Use of Multinational Randomized Clinical Trials to Address Global Payer Needs: Challenges and Opportunities for Statisticians

• Activity Number: 218
• Type: Topic Contributed
• Date/Time: Monday, August 4, 2014 : 2:00 PM to 3:50 PM
• Sponsor: Social Statistics Section
• Author(s): John Cook*+
• Companies: Merck
• Keywords: payer perspective ; multinational clinical trial ; study design

Abstract

The design and analysis of randomized clinical trials have historically served to address the needs of regulatory agencies - to determine the efficacy and safety of new products. For payers, interest often lies in determining if these products provide sufficient economic value. In many cases, decisions concerning the design of the study render them less useful for conducting an economic evaluation. For other trials, approaches to the analysis yield information of limited value to payers.

When designing a study, one should be cognizant of the impact certain decisions (e.g., end points, comparator, follow-up time) may have on the perceived value of a new treatment. Further, common ways in which statisticians approach the analysis (e.g., time to event and estimating the relative risk reduction) may not yield relevant information for payers. With proper planning, analyses can be conducted to address both regulatory and payer needs.

During this presentation, we will discuss the challenges that certain design features and analysis approaches present and look at how these studies can be transformed to provide information relevant to payers.