Sample size considerations for Japanese patients in a multi-regional trial based on MHLW guidance.
Since the publication of the International Conference on Harmonization E5 guideline, new drug approvals in Japan based on the bridging strategy have been increasing. To further streamline and expedite new drug development in Japan, the Ministry of Health, Labour and Welfare, the Japanese regulatory authority, recently issued the 'Basic Principles on Global Clinical Trials' guidance to promote Japan's participation in multi-regional trials. The guidance, in a Q&A format, provides two methods as examples for recommending the number of Japanese patients in a multi-regional trial. Method 1 in the guidance is the focus of this paper. We derive formulas for the sample size calculations for normal, binary and survival endpoints. Computations and simulation results are provided to compare different approaches. Trial examples are used to illustrate the applications of the approaches.
Quan
Hui
H
Biostatistics and Programming, Sanofi-Aventis, Bridgewater, NJ, USA. hui.quan@sanofi-aventis.com
Zhao
Peng-Liang
PL
Zhang
Ji
J
Roessner
Martin
M
Aizawa
Kyo
K
D044466:Asian People; D002986:Clinical Trials as Topic / Q000379:methods / Q000706:statistics & numerical data*; D003198:Computer Simulation; D017277:Drug Approval / Q000379:methods / Q000706:statistics & numerical data*; D017408:Guidelines as Topic; D006801:Humans; D015233:Models, Statistical; D015337:Multicenter Studies as Topic / Q000379:methods / Q000706:statistics & numerical data*; D018401:Sample Size; D016019:Survival Analysis