Analysis of New Drug Approvals Suggests Reliance on Less Rigorous Standards

An analysis in JAMA Network Open finds a trend toward less rigorous standards for novel drug approvals in the US. For their study, researchers from Stanford University wanted to understand the effect of the 21st Century Cures Act, which gave the US Food and Drug Administration greater flexibility in applying evidence-based standards for novel drug approvals. Using data from FDA's novel drug approvals website, the investigators examined approvals of all novel drugs approved between August 1, 2022, and January 1, 2023. They note that the 37 drugs approved in 2022 were evaluated in 413 studies. Most studies —79 percent — had industry sponsorship, they note, adding that the National Institutes of Health sponsored less than 1 percent of them. Their findings also indicated that 24 drugs, or 65 percent, were approved based on a single study. Only four drugs — abrocitinib, oteseconazole, xenon Xe 129 hyperpolarized, and tirzepatide — were approved based on three or more studies. "Our results highlight a trend toward less rigorous standards for novel drug approvals that have evolved over the past few decades," the authors say. The results, they add, are in sync with previous studies that reported systematic decreases in the number of trials used for approvals. The authors caution, though, that there are limitations to their analysis, including possible writing and misclassification errors in the data.