Best Practices and Gaps in Current Regulatory and Health Technology Assessment Real‐World Evidence Policies for Medicines and Medical Devices: Current State of Play and Next Steps

Real-world data (RWD) offers a valuable opportunity to access vast information that can drive research and generate real-world evidence (RWE), offering insights into the actual delivery of care and patient outcomes beyond the limitations of traditional clinical trials. However, policy development has struggled to keep pace as decision-makers balance the potential benefits of RWE with lingering technical concerns about its limitations. To identify emerging best practices and policy gaps, the Integration of Heterogeneous Data and Evidence towards Regulatory and HTA Acceptance (IDERHA) team conducted a landscape review of current policy documents on the use of RWD and RWE in regulatory and Health Technology Assessment (HTA) decision-making for medicines and medical devices. The review report was released for public consultation in 2024. Our analysis highlights a growing body of knowledge on RWE, with increasing momentum towards developing standardized best practices. Despite notable progress in policy development over the past 5 years, broader acceptance and use of RWE in regulatory and HTA decision-making remain uneven and inconsistent. In the year since the original analysis was completed, there have been more policies released and finalized. Other researchers have conducted additional policy reviews and had similar findings as the IDHERA team. This underscores the importance of aligning expectations in the emerging field of RWE on appropriate research standardization, RWD utilization and laying the groundwork for further policy development. These efforts will contribute significantly to the dialogue on optimizing health data to improve patient care.