真实世界证据时代的分散临床试验:统计学视角

IF 3.1 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL
Jie Chen, Junrui Di, Nadia Daizadeh, Ying Lu, Hongwei Wang, Yuan-Li Shen, Jennifer Kirk, Frank W. Rockhold, Herbert Pang, Jing Zhao, Weili He, Andrew Potter, Hana Lee
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引用次数: 0

摘要

越来越多的趋势是,与临床试验有关的活动在传统试验地点以外的地点进行(因此分散的临床试验或dct),其中一些是在现实世界的临床实践环境中进行的。尽管dct有许多好处,但这也带来了与dct的设计、实施和分析相关的一些问题。美国统计协会生物制药部门的真实世界证据科学工作组一直在审查dct领域,并在本文中从统计角度提供了分散试验的考虑。本文首先讨论了可能对试验产生统计影响的关键分散因素,然后总结了dct的监管指导、框架和倡议。重点讨论了系统的设计(包括估算的构建)、实施、统计分析计划(包括缺失数据的处理)和安全事件的报告。还简要讨论了一些其他考虑因素(例如,道德考虑、技术基础设施、研究监督、数据安全和隐私以及法规遵从性)。本文旨在为医疗产品的分散试验提供统计考虑,以支持监管决策。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Decentralized Clinical Trials in the Era of Real-World Evidence: A Statistical Perspective

Decentralized Clinical Trials in the Era of Real-World Evidence: A Statistical Perspective

There has been a growing trend that activities relating to clinical trials take place at locations other than traditional trial sites (hence decentralized clinical trials or DCTs), some of which are at settings of real-world clinical practice. Although there are numerous benefits of DCTs, this also brings some implications on a number of issues relating to the design, conduct, and analysis of DCTs. The Real-World Evidence Scientific Working Group of the American Statistical Association Biopharmaceutical Section has been reviewing the field of DCTs and provides in this paper considerations for decentralized trials from a statistical perspective. This paper first discusses selected critical decentralized elements that may have statistical implications on the trial and then summarizes regulatory guidance, framework, and initiatives on DCTs. More discussions are presented by focusing on the design (including construction of estimand), implementation, statistical analysis plan (including missing data handling), and reporting of safety events. Some additional considerations (e.g., ethical considerations, technology infrastructure, study oversight, data security and privacy, and regulatory compliance) are also briefly discussed. This paper is intended to provide statistical considerations for decentralized trials of medical products to support regulatory decision-making.

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来源期刊
Cts-Clinical and Translational Science
Cts-Clinical and Translational Science 医学-医学:研究与实验
CiteScore
6.70
自引率
2.60%
发文量
234
审稿时长
6-12 weeks
期刊介绍: Clinical and Translational Science (CTS), an official journal of the American Society for Clinical Pharmacology and Therapeutics, highlights original translational medicine research that helps bridge laboratory discoveries with the diagnosis and treatment of human disease. Translational medicine is a multi-faceted discipline with a focus on translational therapeutics. In a broad sense, translational medicine bridges across the discovery, development, regulation, and utilization spectrum. Research may appear as Full Articles, Brief Reports, Commentaries, Phase Forwards (clinical trials), Reviews, or Tutorials. CTS also includes invited didactic content that covers the connections between clinical pharmacology and translational medicine. Best-in-class methodologies and best practices are also welcomed as Tutorials. These additional features provide context for research articles and facilitate understanding for a wide array of individuals interested in clinical and translational science. CTS welcomes high quality, scientifically sound, original manuscripts focused on clinical pharmacology and translational science, including animal, in vitro, in silico, and clinical studies supporting the breadth of drug discovery, development, regulation and clinical use of both traditional drugs and innovative modalities.
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