Outcome measures of phase III anticancer drug trials in China

Abstract

[...]103 of these 503 phase III anticancer clinical trials were subsequently excluded from this study for various reasons. [...]data correction and reassignment were performed. The reasons why multiple endpoints were used in the primary analysis were to increase the power of statistical tests (or reduce the required sample size) by aggregating information from multiple endpoints and to describe treatment effects more comprehensively in diseases that manifest in a multifaceted way where a single endpoint does not suffice to fully represent the treatment effect. According to Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics, the surrogate endpoints DFS and event-free survival (EFS) used as primary endpoints in the adjuvant setting for breast cancer, colorectal cancer, gastrointestinal stromal tumors, melanoma, and renal cell carcinoma seem to be well accepted by the US FDA. The fundamental reason may lie in the heterogeneity of the correlation intensity between surrogate endpoints and OS, which may be attributed to multiple factors, such as the specific disease, context of use, magnitude of the effect, disease setting, location of metastatic sites, available therapy, and risk-benefit relationship.