The US Food and Drug Administration (FDA)'s accelerated approval program, in which drugs are approved on the basis of surrogate endpoints, has been a source of controversy in the context of risk-benefit trade-offs. The FDA recently reported on the 25-year experience with the program and concluded that the low failure rate in post-approval confirmatory trials was evidence that the program was operating effectively. However, many studies have revealed manufacturers' poor adherence to obligation of conducting confirmatory trials, and also concerned the harm and cost of cancer drugs approved through this program based on the limited evidence. To date, few studies have investigated accelerated approval of non-cancer drugs. Therefore, the purpose of this study is to review trends in the FDA's utilization of its accelerated approval program for non-cancer drugs and clarify the status of fulfillment of social obligations by the FDA and sponsors in the program. The FDA' s documents will be reviewed at Drugs@FDA to identify pre-approval and confirmatory trials and regulatory updates for drug indications granted accelerated approval other than cancer treatments. We will further search PubMed, Google/Google Scholar, and the sponsors' websites to match each identified confirmatory trial to publications in the medical literature.
Characteristics of Preapproval and Postapproval Studies for Drugs Granted Accelerated Approval by the US Food and Drug Administration