Design and Evaluation of Multi-regional Clinical Trials with Heterogeneous Treatment Effect Across Regions
Date Issued
2011
Date
2011
Author(s)
Chen, Chi-Tian
Abstract
To speed up drug development to allow faster access to medicines for patients globally, conducting multi-regional clinical trials incorporating subjects from many countries around the world under the same protocol may be desired. Several statistical methods have been purposed for the design and evaluation of multi-regional clinical trials. However, in most of recent approaches for sample size determination in multi-regional clinical trials, a common treatment effect of the primary endpoint (continuous or binary endpoint) across regions is usually assumed. In practice, it might be expected that there is a difference in treatment effect due to regional difference (e.g., ethnic difference). For continuous endpoint, a random effect model for heterogeneous treatment effect across regions is proposed for the design and evaluation of multi-regional clinical trials in this dissertation. For binary endpoint, a power-function distribution is used to describe heterogeneous treatment effect across regions for the design and evaluation of multi-regional clinical trials. We also address consideration on the determination of the number of subjects in a specific region to establish the consistency of treatment effects between the specific region and the entire group.
Subjects
Bridging study
ethnic effect
multi-regional clinical trials
random effect model
power-function distribution
Type
thesis
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