Speaker: Jessica Franklin, PhD, Brigham and Women's Hospital and Harvard Medical School

Randomized controlled trials (RCTs) remain the gold standard for establishing the causal relationship between medications and health outcomes. However, for some clinical questions RCTs may be infeasible, unethical, costly, or generalizable to only a very narrow population. In these cases, observational studies from routinely collected "real-world" health data (RWD) are crucial for supplementing the evidence from RCTs, but concerns about the validity of real-world observational studies continue to detract from their utility for decision-making. To explore the validity of observational studies, we have launched RCT DUPLICATE, a large, comprehensive, prospective comparison of advanced observational RWD study approaches and RCTs, thereby providing guidance on how to optimize the performance of causal inference methods applied to RWD for the study of comparative effectiveness and safety of medications. In this talk, I will share the design and rationale of the project as well as initial learnings in RWD study implementation.