Purpose: Explore opportunities and lessons learned for using real-world data (RWD) to generate real-world evidence (RWE) in the support of benefit:risk assessments for regulatory decisions.

Overview/Description: Real World Data (RWD) may provide supplemental information when RCTs are not feasible, ethical, or include only a small number of patients (e.g. rare diseases, rare oncology subtypes, pediatrics). Generating RWE may enhance understanding of long-term outcomes, generate external controls to support single-arm trials, and support label expansion to new indications or biomarkers. While RWD holds great promise, it is subject to considerable set of unique biases and other limitations which are now highlighted even more during the massive health system related disruptions in the COVID-19 era. These disruptions could impact the design and interpretation of future studies using RWD sources and highlight the need to understand data capture and characterize RWD before using it to generate RWE. This workshop will focus on an evaluating current progress, lessons learned, and future opportunities for RWD utilization. Dr. Rivera will provide an overview of the current global regulatory landscape and demonstrate new applications of RWE to support regulatory decision making. Dr. Burcu will describe methodological challenges in the design of fit-for-purpose observational research. Dr. Lederer will provide an overview on the assessment of validity and performance of real-world endpoints and lessons learned. Dr. Oehrlein will illustrate the importance of patient perspective on the evolving use of RWE rising to meet public health challenge. There will be allocated 15 minutes of interactive panel discussions responding to audience questions and comments.