Characteristics of Clinical Studies Used for US Food and Drug Administration Supplemental Indication Approvals of Drugs and Biologics, 2017 to 2019

Abstract

Key Points Question What is the strength of evidence supporting supplemental new indication approvals for drugs and biologics and how does it compare with the evidence that supported their original approval? Findings In this cross-sectional study of 107 therapeutics approved for 146 supplemental indications by the US Food and Drug Administration between 2017 and 2019, supplemental approvals for oncology drugs were based on fewer pivotal efficacy trials with less rigorous designs than supplemental approvals for other therapeutic areas. Supplemental approvals were based on fewer pivotal trials than their original indication approvals, but their designs were similar. Meaning These findings suggest that there was little difference in the evidence supporting supplemental and original indication approvals, but the number and design of pivotal trials supporting supplemental indication approvals varied according to therapeutic area.