Master Protocol and Designs for Setting Where Randomized Controlled Trials Are Not Feasible*

• Activity Number: 115 - Novel Statistical Methods for Emerging Problems in Modern Clinical Trials and Drug Development
• Type: Topic Contributed
• Date/Time: Monday, July 29, 2019 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Sue-Jane Wang*
• Companies: Center for Drug Evaluation and Research U.S. Food and Drug Administration
• Keywords: master protocol; post marketing requirement; medical imaging

Abstract

In recent drug development, master protocol has been recognized as a methodologic innovation. Commonly known master protocol has been applied to umbrella, basket and platform trials during the investigational stage of a drug or drugs development. In this paper, we present a master protocol design in a post-approval setting. Specifically, medical practice after drug approval must be a key element for any proposed novel design to be of relevance, practical and feasible to implement. We will give rationales, present a variety of scenarios with examples from Division of Medical Imaging and compare their utilities. Some analytical approaches will also be discussed.

*This abstract reflects the views of the author and should not be construed to represent the views or policies of the U.S. Food and Drug Administration.