Early regulatory 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, according to the authors of a Policy Forum entry in the December 3 issue of the journal Science.
Holly Fernandez Lynch, JD, MBE, assistant professor of medical ethics and health policy with the University of Pennsylvania’s Perelman School of Medicine, and Christopher T. Robertson, professor of law at Boston University, note that this topic is especially concerning as patients in myriad disease areas push for early drug approvals. For example, the recent early approval for Aduhelm (aducanumab), a controversial treatment for Alzheimer’s disease, by the U.S. Food and Drug Administration showed the agency’s willingness to embrace early approval pathways in ways that risk its reputation, they write.
To improve the balance between access and proof, the authors say experts must understand why post-approval studies often flounder. They identify two primary challenges to early approval pathways:
- insufficient incentives for drug company sponsors to pursue rigorous post-approval trials; and
- 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. They add that accelerated approval is an important regulatory pathway worth trying to save, if the evidence suggests that meaningful improvements in confirmatory trials can be realized.
Edited by Gary Cramer