Sample Size Considerations for Japanese Patients in a Multi-Regional Trial Based on MHLW Guidance

• Activity Number: 547
• Type: Topic Contributed
• Date/Time: Thursday, August 6, 2009 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Hui Quan*+ and Peng-Liang Zhao and Ji Zhang and Martin Roessner and Kyo Aizawa
• Companies: sanofi-aventis and sanofi-aventis and sanofi-aventis and sanofi-aventis and sanofi-aventis
• Keywords: Bridging study ; ethnic effect ; delta method ; survival endpoint ; risk reduction

Abstract

Since the publication of the ICH 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, 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.