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Taking the Analysis of Trial-Based Economic Evaluations to the Next Level: The Importance of Accounting for Clustering

Author

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  • Mohamed El Alili

    (Vrije Universiteit Amsterdam, Amsterdam Public Health Research Institute)

  • Johanna M. Dongen

    (Vrije Universiteit Amsterdam, Amsterdam Public Health Research Institute
    Vrije Universiteit Amsterdam, Amsterdam Movement Sciences Research Institute)

  • Keith S. Goldfeld

    (NYU School of Medicine)

  • Martijn W. Heymans

    (Amsterdam UMC, Location VU, Amsterdam Public Health Research Institute)

  • Maurits W. Tulder

    (Vrije Universiteit Amsterdam, Amsterdam Public Health Research Institute
    Vrije Universiteit Amsterdam, Amsterdam Movement Sciences Research Institute
    Aarhus University Hospital)

  • Judith E. Bosmans

    (Vrije Universiteit Amsterdam, Amsterdam Public Health Research Institute)

Abstract

Objectives The aim of this study was to assess the performance and impact of multilevel modelling (MLM) compared with ordinary least squares (OLS) regression in trial-based economic evaluations with clustered data. Methods Three thousand datasets with balanced and unbalanced clusters were simulated with correlation coefficients between costs and effects of − 0.5, 0, and 0.5, and intraclass correlation coefficients (ICCs) varying between 0.05 and 0.30. Each scenario was analyzed using both MLM and OLS. Statistical uncertainty around MLM and OLS estimates was estimated using bootstrapping. Performance measures were estimated and compared between approaches, including bias, root mean squared error (RMSE) and coverage probability. Cost and effect differences, and their corresponding confidence intervals and standard errors, incremental cost-effectiveness ratios, incremental net-monetary benefits and cost-effectiveness acceptability curves were compared. Results Cost-effectiveness outcomes were similar between OLS and MLM. MLM produced larger statistical uncertainty and coverage probabilities closer to nominal levels than OLS. The higher the ICC, the larger the effect on statistical uncertainty between MLM and OLS. Significant cost-effectiveness outcomes as estimated by OLS became non-significant when estimated by MLM. At all ICCs, MLM resulted in lower probabilities of cost effectiveness than OLS, and this difference became larger with increasing ICCs. Performance measures and cost-effectiveness outcomes were similar across scenarios with varying correlation coefficients between costs and effects. Conclusions Although OLS produced similar cost-effectiveness outcomes, it substantially underestimated the amount of variation in the data compared with MLM. To prevent suboptimal conclusions and a possible waste of scarce resources, it is important to use MLM in trial-based economic evaluations when data are clustered.

Suggested Citation

  • Mohamed El Alili & Johanna M. Dongen & Keith S. Goldfeld & Martijn W. Heymans & Maurits W. Tulder & Judith E. Bosmans, 2020. "Taking the Analysis of Trial-Based Economic Evaluations to the Next Level: The Importance of Accounting for Clustering," PharmacoEconomics, Springer, vol. 38(11), pages 1247-1261, November.
    • Handle: RePEc:spr:pharme:v:38:y:2020:i:11:d:10.1007_s40273-020-00946-y
    DOI: 10.1007/s40273-020-00946-y
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    References listed on IDEAS

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    1. Elisabeth Fenwick & Bernie J. O'Brien & Andrew Briggs, 2004. "Cost‐effectiveness acceptability curves – facts, fallacies and frequently asked questions," Health Economics, John Wiley & Sons, Ltd., vol. 13(5), pages 405-415, May.
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    5. Borislava Mihaylova & Andrew Briggs & Anthony O'Hagan & Simon G. Thompson, 2011. "Review of statistical methods for analysing healthcare resources and costs," Health Economics, John Wiley & Sons, Ltd., vol. 20(8), pages 897-916, August.
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    Cited by:

    1. Thomas Lung & Lei Si & Richard Hooper & Gian Luca Di Tanna, 2021. "Health Economic Evaluation Alongside Stepped Wedge Trials: A Methodological Systematic Review," PharmacoEconomics, Springer, vol. 39(1), pages 63-80, January.
    2. Ângela Jornada Ben & Johanna M. Dongen & Mohamed El Alili & Martijn W. Heymans & Jos W. R. Twisk & Janet L. MacNeil-Vroomen & Maartje Wit & Susan E. M. Dijk & Teddy Oosterhuis & Judith E. Bosmans, 2023. "The handling of missing data in trial-based economic evaluations: should data be multiply imputed prior to longitudinal linear mixed-model analyses?," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 24(6), pages 951-965, August.

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