Design and Evaluation of Multiregional Clinical Trials: Experience in Japan

• Activity Number: 650
• Type: Topic Contributed
• Date/Time: Thursday, August 2, 2012 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Yuki Ando*+
• Companies: Pharmaceuticals & Medical Devices Agency
• Keywords: multi regional clinical trial ; ethnic difference ; subgroup

Abstract

Multi regional clinical trial (MRCT) is recognized as one of the efficient tools for providing new drug faster to patients in several countries. In Japanese new drug review process, we focus on the data from Japanese subjects in MRCTs, considering the possibility of ethnic difference in the efficacy and safety of the drug. However, it is statistically difficult to evaluate data from one region, because the subjects from one region comprise just a subgroup of entire subjects. This issue will arise especially in large scale MRCT which is often conducted in cardiovascular drug development with relatively small proportion of the sample size of one region. In this paper, issues in new drug evaluation based on the results of MRCTs will be reviewed mainly based on the review reports by the Pharmaceuticals and Medical Devices Agency, Japan. Also the limitation of interpreting results of one region will be discussed with relation to the generalization down to the patient population and post approval data collection in the region.