Randomized controlled trials are regarded as the gold standard for regulatory decision-making. Real-world evidence (RWE), however, is being adopted more and more beyond assessing post-marketing safety and as supportive evidence to prove drug effectiveness in both pre- and post-approval settings. To ensure high-quality, reliable, and transparent RWE, regulatory agencies are working to formulate positions and develop guidance to manufacturers on the role of RWE to inform decision-making.
Is there a consensus across regulatory agencies? Is the evidence hierarchy changing or is RWE still considered secondary to randomized trials? During the session, existing guidance documents from regulatory agencies will be discussed, exploring key elements and common themes among FDA, EMA, Health Canada, and PMDA including transparency, fit-for-purpose real-world data, collaboration between stakeholders, principled database epidemiology, replicability, and supporting data assets and governance. We will identify perceived barriers across agencies -- methodological and data challenges -- as well as limitations of the current guidance, including clarity on how manufacturers can operationalize the recommendations. Case examples of RWE adoption and rejection among agencies will be discussed to illustrate common themes and challenges with a focus on regulatory submissions in oncology. Participants will gain strategic insights into avoidable pitfalls in RWE submissions that might negatively influence a regulatory outcome.
Identify existing recommendations for using real-world evidence in regulatory decision-making.
Describe commonalities among the different regulatory guidance documents from the United States, Europe, Canada, Japan, and China agencies.
Discuss the pitfalls in real-world evidence submissions that might negatively influence a regulatory decision.