Mütze Tobias, Bell James, Englert Stefan, Hougaard Philip, Jackson Dan, Lanius Vivian, Ravn Henrik
Statistical Methodology, Novartis Pharma AG, Basel, Switzerland.
Clinical Operations, Elderbrook Solutions GmbH, Buckinghamshire, UK.
Pharm Stat. 2025 Jan-Feb;24(1):e2432. doi: 10.1002/pst.2432. Epub 2024 Aug 13.
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.
国际人用药品注册技术协调会E9（R1）指南概述了估计量框架，该框架使临床试验的规划、设计、实施、分析和解释保持一致。文献中已概述了在临床试验中使用此框架的益处和价值，并就如何选择估计量和定义估计量属性提供了指导。尽管在临床试验中实施估计量方面已取得进展，但据我们所知，尚未有关于临床试验中的估计量应满足哪些基本原则才能得到明确界定并与ICH E9（R1）指南中提出的理念相一致的公开讨论。因此，在这篇观点文章中，我们提出了定义估计量的四项关键原则。这些原则构成了明确界定治疗效果的基础，反映了估计量的思维过程。我们希望这一观点将补充ICH E9（R1），并激发关于临床试验中的估计量应具备哪些基本属性的讨论，且此类讨论最终将提高临床试验中定义估计量的清晰度和精确性。
