All I want for Christmas…is a precisely defined research question
File(s)
Published version
Author(s)
Cro, Suzie
Phillips, Rachel
Type
Journal Article
Abstract
This Christmas, five years on from the publication of the International Conference on
Harmonisation E9 Addendum on Estimands and Sensitivity Analysis in Clinical Trials
(ICH E9 (R1) addendum), we describe a fictitious, festive randomised controlled trial
conducted by Santa Claus to answer the question: does a reindeer’s pre-flight meal
impact performance?
It is important that all clinical trials are designed, conducted and analysed to address
clear and meaningful research questions. To address this, the ICH E9 (R1) addendum
introduced the estimand framework. These guidelines clarified the importance of
precisely defining the treatment effects targeted in trials of health care interventions by
using estimands. However, estimands can be challenging to understand and
appreciate. We use this fictitious example to explain what an estimand is and
demonstrate why there is a need for their use, turning Santa’s initial vague research
question into a more precise question in line with the addendum by using estimands.
Whilst light-hearted in nature we hope that this article will facilitate application of the
international trial guidelines (ICH E9 (R1)) that are now adopted worldwide and require
the use of estimands.
Harmonisation E9 Addendum on Estimands and Sensitivity Analysis in Clinical Trials
(ICH E9 (R1) addendum), we describe a fictitious, festive randomised controlled trial
conducted by Santa Claus to answer the question: does a reindeer’s pre-flight meal
impact performance?
It is important that all clinical trials are designed, conducted and analysed to address
clear and meaningful research questions. To address this, the ICH E9 (R1) addendum
introduced the estimand framework. These guidelines clarified the importance of
precisely defining the treatment effects targeted in trials of health care interventions by
using estimands. However, estimands can be challenging to understand and
appreciate. We use this fictitious example to explain what an estimand is and
demonstrate why there is a need for their use, turning Santa’s initial vague research
question into a more precise question in line with the addendum by using estimands.
Whilst light-hearted in nature we hope that this article will facilitate application of the
international trial guidelines (ICH E9 (R1)) that are now adopted worldwide and require
the use of estimands.
Date Issued
2024-12-14
Date Acceptance
2024-10-30
Citation
Trials, 2024, 25
ISSN
1745-6215
Publisher
BMC
Journal / Book Title
Trials
Volume
25
Copyright Statement
© The Author(s) 2024. Open Access This article is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License, which permits any non-commercial use, sharing, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if you modified the licensed material. You do not have permission under this licence to share adapted material derived from this article or parts of it. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc-nd/4.0/.
Identifier
https://trialsjournal.biomedcentral.com/articles/10.1186/s13063-024-08604-w
Publication Status
Published
Article Number
784
Date Publish Online
2024-12-14