as cost and accessibility. We propose that attention to three important questions related to treatment efficacy, clinical effectiveness and safety should guide evidence-based consideration of this important class of drugs. These are: (1) Were trial statistical analyses appropriate and did they convincingly support claims of efficacy? (2) Do reported treatment effects outweigh safety concerns and are they generalizable to a representative clinical population of people with Alzheimer’s disease? and (3) Do the data convincingly demonstrate disease course modification, suggesting that increasing clinical benefits beyond the duration of the trials are likely? We suggest specific approaches to interpreting trial results for these drugs and highlight important areas of uncertainty where additional data and a cautious interpretation of existing results is warranted. Safe, effective and accessible treatments for Alzheimer’s disease are eagerly awaited by millions of patients and their caregivers worldwide. While amyloid-targeting immunotherapies may be promising disease-modifying Alzheimer’s disease treatments, rigorous and unbiased assessment of clinical trial data is critical to regulatory decision-making and subsequently determining their provision and utility in routine clinical practice. Our recommendations provide a framework for evidence-based appraisal of these drugs by regulators, payors, physicians and patients.

Type: Article
Title: Key questions for the evaluation of anti-amyloid immunotherapies for Alzheimer’s disease
Open access status: An open access version is available from UCL Discovery
DOI: 10.1093/braincomms/fcad175
Publisher version: https://doi.org/10.1093/braincomms/fcad175
Language: English
Additional information: This version is the version of record. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: amyloid immunotherapy, Alzheimer, dementia, MCI, disease-modifying
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Division of Psychiatry
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Division of Psychiatry > Mental Health of Older People
URI: https://discovery.ucl.ac.uk/id/eprint/10172227
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