Sample Size Consideration for Treatment Effect Consistency Assessment in Multi-Regional Clinical Trials and Bridging Studies

• Activity Number: 519
• Type: Topic Contributed
• Date/Time: Wednesday, August 7, 2013 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Hui Quan*+ and Gang Li and Josh Chen and Yue Shentu
• Companies: Sanofi and Johnson & Johnson and Merck and Merck
• Keywords: Assurance probability ; emerging markets ; Extrapolation ; random-effect model ; fixed-effect model ; shrinkage estimate

Abstract

Multi-regional clinical trial (MRCT) strategy is an efficacy way for global new drug development. Nevertheless, there are situations where bridging study approach may be a more realistic approach for assessing treatment effect for a specific region. In a MRCT or bridging study, one objective is to demonstrate consistency of treatment effects across regions so that the overall treatment effect or treatment effect of the original regions can be extrapolated to a specific region. In this presentation, we compare the sample size requirements for desired assurance probability for consistency assessment based on different consistency definitions. Computations and simulations are used to demonstrate these comparisons. With the information, trial designers can select the more appropriate approach for their specific need.