Identifying compliant participants through data matching improved estimation of intervention efficacy: randomized trials with opt-in/opt-out

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Ng, Shu Kay
Byrnes, Joshua
Scuffham, Paul
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2019
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Abstract

OBECTIVE: We propose a data-matching approach to estimate intervention efficacy for randomised controlled trials (RCTs) when there is non-compliance to the allocated treatment with induced selection bias. STUDY DESIGN: and Settings: We considered a large RCT to compare healthcare costs and hospital length-of-stay 12 months post randomisation. Participants allocated to the intervention group were eligible to receive health-coaching and disease-management services. An opt-out approach was adopted for recruitment. Control-group participants received usual care but were allowed to opt-in to receive the intervention. Using "nearest-neighbour"-matched data, we identified compliant participants in both arms to estimate intervention efficacy. Results were compared with intention-to-treat (ITT), instrumental-variable (IV)-adjusted ITT, per-protocol (PP), and as-treated (AT) analyses. RESULTS: The ITT estimated an intervention effect of a 1.5% reduction in cost, but 56.7% of intervention-group participants did not receive health-coaching. The PP and AT found an increase in cost of 9.4% and 17.1%, respectively. The matching method estimated a 12.3% reduction in cost. After adjustment for baseline covariates, the intervention group had lower same-day admission cost (13.6%; 95% CI: 7.3%-20.0%; p<0.001) and shorter hospital stay (11.2%; 95% CI: 2.6%-19.9%; p=0.021). CONCLUSION: Opt-in/opt-out strategies in RCTs misled intervention comparisons and the matching approach improved estimation of intervention efficacy.

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Journal of Clinical Epidemiology

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115

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© 2019 Elsevier. Licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International Licence (http://creativecommons.org/licenses/by-nc-nd/4.0/) which permits unrestricted, non-commercial use, distribution and reproduction in any medium, providing that the work is properly cited.

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Health economics

Epidemiology

Non-compliance

health coaching

nearest-neighbour matching

randomised controlled trial

selection bias

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Ng, SK; Byrnes, J; Scuffham, P, Identifying compliant participants through data-matching improved estimation of intervention efficacy: Randomised trials with opt-in/opt-out strategies., Journal of Clinical Epidemiology, 2019, 115, pp. 125-132

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