Designing, Conducting, Monitoring, and Analyzing Data from Pragmatic Randomized Clinical Trials: Proceedings from a Multi-stakeholder Think Tank Meeting
Designing, Conducting, Monitoring, and Analyzing Data from Pragmatic Randomized Clinical Trials: Proceedings from a Multi-stakeholder Think Tank Meeting
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DOI：
10.1007/s43441-020-00175-7
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发表时间：
2020-06-08
影响因子：
1.5
通讯作者：
Ellenberg, Susan S.
中科院分区：
文献类型：
--
作者：
Lentz, Trevor A.;Curtis, Lesley H.;Ellenberg, Susan S.
2018 年底，美国食品药品监督管理局 (FDA) 概述了一个框架，用于评估使用真实世界证据 (RWE) 来支持监管决策的可能性。创建该框架是为了促进产生高质量 RWE 的研究，包括实用临床试验 (PCT)，这是一种随机试验，旨在通过评估干预措施的实际有效性来为临床或政策决策提供信息。专家们普遍认为，使用现有的医疗保健和患者生成的数据有望使随机试验更加高效、成本更低且更具普遍性。然而，必须权衡依赖现实世界数据源的好处与确保数据完整性和完整性的困难。此外，使用可能无法实时获得的医疗保健系统数据来适当监测新药随机试验中的患者安全可能相当困难。认识到这些问题和其他问题对于 PCT 的发展和可接受性至关重要，来自学术界、行业、专业组织、监管机构、政府机构和患者权益倡导者的一群利益相关者在 2019 年 1 月（华盛顿特区）举行的题为“监测和分析实用简化随机临床试验数据”的智库会议上讨论了 PCT 增长和可持续性的前进道路。这次会议的目标是：(1) 评估有可能产生高质量证据的针对 PCT 的研究设计和方法学选择； (2) 讨论确保 PCT 数据质量的最佳实践； (3) 确定适当的研究监测方法。本手稿总结了智库会议的记录。
In late 2018, the Food and Drug Administration (FDA) outlined a framework for evaluating the possible use of real-world evidence (RWE) to support regulatory decision-making. This framework was created to facilitate studies that would generate high-quality RWE, including pragmatic clinical trials (PCTs), which are randomized trials designed to inform clinical or policy decisions by assessing the real-world effectiveness of an intervention. There is general agreement among experts that the use of existing healthcare and patient-generated data holds promise for making randomized trials more efficient, less costly, and more generalizable. Yet the benefits of relying on real-world data sources must be weighed against difficulties with ensuring data integrity and completeness. Additionally, appropriately monitoring patient safety in randomized trials of new drugs using healthcare system data that might not be available in real time can be quite difficult. Recognizing that these and other concerns are critical to the development and acceptability of PCTs, a group of stakeholders from academia, industry, professional organizations, regulatory bodies, government agencies, and patient advocates discussed a path forward for PCT growth and sustainability at a think tank meeting entitled "Monitoring and Analyzing Data from Pragmatic Streamlined Randomized Clinical Trials," which took place in January 2019 (Washington, DC). The goals of this meeting were to: (1) evaluate study design and methodological options specific to PCTs that have the potential to yield high-quality evidence; (2) discuss best practices to ensure data quality in PCTs; and (3) identify appropriate methods for study monitoring. Proceedings from the think tank meeting are summarized in this manuscript.