Real-world evidence: state-of-the-art and future perspectives
Publication: Journal of Comparative Effectiveness Research
Abstract
Recent developments in digital infrastructure, advanced analytical approaches, and regulatory settings have facilitated the broadened use of real-world evidence (RWE) in population health management and evaluation of novel health technologies. RWE has uniquely contributed to improving human health by addressing unmet clinical needs, from assessing the external validity of clinical trial data to discovery of new disease phenotypes. In this perspective, we present exemplars across various health areas that have been impacted by real-world data and RWE, and we provide insights into further opportunities afforded by RWE. By deploying robust methodologies and transparently reporting caveats and limitations, real-world data accessed via secure data environments can support proactive healthcare management and accelerate access to novel interventions in England.
Plain language summary
What is this article about?
Research involving real-world data (RWD) from patients, which most commonly comprises routine electronic health records, differs from clinical trials but should be held to equally high standards. When conducted properly, RWD research produces real-world evidence (RWE), which can greatly inform on health areas like chronic conditions such as neurological disorders or metabolic disorders. This article provides an overview of impacts and opportunities from RWD data sources in England, by describing exemplars across different health areas. Further, the authors evaluated recent technology appraisals submitted to the National Institute for Health and Care Excellence, to understand how RWE has been utilized. The article also provides recommendations to minimize bias and ensure robust transportability of outcomes to the target patient population.
What were the results?
We provide numerous references of how RWE has increased our knowledge of infectious diseases, neurological disorders, metabolic disorders, as well as mental health conditions. Our evaluations of technology appraisals revealed that RWD is prominently used to inform on cost–effectiveness for innovative technologies, relative to clinical effectiveness.
What do the results of the study mean?
This paper explains how better linked and higher quality data sources, when interrogated using best practices and robust study designs, provide researchers with tools with enormous potential to empower outcomes that benefit patients and strengthen the healthcare system.
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Received: 1 August 2024
Accepted: 31 January 2025
Published online: 7 March 2025
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Real-world evidence: state-of-the-art and future perspectives. (2025) Journal of Comparative Effectiveness Research. DOI: 10.57264/cer-2024-0130
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