Proposing a Coverage With Evidence Development Framework for Accelerated Approval at the U.S. Food and Drug Administration
Dhanush Bearelly, Purav Desai, David T. Zhu, Rajesh BalkrishnanKey Points
The FDA's accelerated approval pathway was designed to get promising drugs to patients faster, approving them based on surrogate endpoints before confirmatory evidence of clinical benefit is available. Sponsors are required to complete confirmatory trials after approval; however, Medicare pays for these therapies immediately, regardless of where those trials are met. Public dollars, in other words, are already moving while the fundamental question of whether these drugs actually work remains unanswered. Financial incentives for manufacturers further complicate this issue; revenue is generated regardless of the trial's progress, reducing the urgency to complete them. We propose extending Coverage with Evidence Development (CED), an existing CMS policy tool, to drugs approved through the accelerated approval pathway. Under this framework, Medicare coverage would continue following FDA approval but would be contingent on participation in evidence‐development activities, including disease registries, real‐world data collection, and confirmatory trials. This approach operates within existing CMS authority and requires no new legislation. Linking reimbursement to evidence generation would better align financial incentives with the public interest and strengthen accountability during the confirmatory trial phase without restricting patient access.