Changes in evidence used for FDA Novel Drug Approvals: 2016-2024.

Abstract

The 21st Century Cures Act (2017) was enacted during a period of evolving FDA flexibility in evidentiary standards for drug approval. To examine trends before and after implementation, we independently reviewed all novel drugs approved between 2016 and 2024. We constructed a database of all novel FDA approvals from January 1, 2016, through December 31, 2024. Each study linked to an approved drug (N = 6,763) was cataloged by study number, sponsorship, and timing of results reporting relative to completion. Since 2016, the number of studies supporting approval has steadily declined. Beginning in 2017, the modal number of studies per approval fell to one. Industry sponsorship increased while NIH-supported studies decreased. Average time to public posting of results exceeded the one-year statutory limit. In the post-2016 period, FDA approvals were supported by fewer studies and by a greater proportion of industry-sponsored studies. Although this may accelerate access to new therapies, it raises concerns about the strength of evidence for safety and effectiveness.