Optimizing Use of Real-World Data in Regulatory Decision Making for Medical Devices Used in Surgical Procedures.

IF 2.6 4区医学 Q3 PHARMACOLOGY & PHARMACY

Pharmacoepidemiology and Drug Safety Pub Date : 2026-06-01 DOI:10.1002/pds.70407

Guy Cafri, Florence Bourgeois, Paul Coplan

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引用次数: 0

Abstract

Purpose: In the United States, the Food and Drug Administration has provided guidance on the contexts in which real-world evidence (RWE) can be used to support regulatory decisions for medical devices.

Methods: In this paper, we review different sources of real-world data (RWD) for medical devices used in surgical procedures and how to optimize the design and analysis features of RWD studies to maximize the strength of RWE. The design and analysis of a study are critical to ensuring that the RWE generated from the RWD can ultimately be used to support a regulatory decision or fulfill a commitment for a medical device. We consider several topics relevant to medical device RWE in our review: misclassification and measurement error, causal inference, and incomplete follow-up.

Results: Some key challenges include indication and outcome misclassification in non‑registry data, tenuous assumptions of single‑arm designs, device‑ and surgeon‑level confounding in comparative studies, and potentially informative loss to follow‑up from censoring (e.g., insurance termination).

Conclusion: The review is intended as a guide to researchers and regulators interested in generating or evaluating medical device RWE for regulatory purposes.

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在外科手术中使用的医疗器械的监管决策中优化使用真实世界数据。

目的：在美国，食品和药物管理局提供了关于在何种情况下可以使用真实世界证据（RWE）来支持医疗器械监管决策的指导。方法：在本文中，我们回顾了外科手术中使用的医疗器械的不同来源的真实世界数据（RWD），以及如何优化RWD研究的设计和分析特点，以最大限度地提高RWE的强度。研究的设计和分析对于确保从RWD生成的RWE最终可用于支持监管决策或履行医疗器械的承诺至关重要。在我们的综述中，我们考虑了与医疗器械RWE相关的几个主题：错误分类和测量误差、因果推断和不完整的随访。结果：一些关键的挑战包括非注册数据中的适应症和结局分类错误，单臂设计的薄弱假设，比较研究中器械和外科医生水平的混淆，以及审查（例如保险终止）可能导致的随访信息丢失。结论：该综述旨在为有兴趣产生或评估医疗器械RWE的研究人员和监管机构提供指南。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Pharmacoepidemiology and Drug Safety 医学-药学

CiteScore

4.80

自引率

7.70%

发文量

173

审稿时长

3 months

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.