Early approval pathways for drugs – intended to strike a compromise between access to promising therapies today, and confirmatory evidence tomorrow – often result in access accompanied by a persistent lack of evidence of benefit, write Holly Fernandez Lynch and Christopher T. Robertson in a Policy Forum. This issue is especially concerning as patients in myriad disease areas push for early drug approvals, as was recently done for Aduhelm, a treatment for Alzheimer’s disease. Through its 2021 approval of Aduhelm (aducanumab), the US Food and Drug Administration (FDA) showed a willingness to embrace early approval pathways in ways that risk the FDA’s reputation, write Lynch and Robertson. To improve the balance between access and proof, they say, experts must understand why post-approval studies often flounder. The authors identify two primary challenges to early approval pathways: (1) insufficient company incentives to pursue rigorous post-approval trials; and (2) insufficient patient incentives to participate in such trials. Both of these challenges call for different policy solutions; the authors provide several. Continued acceptance of early approval pathways should be based on evidence regarding whether, when, and how meaningful post-approval trials will be possible, they emphasize. Accelerated approval is an important regulatory pathway worth trying to save, if the evidence suggests that meaningful improvements in confirmatory trials are possible.
Challenges in confirming drug effectiveness after early approval
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