New Evidence Approach Recommended to Evaluate Medical Product Effectiveness

January 9, 2020

Washington, DC—Including real-world data (RWD) and real-world evidence (RWE) in regulatory decision-making could improve the evaluation of the effectiveness of drugs in the marketplace, according to the Duke-Margolis Center for Health Policy. In a new white paper, Duke-Margolis discusses how submissions reviewed by the Food and Drug Administration (FDA) can benefit from including RWE in evidence packages to support labeling changes regarding a product’s effectiveness through a totality of evidence approach. The paper reflects the input of the nearly 40 members of the Duke Margolis RWE Collaborative.

On January 15, 2020, Duke-Margolis Center Director Mark McClellan will moderate a webinar with experts to discuss this white paper, “Understanding the Need for Non-Interventional Studies Using Secondary Data to Generate Real-World Evidence for Regulatory Decision Making, and Demonstrating Their Credibility,” and what’s next for real-world data and evidence.

“Evaluating an evidence package, including RWE, through a totality of evidence approach has the potential to 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,” according to Duke-Margolis.  “With the increased curation of relevant and reliable data, RWE has the potential to complement the evidence generated from randomized clinical trials and to fill evidentiary gaps for healthcare decision-making.”

This work responds to the call in the 2018 Framework for FDA’s Real-World Evidence Program for the exploration of the use of RWE and RWD for regulatory decision-making regarding the effectiveness of marketed products. As part of its discussion about how an evidence package including RWE can fill evidentiary gaps and inform a product’s effectiveness labeling change, this white paper also provides case studies for existing marketed products and hypothetical case studies reviewed through the lenses of the clinical and regulatory contexts.