An overview of cancer drugs approved by the US Food and Drug Administration based on the surrogate end point of response rate
Importance Approximately one-third of cancer drugs are approved based on response rate
(RR)—the percentage of patients whose tumors shrink beyond an arbitrary threshold—
typically assessed in a single-arm study. Objective To characterize RR end points used by
the US Food and Drug Administration (FDA) for cancer drug approval. Design, Setting, and
Participants A retrospective review of FDA-approved drug indications in oncology from 2006
to 2018. Exposures Data related to cancer type, line of therapy (first-line, second-line, or …
(RR)—the percentage of patients whose tumors shrink beyond an arbitrary threshold—
typically assessed in a single-arm study. Objective To characterize RR end points used by
the US Food and Drug Administration (FDA) for cancer drug approval. Design, Setting, and
Participants A retrospective review of FDA-approved drug indications in oncology from 2006
to 2018. Exposures Data related to cancer type, line of therapy (first-line, second-line, or …