Efficacy Evaluation
Efficacy endpoints used for supporting approvals in WRs include overall response rate (ORR), complete response(CR), event-free survival (EFS), and progression-free survival (PFS).
Efficacy evaluation methods that have been used are Cochran–Mantel–Haenszel test for response rate endpoints, Kaplan-Meier curve for time to event endpoints, t-test, and analysis of covariance (ANCOVA) for change from baseline endpoints.
Studies in nineteen WRs were designed to be powered to evaluate the primary efficacy. Among those 19 WRS, 12 WRs are considered completed. In the actual results submitted in response to WRs, only seven were able to conduct hypothesis testing in actual trials. Main reasons for not able to conduct hypothesis testing are due to present efficacy results descriptively.