Methodological Approaches to Real-World Evidence Generation for Glucagon-like Peptide-1-Based Therapies: Synopsis of a National Institute of Diabetes and Digestive and Kidney Diseases Workshop.

Methodological strategies like pragmatic trials and target trial emulation improve real-world evidence for GLP-1 receptor agonists, addressing key gaps in clinical practice and policy.

• Why it matters: Expanding use of GLP-1RAs in the U.S. raises questions about their optimal application outside controlled trials, necessitating reliable real-world data to inform decision-making.
• What they did: Experts discussed strengths and limitations of diverse data sources and emphasized study design, data quality, and analytic methods to enhance causal inference and generalizability in RWE generation.
• The result: Implementing rigorous approaches enables generation of robust RWE that supports clinicians, patients, payers, and policymakers in optimizing GLP-1RA use and improving health outcomes.