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PharmacoEconomics
Published in: PharmacoEconomics 5/2017

Open Access 01-05-2017 | Original Research Article

Economic Evaluations Alongside Efficient Study Designs Using Large Observational Datasets: the PLEASANT Trial Case Study

Authors: Matthew Franklin, Sarah Davis, Michelle Horspool, Wei Sun Kua, Steven Julious

Published in: PharmacoEconomics | Issue 5/2017

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Abstract

Background

Large observational datasets such as Clinical Practice Research Datalink (CPRD) provide opportunities to conduct clinical studies and economic evaluations with efficient designs.

Objectives

Our objectives were to report the economic evaluation methodology for a cluster randomised controlled trial (RCT) of a UK NHS-delivered public health intervention for children with asthma that was evaluated using CPRD and describe the impact of this methodology on results.

Methods

CPRD identified eligible patients using predefined asthma diagnostic codes and captured 1-year pre- and post-intervention healthcare contacts (August 2012 to July 2014). Quality-adjusted life-years (QALYs) 4 months post-intervention were estimated by assigning utility values to exacerbation-related contacts; a systematic review identified these utility values because preference-based outcome measures were not collected. Bootstrapped costs were evaluated 12 months post-intervention, both with 1-year regression-based baseline adjustment (BA) and without BA (observed).

Results

Of 12,179 patients recruited, 8190 (intervention 3641; control 4549) were evaluated in the primary analysis, which included patients who received the protocol-defined intervention and for whom CPRD data were available. The intervention’s per-patient incremental QALY loss was 0.00017 (bias-corrected and accelerated 95% confidence intervals [BCa 95% CI] –0.00051 to 0.00018) and cost savings were £14.74 (observed; BCa 95% CI –75.86 to 45.19) or £36.07 (BA; BCa 95% CI –77.11 to 9.67), respectively. The probability of cost savings was much higher when accounting for BA versus observed costs due to baseline cost differences between trial arms (96.3 vs. 67.3%, respectively).

Conclusion

Economic evaluations using data from a large observational database without any primary data collection is feasible, informative and potentially efficient.
Clinical Trials Registration Number: ISRCTN03000938.
Appendix
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Metadata
Title
Economic Evaluations Alongside Efficient Study Designs Using Large Observational Datasets: the PLEASANT Trial Case Study
Authors
Matthew Franklin
Sarah Davis
Michelle Horspool
Wei Sun Kua
Steven Julious
Publication date
01-05-2017
Publisher
Springer International Publishing
Published in
PharmacoEconomics / Issue 5/2017
Print ISSN: 1170-7690
Electronic ISSN: 1179-2027
DOI
https://doi.org/10.1007/s40273-016-0484-y

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