Regional Efficacy Assessment in Multi-Regional Clinical Development

• Activity Number: 123 - Topics for the Statistician Clinical Trialist
• Type: Contributed
• Date/Time: Monday, July 31, 2017 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Lanju Zhang* and yijie zhou and lu cui and bo yang and frank shen
• Companies: AbbVie, Inc. and Abbvie and Abbvie and Vertex and Abbvie

Abstract

It is common in a multi-regional clinical development that data from a global trial and a local trial (in a target country) together will be used to support local filing in the target country. This approach is considered efficient development both globally and in the target country. However it remains a challenge how to combine the global trial data and local trial data toward local filing. To address this challenge, we propose a weighted estimator that weights data from the target country and outside of the target country. This weighted estimator provides an unbiased estimate of a global treatment effect with appropriate representation of the target country patient population, where the "appropriate representation" is measured via the weight parameter. This interpretation facilitates drug development discussion with the local health authority, and sample size of the local trial can be determined using this weighted estimator. Weight determination and additional sample size features are also discussed.