Overview
Health regulatory agencies worldwide are increasingly incorporating real-world data (RWD) and real-world evidence (RWE) to complement traditional clinical trials, especially for rare diseases where standard trials are difficult to conduct. Regulators require RWE to meet high standards of "relevance" and "reliability." However, a lack of clear operational guidance has left researchers and database creators uncertain about how to design datasets that fulfill these regulatory expectations.
Drawing on practical experience with registries in Japan and international framework comparisons, researchers proposed a purpose-driven framework outlining the necessary standards for regulatory-grade RWE. The authors categorized regulatory needs into three core areas: drug development for rare diseases and rare molecular subtypes, safety monitoring (pharmacovigilance), and answering clinical questions that traditional trials cannot address. They compared guidance across global health authorities—including the U.S. FDA, European EMA, and Japanese PMDA—highlighting key requirements for study design, data quality management, and governance.
Why this matters
This paper provides a practical roadmap for converting routine healthcare data into credible, regulatory-grade evidence. By establishing a shared framework for data quality and encouraging early collaboration with regulatory agencies, this work helps streamline drug approvals and post-marketing surveillance, ultimately accelerating patient access to safe and effective therapies globally.