Regulatory-grade clinical trial design using real-world data.

Real-world data and evidence provide the potential to address the effectiveness and safety of drugs. The U.S. Food & Drug Administration has initiated a program to evaluate the potential use of real-world evidence for regulatory uses. Whether a study is designed for regulatory purposes or for other purposes, existing regulation and guidance provide a reference for high-quality studies. Clarifying the study objectives and the role of real-world data in the study are important considerations. Robustness and transparency of the analysis allow for greater understanding and acceptance of the study results.