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Treatment estimands in clinical trials of patients hospitalised for COVID-19: ensuring trials ask the right questions

Kahan, BC; Morris, TP; White, IR; Tweed, CD; Cro, S; Dahly, D; Pham, TM; ... Carpenter, JR; + view all (2020) Treatment estimands in clinical trials of patients hospitalised for COVID-19: ensuring trials ask the right questions. BMC Medicine , 18 (1) , Article 286. 10.1186/s12916-020-01737-0. Green open access

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Abstract

When designing a clinical trial, explicitly defining the treatment estimands of interest (that which is to be estimated) can help to clarify trial objectives and ensure the questions being addressed by the trial are clinically meaningful. There are several challenges when defining estimands. Here, we discuss a number of these in the context of trials of treatments for patients hospitalised with COVID-19 and make suggestions for how estimands should be defined for key outcomes. We suggest that treatment effects should usually be measured as differences in proportions (or risk or odds ratios) for outcomes such as death and requirement for ventilation, and differences in means for outcomes such as the number of days ventilated. We further recommend that truncation due to death should be handled differently depending on whether a patient- or resource-focused perspective is taken; for the former, a composite approach should be used, while for the latter, a while-alive approach is preferred. Finally, we suggest that discontinuation of randomised treatment should be handled from a treatment policy perspective, where non-adherence is ignored in the analysis (i.e. intention to treat).

Type: Article
Title: Treatment estimands in clinical trials of patients hospitalised for COVID-19: ensuring trials ask the right questions
Open access status: An open access version is available from UCL Discovery
DOI: 10.1186/s12916-020-01737-0
Publisher version: https://doi.org/10.1186/s12916-020-01737-0
Language: English
Additional information: © The Author(s) 2020. This article is licensed under a Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/).
Keywords: COVID-19, Estimand, Randomised trial, Intercurrent events, Truncation-by-death
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology > MRC Clinical Trials Unit at UCL
URI: https://discovery.ucl.ac.uk/id/eprint/10109891
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