Triple-A RWE: Adequate Data, Appropriate Study Designs, and Actionable Evidence
Gregory Daniel, PhD, MPH, RPh
Deputy Director and Clinical Professor
Duke-Margolis Center For Health Policy, United States
The last several years have seen increasing effort to better understand when and how real-world data and evidence could better support a wide range of regulatory decisions. And while sponsors, researchers, policymakers, and regulatory agencies continue to develop real world evidence (RWE) frameworks to achieve that express aim, guiding principles are emerging related to the selection of adequate real-world data sources and appropriate study designs within specific regulatory contexts: How do specific characteristics related to disease area, data source, or analytical method affect questions around maintaining randomization? When can well-done observational studies suffice for generating actionable evidence? Are we nearing truly proof-of-concept pilot opportunities? This session will use key takeaways from stakeholder case studies to highlight progress to date on addressing these key questions and furthering the regulatory acceptability of RWE.
Learning Objective : Describe unique applications of real world evidence (RWE) to advance healthcare knowledge and decision making; Describe potential uses of RWE to improve the speed and cost effectiveness determining safety and effectiveness of medical products in real-world use.
Jacqueline A. Corrigan-Curay, JD, MD
Director, Office of Medical Policy, CDER
FDA , United States
Paul A. Bleicher, MD, PhD
Chief Executive Officer
OptumLabs , United States