GLYMOUR, M. Maria; WEUVE, Jennifer; DUFOUIL, Carole; MAYEDA, Elizabeth Rose

doi:10.1093/aje/kwac063

Métadonnées

Afficher la notice complète

Licence d’utilisation du document

GLYMOUR, M. Maria

WEUVE, Jennifer

DUFOUIL, Carole

Bordeaux population health [BPH]

Langue

Article de revue

Ce document a été publié dans

American Journal of Epidemiology. 2022-04-06

Résumé en anglais

Alzheimer's disease (AD) is a progressive disorder common among older adults and culminating in profound cognitive impairments and high mortality risk. The US Food and Drug Administration (FDA) recently provided accelerated approval for Aduhelm, a medication for AD treatment. Aduhelm has been described as the first disease-modifying treatment for AD but has not been demonstrated to improve patients' cognitive or functional outcomes. In this commentary, we describe why Aduhelm approval was controversial and aspects of the current evidence of special pertinence to epidemiologists. The FDA decision was primarily based on two randomized controlled trials (RCTs), both terminated early, with conflicting findings about the cognitive benefits of Aduhelm. Both RCTs showed important adverse effects of the medication. The FDA cited the documented reduction in brain amyloid, an AD biomarker hypothesized as a surrogate outcome, to justify accelerated approval. Despite lack of racial/ethnic diversity in the RCT participants, concerns about health disparities have been invoked to argue for public funding of this expensive medication. The Centers for Medicare and Medicaid Services will soon make a funding determination for Aduhelm and similar medications. We end by describing how innovative study designs could accelerate post-approval research and evaluate the proposed surrogate outcomes.< Réduire

Mots clés en anglais

Alzheimer disease

Health disparities

Pharmacoepidemiology

Randomized controlled trials

Regulatory epidemiology

Research methods

Métadonnées

Partager cette publication !

Licence d’utilisation du document

Aduhelm, the newly approved medication for Alzheimer's disease: what epidemiologists can learn and what epidemiology can offer

Langue

Ce document a été publié dans

Résumé en anglais

Mots clés en anglais

URI

DOI

Unités de recherche