Promise and Challenge of RWE for Drug Development

• Activity Number: 575 - Trial Design with Subpopulation Heterogeneity: What Is the Role of Real-World Evidence?
• Type: Topic Contributed
• Date/Time: Thursday, August 6, 2020 : 3:00 PM to 4:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Lei Nie * and Ying Yuan
• Companies: FDA and The University of Texas MD Anderson Cancer Center
• Keywords: RWE; bias; heterogeneity

Abstract

With RWD relating to patient health status or the delivery of health care routinely collected and abundantly available, the 21st Century Cures Act requires the FDA to establish a program to evaluate the potential use of RWE to support the approval of a new indication for a drug approved under section 505(c) of the FD&C Act and to help to support or satisfy postapproval study requirements. This creates a great opportunity for alternative and innovative approaches for drug development, especially for orphan drug development. However, ignoring the uncertainty, quality, or the complexities will lead to incorrect or unreliable conclusions. It is critical to address the bias and confounding through design and analyses when RWE are utilized. Controlling bias and confounding through design and analysis is the key to successful RWE application. We briefly compare a few different approaches to utilized RWE.