Evaluating the Use of Nonrandomized Real‐World Data Analyses for Regulatory Decision Making

Real-World Data (RWD) and Real-World Evidence (RWE) are playing an increasing role in healthcare decision-making worldwide. Policy-makers, payers and practitioners are using these data to support coverage decisions and to develop guidelines and decision-support tools for use in clinical practice.

Real-world data is the data relating to patient health status and/or the delivery of healthcare. RWD can be obtained from a variety of sources, including electronic records and patient registries. Real-world evidence is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from the analysis of RWD. RWE can be generated by different study designs or analyses, including but not limited to, randomized trials, including large simple trials, pragmatic trials, and observational studies (prospective and/or retrospective).

Researchers in this article discuss the potential role of RWE in drug-regulation with a focus on the analysis of healthcare databases. The paper summarizes key issues that investigators and regulators should consider when designing or evaluating such studies as well as a proposed process for implementing analyses that facilitates regulatory review.

The study concludes high-quality non-randomized studies from healthcare databases may improve treatment decision making by patients and their providers by supplementing (not replacing) the evidence available from RCTs.