Evaluation of clinical trial designs for novel anticancer drugs in China: A cohort study of drugs approved between 2015 and 2021.

China has greatly facilitated the approval of many novel anticancer drugs since the drug regulatory reform in 2015. Here, we review the clinical trial designs used in pivotal clinical trials for approved anticancer agents in China from 2015 to 2021. Overall, 79 new molecular entities (NMEs) with 140 anticancer indications were identified. Of these, adaptive randomized controlled trial (RCT) designs were used most frequently in pivotal clinical trials (n = 83, 49%), followed by single-arm design trials (n = 52, 30%) and traditional RCT design trials (n = 36, 21%). The single-arm trials and adaptive RCTs can significantly shorten clinical trial duration compared with traditional RCT designs. Our findings showed that novel clinical trial designs were widely used in China to accelerate the launch of anticancer drugs.

Duke Scholars

Author Shein-Chung Chow Biostatistics & Bioinformatics, Division of Biostatistics