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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
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 | |||||||||
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| Subjects: | R Medicine > R Medicine (General) R Medicine > RA Public aspects of medicine R Medicine > RC Internal medicine |
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| 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 |
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| 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: |
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| 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: |
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