Tobias Mütze, James Bell, Stefan Englert, Philip Hougaard, Dan Jackson, Vivian Lanius, Henrik Ravn
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引用次数: 0
摘要
ICH E9(R1)指南概述了临床试验的规划、设计、实施、分析和解释的估算指标框架。文献中概述了在临床试验中使用该框架的好处和价值,并就如何选择估计指标和定义估计指标属性提供了指导。尽管在临床试验中实施估计指标方面取得了进展,但据我们所知,目前还没有关于临床试验中的估计指标应符合哪些基本原则的公开讨论,这些原则应定义明确,并与 ICH E9(R1) 指南中提出的观点保持一致。因此,在这篇观点文章中,我们提出了定义估算指标的四项关键原则。这些原则构成了定义明确的治疗效果的基础,反映了估计值的思维过程。我们希望本观点能够补充 ICH E9(R1),并激发关于临床试验中的估计指标应具备哪些基本属性的讨论,并希望这些讨论最终能够提高临床试验中定义估计指标的清晰度和精确度。
Principles for Defining Estimands in Clinical Trials-A Proposal.
The ICH E9(R1) guideline outlines the estimand framework, which aligns planning, design, conduct, analysis, and interpretation of a clinical trial. The benefits and value of using this framework in clinical trials have been outlined in the literature, and guidance has been provided on how to choose the estimand and define the estimand attributes. Although progress has been made in the implementation of estimands in clinical trials, to the best of our knowledge, there is no published discussion on the basic principles that estimands in clinical trials should fulfill to be well defined and consistent with the ideas presented in the ICH E9(R1) guideline. Therefore, in this Viewpoint article, we propose four key principles for defining an estimand. These principles form a basis for well-defined treatment effects that reflect the estimand thinking process. We hope that this Viewpoint will complement ICH E9(R1) and stimulate a discussion on which fundamental properties an estimand in a clinical trial should have and that such discussions will eventually lead to an improved clarity and precision for defining estimands in clinical trials.
期刊介绍:
Pharmaceutical Statistics is an industry-led initiative, tackling real problems in statistical applications. The Journal publishes papers that share experiences in the practical application of statistics within the pharmaceutical industry. It covers all aspects of pharmaceutical statistical applications from discovery, through pre-clinical development, clinical development, post-marketing surveillance, consumer health, production, epidemiology, and health economics.
The Journal is both international and multidisciplinary. It includes high quality practical papers, case studies and review papers.