LAUSR

O ver the past couple of decades, the US Food and Drug Administration (FDA) has increasingly used data from single-arm trials to approve cancer drugs. Of the 563 new oncology drug indications approved by the FDA between 2002 and 2021, 176 (31%) were based on single-arm studies in which all participants received the investigational drug. New molecular entities or original biologics accounted for 87 (49%) of the 176 single-arm study–based approvals, and 89 (51%) were for supplemental indications. These are some of the statistics reported in a study by FDA investigators published in JAMA Oncology.1 Most of the singlearm trials (98%) were approved with the response rate as the most common end point.