Contemporary Clinical Trials
Volume 58, Pages 1-94 (July 2017)
Study Design, Statistical Design, Study Protocols
Example-based illustrations of design, conduct, analysis and result interpretation of multi-regional clinical trials
Original Research Article
Hui Quan, Xuezhou Mao, Yoko Tanaka, Bruce Binkowitz, Gang Li, Josh Chen, Ji Zhang, Peng-Liang Zhao, Soo Peter Ouyang, Mark Chang
Extensive research has been conducted in the Multi-Regional Clinical Trial (MRCT) area. To effectively apply an appropriate approach to a MRCT, we need to synthesize and understand the features of different approaches. In this paper, examples are used to illustrate considerations regarding design, conduct, analysis and interpretation of result of MRCTs. We start with a brief discussion of region definitions and the scenarios where different regions have differing requirements for a MRCT. We then compare different designs and models as well as the corresponding interpretation of the results. We highlight the importance of paying special attention to trial monitoring and conduct to prevent potential issues associated with the final trial results. Besides evaluating the overall treatment effect for the entire MRCT, we also consider other key analyses including quantification of regional treatment effects within a MRCT, and assessment of consistency of these regional treatment effects.