Public Webinar: Bolstering RWE Study Credibility and Its Role in a Totality of Evidence Approach

Stakeholders are eager to increase the use of real-world data (RWD) throughout the drug lifecycle – development, approval, and access. In particular, they are interested in analyzing RWD to generate real-world evidence (RWE) about the use and potential benefits or risks of medical products that is actionable by a wide array of health care decision makers.

In keeping with Congressional mandates in the 21st Century Cures Act and 6th Prescription Drug User Fee Act, the US Food and Drug Administration (FDA) is exploring the potential use of RWD and RWE in regulatory decision-making. Key FDA activity in this area includes the release of the December 2018 Framework for its Real-World Evidence Program that outlines core considerations for using RWD and RWE to support labeling changes related to effectiveness for marketed drugs and biologics.

Complementary to RCTs, RWE is a valuable source of evidence that may provide more representative information on a therapy’s impact in a broader patient population, capture the evolving standard of care, and better reflect routine clinical care. However, uncertainty exists about how to evaluate the quality of studies that take place in the real-world setting, and how RWE should be used as part of an evidence package to support a labeling change.

The Duke-Margolis Center for Health Policy is hosting a public webinar to discuss the Real-World Evidence Collaborative’s work on demonstrating RWE study credibility and how RWE may contribute to an evidence package through a totality of evidence approach. The webinar will be moderated by Mark McClellan, MD, PhD, director of Duke-Margolis and will include a panel of experts who will discuss the key takeaways from recent RWE Collaborative publications and identify next steps for advancing the use of RWE for regulatory decision-making. Key topic areas include:

• Describing ethical, operational, and resource barriers to conducting interventional studies in the real world;
• Mapping designs and analysis techniques for non-interventional studies using secondary data to the characteristics of adequate and well-controlled studies as defined by 21 CFR 314.126;
• Identifying the ways RWE studies can contribute to an evidence package to support labeling changes related to effectiveness; and
• Understanding how an evidence package including RWE can contribute to substantial evidence within a totality of evidence approach to inform an effectiveness labeling change.