Muetze, Tobias, Bell, James, Englert, Stefan, Hougaard, Philip, Jackson, Dan, Lanius, Vivian and Ravn, Henrik (2024) Principles for defining estimands in clinical trials – a proposal. Pharmaceutical Statistics. ISSN 1539-16041539-1612

Abstract

The ICH E9(R1) guideline outlines the estimand framework to align 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 fulfil to be well-defined and consistent with the ideas presented in the ICH E9(R1) guideline. Therefore, in this Viewpoint article, we propose five key principles for defining an estimand. These principles form a basis for well-defined treatment effects reflecting the estimand thinking process. We hope that this Viewpoint will 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.

Item Type:	Article
Date Deposited:	28 Aug 2024 00:46
Last Modified:	28 Aug 2024 00:46
URI:	https://oak.novartis.com/id/eprint/53490