Tsou Hsiao-Hui, Chow Shein-Chung, Lan K K Gordon, Liu Jen-Pei, Wang Mey, Chern Herng-Der, Ho Low-Tone, Hsiung Chao A, Hsiao Chin-Fu
Division of Biostatistics and Bioinformatics, Institute of Population Health Sciences, National Health Research Institutes, Zhunan, Miaoli County, Taiwan.
Pharm Stat. 2010 Jul-Sep;9(3):201-6. doi: 10.1002/pst.442.
In recent years, global collaboration has become a conventional strategy for new drug development. To accelerate the development process and to shorten approval time, the design of multi-regional trials incorporates subjects from many countries around the world under the same protocol. After showing the overall efficacy of a drug in all global regions, one can also simultaneously evaluate the possibility of applying the overall trial results to all regions and subsequently support drug registration in each of them. Recently, the trend for simultaneous clinical development in Asian countries being undertaken simultaneously with clinical trials conducted in Europe and the United States has been rapidly rising. In this paper, proposals of statistical consideration to multi-regional trials are provided. More specifically, three aspects are addressed: the definition of the 'Asian region,' the consistency criterion between the 'Asian region' and the overall regions, and the sample size determination for the multi-regional trial.
近年来，全球合作已成为新药研发的常规策略。为加快研发进程并缩短审批时间，多区域试验的设计按照同一方案纳入来自世界许多国家的受试者。在证明一种药物在全球所有区域的总体疗效后，还可以同时评估将总体试验结果应用于所有区域并随后支持在每个区域进行药物注册的可能性。最近，亚洲国家与欧美国家同时开展临床研发的趋势迅速上升。本文针对多区域试验提供了统计考量建议。更具体地说，涉及三个方面：“亚洲区域”的定义、“亚洲区域”与总体区域之间的一致性标准以及多区域试验的样本量确定。
