A Brief Report on Proposed Areas of International Harmonization of Real-World Evidence Relevance, Reliability and Quality Standards Among Medical Product Regulators.
Maryam Nafie, Valerie J Parker, Mark McClellan, Rachele M Hendricks-Sturrup
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引用次数: 0
Abstract
Background: International harmonization of real-world data and evidence (RWD/E) standards is a goal among real-world data/real-world evidence (RWD/E) policy stakeholders. The Duke-Robert J. Margolis Institute for Health Policy developed an online 'International Harmonization of RWE Standards Dashboard' to provide timely updates around these goals.
Methods: Guidance for industry (draft and final) and related literature available online by medical product regulators globally was sought and, where needed, translated into English language using a certified translator. Consultations were then held with practicing experts to identify, collate, and interpret documents. An online Tableau tool was assembled to collate guidance documents and regulatory definitions of the following key terms used among the community to describe fit-for-use RWE in regulatory submissions: relevance, reliability, and quality.
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 RWD/E. Across these terms, we propose areas of definitional alignment: data representativeness and research and regulatory concern (relevance), accuracy in data interpretation and quality and integrity during data accrual (reliability), and data quality assurance across sites and time (quality). We propose areas of definitional misalignment regarding clinical context, data availability and representativeness, and ensuring study sample sizes and/or datasets are adequate to address a given study question.
Conclusions: Our assessment of definitions provided among these four regulators lends us to propose distinct areas for harmonization based on our assessment of where regulators appear to align and highlight opportunities to address misalignment.
期刊介绍：
The aim of Pharmacoepidemiology and Drug Safety is to provide an international forum for the communication and evaluation of data, methods and opinion in the discipline of pharmacoepidemiology. The Journal publishes peer-reviewed reports of original research, invited reviews and a variety of guest editorials and commentaries embracing scientific, medical, statistical, legal and economic aspects of pharmacoepidemiology and post-marketing surveillance of drug safety. Appropriate material in these categories may also be considered for publication as a Brief Report.
Particular areas of interest include:
design, analysis, results, and interpretation of studies looking at the benefit or safety of specific pharmaceuticals, biologics, or medical devices, including studies in pharmacovigilance, postmarketing surveillance, pharmacoeconomics, patient safety, molecular pharmacoepidemiology, or any other study within the broad field of pharmacoepidemiology;
comparative effectiveness research relating to pharmaceuticals, biologics, and medical devices. Comparative effectiveness research is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition, as these methods are truly used in the real world;
methodologic contributions of relevance to pharmacoepidemiology, whether original contributions, reviews of existing methods, or tutorials for how to apply the methods of pharmacoepidemiology;
assessments of harm versus benefit in drug therapy;
patterns of drug utilization;
relationships between pharmacoepidemiology and the formulation and interpretation of regulatory guidelines;
evaluations of risk management plans and programmes relating to pharmaceuticals, biologics and medical devices.
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