Randomized clinical trials (RCTs) have traditionally been the gold standard of high-quality, robust scientific evidence, providing information on the safety and efficacy of a medical product or device.1 However, RCTs alone cannot answer all the questions necessary for clinical decision making, especially when the trial population is not representative of the patient population that would typically be seen in clinical practice.

With the enactment of the 21st Century Cures Act in December 2016 — which aimed to expedite the US Food and Drug Administration’s (FDA’s) approval process — there was an increased focus on post-marketing studies and real-world data.2

Real-world evidence (RWE) refers to information and evidence gathered from data collected outside of the traditional clinical research setting.1 Real-world data can be found in disease and product registries, electronic health records (EHRs), insurance claims data, personal health apps.1

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Traditionally, observational RWE studies have been used in clinical epidemiological studies, in safety surveillance of medical products, in post-marketing studies required by the FDA, to measure quality in the delivery of health care, and to track patterns in therapeutic uses.1,2 For instance, registry data is now accepted by regulatory authorities in the evaluation of medical devices.1

Real-world data culled from observational studies also reveal information about adherence, concomitant therapies, treatment decision making, and the resulting outcomes of the treatments chosen, all of which may represent information that is not always presented in RCTs. In comparison with RCTs, RWE studies are significantly less costly and time consuming, provide easier access to long-term effectiveness data, and can overcome some of the feasibility barriers of running RCTs, such as when ethical responsibilities preclude researchers from randomly selecting patients for treatment with a placebo when more effective treatments are available.

As such, RWE studies use data from real-world clinical practice, allowing practitioners to be able to better apply the study’s findings to a more general population.1