Revisiting real-world data studies: Progress, value, and challenges

<p>This review highlights the indispensable role of real-world data studies (RWS) in complementing randomized controlled trials by generating real-world evidence (RWE) that reflects diverse patient populations and clinical settings. It explores the origins and regulatory frameworks of RWS, the evolution of real-world data sources, and their expanding applications in evaluating post-marketing medical products, optimizing pre-marketing medical product development, measuring disease burden, assessing medical professional competence, evaluating healthcare service quality, and informing clinical guidelines and public health policies. The contributions of RWE to personalized medicine, healthcare resource management, and regulatory decisions underscore its significance in evidence-based practice. Despite its potential, RWS faces challenges such as data quality, purpose-driven data sharing, ethical standards, RWE validity and transparency, RWE translation, and multidisciplinary expertise, and this review proposes some strategies to advance these fields. By addressing these challenges, RWS can enhance their impact on healthcare innovation and translate into better patient outcomes globally.</p>