ISSUE: - International randomized controlled trials are common and regulators (such as the FDA) acknowledge the globalization of drug development and trial conduct. However, it is not always feasible to study large populations of patients within each country where the manufacturer is seeking access. This challenge is present in RCTs and real-world data studies. While country-specific RWD exists, there are often challenges in accessing this data for commercial purposes. The ideal situation is submitting within country data to demonstrate effectiveness, safety, and value, but regulatory and HTA agencies understand the limitations and barriers to access. These decision-makers have signaled receptivity to international RWD (i.e., RWE from outside their country) if clinical practice, care delivery, healthcare systems, and patient populations are similar and data are reliable and relevant. Currently, there is a lack of guidance on what constitutes sufficient similarity in healthcare systems. This panel will discuss the ideal settings to use international RWD and gaps in current guidance from decision-makers. Panelists will also debate a working definition of “similarity” between healthcare systems and criterion needed to demonstrate generalizability.

OVERVIEW: - The moderator will present an overview of the current decision-maker recommendations on using international RWD and identify gaps in the recommendations. Each panelist will briefly present their perspective on the topic and their recommendations for closing the gaps in current guidance. The moderator will facilitate the debate on what “similarity between healthcare systems” should look like and how this may vary (e.g., by disease condition). The moderator will also incorporate questions from the audience to enrich the debate.