Evaluating the Use of Nonrandomized Real-World Data Analyses for Regulatory Decision Making
Evaluating the Use of Nonrandomized Real-World Data Analyses for Regulatory Decision Making
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DOI：
10.1002/cpt.1351
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发表时间：
2019-04-01
影响因子：
6.7
通讯作者：
Schneeweiss, Sebastian
中科院分区：
文献类型：
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作者：
Franklin, Jessica M.;Glynn, Robert J.;Schneeweiss, Sebastian
在被称为真实世界证据(RWE)的高度控制的平行组随机试验之外，对纵向医疗保健数据的分析在医学文献中受到越来越多的关注。在这篇文章中，我们讨论了RWE在药品监管中的潜在作用，重点是对医疗保健数据库的分析。我们提供了几个已经使用RWE的案例，以及RWE可能支持监管决策的案例。我们总结了调查人员和监管机构在设计或评估此类研究时应考虑的关键问题，并提出了一个结构化的过程来实施分析，以促进监管审查。我们从医疗保健数据库的分析中评估了支持RWE有效性、透明度和重复性的经验证据基础，并讨论了仍需做的工作，以确保此类分析可以为治疗的有效性和安全性提供决策准备证据。
The analysis of longitudinal healthcare data outside of highly controlled parallel-group randomized trials, termed real-world evidence (RWE), has received increasing attention in the medical literature. In this paper, we discuss the potential role of RWE in drug regulation with a focus on the analysis of healthcare databases. We present several cases in which RWE is already used and cases in which RWE could potentially support regulatory decision making. We summarize key issues that investigators and regulators should consider when designing or evaluating such studies, and we propose a structured process for implementing analyses that facilitates regulatory review. We evaluate the empirical evidence base supporting the validity, transparency, and reproducibility of RWE from analysis of healthcare databases and discuss the work that still needs to be done to ensure that such analyses can provide decision-ready evidence on the effectiveness and safety of treatments.