Turning real-world data into real-world evidence: some practical guidance

Abstract

Background: Real-world evidence (RWE), the understanding of treatment effectiveness in clinical practice generated from longitudinal patient-level data that the routine operation of the healthcare system produces, complements evidence from randomized, controlled trials (RCTs). Objectives: The aim of this contribution is to highlight how robust and actionable RWE is generated. Methods: Best practices were condensed into a comprehensive yet concise guide. Results: Conducting RWE studies follows a structured approach: (1) A design layer connects the research question with the appropriate study design, (2) a measurement layer transforms the longitudinal patient-level data stream into variables that identify the study population, the pre-exposure patient characteristics, the treatment, and outcomes, and (3) an analysis layer focuses on the causal treatment effect estimation. Conclusions: RWE, if properly conducted, is a useful complement to randomized trial evidence.