The estimands framework: a primer on the ICH E9(R1) addendum
File(s)
Published version
Author(s)
Kahan, Brennan
Hindley, Joanna
Edwards, Mark
Cro, Suzie
Morris, Tim
Type
Journal Article
Abstract
Estimands can be used in studies of healthcare interventions to clarify the interpretation of treatment effects. The addendum to the ICH E9 harmonised guideline on statistical principles for clinical trials (ICH E9(R1)) describes a framework for using estimands as part of a study. This paper provides an overview of the estimands framework, as outlined in the addendum, with the aim of explaining why estimands are beneficial; clarifying the terminology being used; and providing practical guidance on using estimands to decide the appropriate study design, data collection, and estimation methods. This article illustrates how to use the estimands framework by applying it to an ongoing trial in emergency bowel surgery. Estimands can be a useful way of clarifying the exact research question being evaluated in a study, both to avoid misinterpretation and to ensure that study methods are aligned to the overall study objectives.
Date Issued
2024-01-23
Date Acceptance
2023-11-07
Citation
BMJ: British Medical Journal, 2024, 384
ISSN
0959-535X
Publisher
BMJ Publishing Group
Journal / Book Title
BMJ: British Medical Journal
Volume
384
Copyright Statement
© 2024 The Author(s). This is an Open Access article distributed in accordance with the terms of the Creative Commons Attribution (CC BY 4.0) license, which permits others to distribute, remix, adapt and build upon this work, for commercial use, provided the original work is properly cited. See: http://creativecommons.org/licenses/by/4.0/.
License URL
Identifier
https://www.bmj.com/content/384/bmj-2023-076316
Subjects
Humans
Models, Statistical
Research Design
Humans
Models, Statistical
Research Design
1103 Clinical Sciences
1117 Public Health and Health Services
General & Internal Medicine
Publication Status
Published
Article Number
e076316
Date Publish Online
2024-01-23