Real world data: an opportunity to supplement existing evidence for the use of long-established medicines in health care decision making

TLDR

Real‑world evidence can augment randomized controlled trial data by providing safety and effectiveness information in large, heterogeneous populations, thereby offering a broader view of a medication’s clinical use and supporting regulatory decisions. The study seeks to deepen understanding of real‑world data collection and analysis to fully realize RWE’s potential. Keywords: electronic health records, evidence‑based medicine, real world data, real world evidence, randomized controlled trials.

Abstract

Abstract: Evidence from medication use in the real world setting can help to extrapolate and/or augment data obtained in randomized controlled trials and establishes a broad picture of a medication's place in everyday clinical practice. By supplementing and complementing safety and efficacy data obtained in a narrowly defined (and often optimized) patient population in the clinical trial setting, real world evidence (RWE) may provide stakeholders with valuable information about the safety and effectiveness of a medication in large, heterogeneous populations. RWE is emerging as a credible information source; however, there is scope for enhancements to real world data (RWD) sources by understanding their complexities and applying the most appropriate analytical tools in order to extract relevant information. In addition to providing information for clinicians, RWE has the potential to meet the burden of evidence for regulatory considerations and may be used in approval of new indications for medications. Further understanding of RWD collection and analysis is needed if RWE is to achieve its full potential. Keywords: electronic health records, evidence-based medicine, real world data, real world evidence, randomized controlled trial