Challenges in confirming drug effectiveness after early approval

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American Association for the Advancement of Science




It’s easy to understand the urge to make potentially beneficial drugs quickly available to patients in need. It’s also easy to go too far. Through its 2021 approval of Aduhelm (aducanumab) for treatment of Alzheimer’s disease, the US Food and Drug Administration (FDA) showed a willingness to embrace early approval pathways in ways that risk FDA’s reputation and undermine its core role in keeping the market free of worthless or dangerous medical products. Early approval pathways are intended to strike a careful compromise: access to promising therapies today, confirmatory evidence tomorrow. However, this often results in access—often at a hefty cost—accompanied by a persistent lack of evidence of benefit. To improve the balance between access and proof, we must understand why postapproval studies often flounder. Is it due to insufficient incentives for companies to pursue rigorous trials after approval, disincentives for patients to enroll in them, or both? To guide policy-makers in capturing the promise of early approval and to inform assessment of newly proposed pathways favored by some patient groups, we recommend careful empirical assessment of what is currently inhibiting postapproval studies and what could make them more successful.

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