Eliminating ambiguous treatment effects using estimands
File(s)kwad036.pdf (292.72 KB)
Published version
Author(s)
Kahan, Brennan
Cro, Suzie
Li, Fan
O Harhay, Michael
Type
Journal Article
Abstract
Most reported treatment effects in medical research studies are ambiguously defined, which can lead to misinterpretation of study results. This is because most studies do not attempt to describe what the treatment effect represents, and instead require readers to deduce this based on the reported statistical methods. However, this approach is fraught, as many methods provide counterintuitive results. For example, some methods include data from all patients, yet the resulting treatment effect applies only to a subset of patients, whereas other methods will exclude certain patients while results will apply to everyone. Additionally, some analyses provide estimates pertaining to hypothetical settings where patients never die or discontinue treatment. Herein we introduce estimands as a solution to the aforementioned problem. An estimand is a clear description of what the treatment effect represents, thus saving readers the necessity of trying to infer this from study methods and potentially getting it wrong. We provide examples of how estimands can remove ambiguity from reported treatment effects and describe their current use in practice. The crux of our argument is that readers should not have to infer what investigators are estimating; they should be told explicitly.
Date Issued
2023-06-01
Date Acceptance
2023-01-23
Citation
American Journal of Epidemiology, 2023, 192, pp.987-984
ISSN
0002-9262
Publisher
Oxford University Press
Start Page
987
End Page
984
Journal / Book Title
American Journal of Epidemiology
Volume
192
Copyright Statement
© The Author(s) 2023. Published by Oxford University Press on behalf of the Johns Hopkins Bloomberg School of Public Health
This is an Open Access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited.
This is an Open Access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited.
License URL
Identifier
https://academic.oup.com/aje/advance-article/doi/10.1093/aje/kwad036/7036583
Publication Status
Published
Article Number
kwad036
Date Publish Online
2023-02-14