P321: Proposed Areas of International Harmonization of Real-World Evidence Standards Among Medical Product Regulators
Poster Presenter
Valerie J. Parker
Assistant Research Director
Duke-Margolis Institute For Health Policy (DMI) United States
Objectives
Propose areas of regulatory harmonization, based on observed and emerging signs of real-world evidence standards alignment in guidance documents published to date among regulators globally, around real-world data relevance, reliability, and quality.
Method
Medical product regulator guidance for industry (e.g., regulator websites, peer-reviewed articles, etc.) were sourced and used to build a public Tableau tool. Definitions used to describe fit-for-use RWD (relevance, reliability, and quality) were assessed for regulatory (mis)alignment.
Results
As of February 2025, the United States Food and Drug Administration (FDA) has released the most RWE guidance documents to date (n= 13; 4 draft, 9 final). Four (4) regulators globally (US FDA, EMA, Taiwan FDA, Brazil ANVISA) have directly defined at least two (2) out of the three key terms (reliability, relevance, quality), indicating alignment around the importance of these terms used in the context of real-world data/evidence. Across these terms, we propose areas of definitional alignment among regulators: data representativeness and research & regulatory concern (relevance), accuracy in data interpretation and quality & integrity during data accrual (reliability), and data quality assurance across sites and time (quality). We identify opportunities for regulators to address definitional misalignment with respect to clinical context, data availability and representativeness, and ensuring study sample sizes and/or datasets are adequate to address a given study question.
Conclusion
Regulators engaged in supporting the international harmonization of real-world evidence standards should consider our proposed areas of alignment and opportunities to address signs of potential misalignment. This work remains critical to continue to help shape national and international real-world evidence regulatory policy conversations; and guide operational goals and standards for real-world evidence consideration broadly among regulators, health technology assessment bodies, and payers. Importantly, this ongoing work will support researchers and other regulatory stakeholders seeking to identify, in real time, global regulatory policy developments focused on real-world evidence.
