Decision Rules and Associated Sample Size Planning for Regional Approval Utilizing Multiregional Clinical Trials

Abstract

Multiregional clinical trials provide the potential to make safe and effective medical products simultaneously available to patients globally. As regulatory decisions are always made in a local context, this poses huge regulatory challenges. In this article we propose two conditional decision rules that can be used for medical product approval by local regulatory agencies based on the results of a multiregional clinical trial. We also illustrate sample size planning for such trials.

Key Words:

• Decision rule
• Multiregional clinical trial
• Sample size planning

ACKNOWLEDGMENTS

Xiaoyuan Chen and Nelson Lu contributed equally. The views expressed are those of the authors and do not represent the official position of the U.S. Food and Drug Administration or China State Food and Drug Administration.