Multiple Endpoints in Multiregional Clinical Trials

Abstract

Recently, multi-regional clinical trials (MRCTs), which incorporate subjects from many countries or regions around the world under the same protocol, have been widely conducted by many global pharmaceutical companies. The objective of such trials is to accelerate the development process for a drug and shorten the drug’s approval time in key markets. In this chapter, we will introduce recent developments for MRCT with multiple primary endpoints. As we know, in some therapeutic areas (e.g., Alzheimer’s disease), the clinical efficacy of a new treatment may be characterized by a set of possibly correlated endpoints, known as multiple co-primary endpoints. Therefore, we will first focus on a specific region and establish four statistical criteria for evaluating consistency between the specific region and overall results in MRCTs with multiple co-primary endpoints. The sample size required for the region of interest can also be evaluated based on these four criteria.