FDA sets single pivotal trial as new default for drug approvals

FDA Commissioner Martin Makary and CBER Director Vinay Prasad published a New England Journal of Medicine perspective on February 18 announcing that one adequate and well-controlled trial, combined with confirmatory evidence, would serve as the new default basis for marketing authorization of novel products. The change reversed the two-trial standard from the 1962 Kefauver-Harris Amendment, enacted after the thalidomide crisis. Industry groups welcomed the cost savings while patient safety advocates warned of replication risk.