The increasing availability of rich healthcare datasets, advanced data extraction technologies, and supportive frameworks for transparent and principled study design has ushered in a “new era” in which real-world evidence (RWE) has become an undeniably valuable tool for understanding treatment effects and answering critical questions about the benefits and risks of treatment that clinical trials leave unaddressed.
This review article from Clinical Pharmacology & Therapeutics examines the evolution and growing importance of real-world data (RWD) and RWE in healthcare decision-making. The authors trace how RWE has developed from primarily being used for safety surveillance to now informing therapeutic effectiveness, regulatory approvals, and clinical practice decisions. They explain how methodological advances in pharmacoepidemiology, particularly in trial emulation techniques, have helped address historical concerns about observational study validity. The article also details how various regulatory bodies, including the FDA, EMA, and others, have developed frameworks and guidance documents to standardize and validate RWE studies.
The authors present several high-impact examples of RWE studies, including the first FDA drug approval based solely on RWE (tacrolimus for lung transplant patients) and crucial COVID-19 vaccine effectiveness studies in pregnant women. They argue that well-designed RWE studies can complement randomized controlled trials (RCTs) by providing evidence in situations where RCTs would be impractical, unethical, or insufficient. Looking forward, they emphasize that while RWE shows great promise for informing healthcare decisions, realizing its full potential will require continued methodological advancement, adherence to rigorous standards, and collaboration among stakeholders in the healthcare ecosystem.