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Treatment effects may remain the same even when trial participants differed from the target population

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Bradburn, M. J., Lee, Ellen Cecilia, White, D. A., Hind, Daniel, Waugh, Norman, Cooke, D. D., Hopkins, D., Mansell, Peter and Heller, S. R. (2020) Treatment effects may remain the same even when trial participants differed from the target population. Journal of Clinical Epidemiology, 124 . pp. 126-138. doi:10.1016/j.jclinepi.2020.05.001 ISSN 0895-4356.

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Official URL: https://doi.org/10.1016/j.jclinepi.2020.05.001

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Abstract

Objective
RCTs have been criticised for lacking external validity. We assessed whether a trial in people with type I diabetes mellitus (T1DM) mirrored the wider population, and applied sample-weighting methods to assess the impact of differences on our trial's findings.
Study design and setting
The REPOSE trial was nested within a large UK cohort capturing demographic, clinical and quality of life (QoL) data for people with T1DM undergoing structured diabetes-specific education. We firstly assessed whether our RCT participants were comparable to this cohort using propensity score modelling. Following this we re-weighted the trial population to better match the wider cohort and re-estimated the treatment effect.
Results
Trial participants differed from the cohort in regards to sex, weight, HbA1c and also QoL and satisfaction with current treatment. Nevertheless, the treatment effects derived from alternative model weightings were similar to that of the original RCT.
Conclusions
Our RCT participants differed in composition to the wider population but the original findings were unaffected by sampling adjustments. We encourage investigators take steps to address criticisms of generalisability, but doing so is problematic: external data, even if available, may contain limited information and analyses can be susceptible to model misspecification.

Item Type: Journal Article
Subjects: R Medicine > R Medicine (General)
R Medicine > RA Public aspects of medicine
R Medicine > RC Internal medicine
Divisions: Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences
Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences > Population, Evidence & Technologies (PET)
Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School
Library of Congress Subject Headings (LCSH): Clinical trials, Diabetes -- Patients, Diabetes -- Research, Diabetes -- Treatment
Journal or Publication Title: Journal of Clinical Epidemiology
Publisher: Elsevier Inc.
ISSN: 0895-4356
Official Date: 2 August 2020
Dates:
DateEvent
2 August 2020Published
10 May 2020Available
4 May 2020Accepted
Volume: 124
Page Range: pp. 126-138
DOI: 10.1016/j.jclinepi.2020.05.001
Status: Peer Reviewed
Publication Status: Published
Access rights to Published version: Restricted or Subscription Access
Copyright Holders: © 2020 Elsevier Inc. All rights reserved
Date of first compliant deposit: 19 May 2020
Date of first compliant Open Access: 10 May 2021
RIOXX Funder/Project Grant:
Project/Grant IDRIOXX Funder NameFunder ID
08/107/01[NIHR] National Institute for Health Researchhttp://dx.doi.org/10.13039/501100000272
PG-0606-1184[NIHR] National Institute for Health Researchhttp://dx.doi.org/10.13039/501100000272

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