Classification of Endpoints and Strong Control of Type I Error Rates for Both Primary and Secondary Endpoints in Clinical Trials

• Activity Number: 645
• Type: Topic Contributed
• Date/Time: Thursday, August 4, 2011 : 10:30 AM to 12:20 PM
• Sponsor: International Chinese Statistical Association
• Author(s): Abdul J. Sankoh and Haihong Li*+
• Companies: Vertex Pharmaceuticals
• Keywords: Multiple testing ; Primary endpoints ; Secondary endpoints ; most powerful test

Abstract

In randomized clinical trials, efficacy response variables are often classified as primary and secondary endpoints. With recent advancements in clinical trial design and the availability of rigorous statistical methods that provide strong control of the type 1 error rate for multiple testing of hypotheses, it is now common for sponsors to design clinical trials with prospectively specified multiple testing of hypotheses of both primary and secondary endpoints with the intent to seek labeling claims for secondary endpoints. One of these advancements is the adaptive alpha allocation approach proposed by Li & Mehrotra (Statistics in Medicine 2008; 27:5377-5391). We have showed in earlier work that this approach can be made more powerful. We further explore the construction of procedures in this framework with more general functional forms applied to the p-values from the first family when allocating significance levels in the second family, and discuss the optimality of these testing procedures.