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Assessing generalisability by location in trial‐based cost‐effectiveness analysis: the use of multilevel models

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  • Andrea Manca
  • Nigel Rice
  • Mark J. Sculpher
  • Andrew H. Briggs

Abstract

An Erratum has been published for this article in Health Economics 14(5) 2005, 486. Cost‐effectiveness analysis (CEA) in health care is increasingly conducted alongside multicentre and multinational randomised controlled clinical trials (RCTs). The increased use of stochastic CEA is designed to account for between‐patient sampling variability in cost‐effectiveness data assuming that observations are independently distributed. However, between‐location variability in cost‐effectiveness may result if there is a hierarchical structure in the data; that is, if there is correlation in costs and outcomes between patients recruited in particular locations. This may be expected in multi‐location trials given that centres and countries often differ in factors such as clinical practice, patient case‐mix and the unit costs of delivering health care. A failure to acknowledge this feature may lead to misleading conclusions in a trial‐based economic study. Multilevel modelling (MLM) is an analytical framework that can be used to handle hierarchical cost‐effectiveness data. Using data from a recently conducted economic analysis, this paper shows how multilevel modelling can be used to obtain (a) more appropriate estimates of the population average incremental cost‐effectiveness and associated standard errors compared to standard stochastic CEA; and (b) location‐specific estimates of incremental cost‐effectiveness which can be used to explore appropriately the variability between centres/countries of the cost‐effectiveness results. Copyright © 2004 John Wiley & Sons, Ltd.

Suggested Citation

  • Andrea Manca & Nigel Rice & Mark J. Sculpher & Andrew H. Briggs, 2005. "Assessing generalisability by location in trial‐based cost‐effectiveness analysis: the use of multilevel models," Health Economics, John Wiley & Sons, Ltd., vol. 14(5), pages 471-485, May.
    • Handle: RePEc:wly:hlthec:v:14:y:2005:i:5:p:471-485
    DOI: 10.1002/hec.914
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    Cited by:

    1. Manca, A & Austin, P. C, 2008. "Using propensity score methods to analyse individual patient-level cost-effectiveness data from observational studies," Health, Econometrics and Data Group (HEDG) Working Papers 08/20, HEDG, c/o Department of Economics, University of York.
    2. Mark J. Sculpher & Karl Claxton & Mike Drummond & Chris McCabe, 2006. "Whither trial‐based economic evaluation for health care decision making?," Health Economics, John Wiley & Sons, Ltd., vol. 15(7), pages 677-687, July.
    3. Md Abu Manju & Math J. J. M. Candel & Gerard J. P. van Breukelen, 2019. "SamP2CeT: an interactive computer program for sample size and power calculation for two-level cost-effectiveness trials," Computational Statistics, Springer, vol. 34(1), pages 47-70, March.
    4. Manju, Md Abu & Candel, Math J.J.M. & van Breukelen, Gerard J.P., 2021. "Robustness of cost-effectiveness analyses of cluster randomized trials assuming bivariate normality against skewed cost data," Computational Statistics & Data Analysis, Elsevier, vol. 157(C).
    5. Fernando Antonanzas & Roberto Rodríguez‐Ibeas & Carmelo Juárez & Florencia Hutter & Reyes Lorente & Mariola Pinillos, 2009. "Transferability indices for health economic evaluations: methods and applications," Health Economics, John Wiley & Sons, Ltd., vol. 18(6), pages 629-643, June.
    6. Oliveira, Monica D. & Bevan, Gwyn, 2008. "Modelling hospital costs to produce evidence for policies that promote equity and efficiency," European Journal of Operational Research, Elsevier, vol. 185(3), pages 933-947, March.
    7. Adrian Gheorghe & Tracy E Roberts & Jonathan C Ives & Benjamin R Fletcher & Melanie Calvert, 2013. "Centre Selection for Clinical Trials and the Generalisability of Results: A Mixed Methods Study," PLOS ONE, Public Library of Science, vol. 8(2), pages 1-9, February.
    8. Lange, Ian, 2008. "Contract Parameters' Impacts on Coal Prices," Stirling Economics Discussion Papers 2008-26, University of Stirling, Division of Economics.
    9. Thompson, Simon G. & Nixon, Richard M. & Grieve, Richard, 2006. "Addressing the issues that arise in analysing multicentre cost data, with application to a multinational study," Journal of Health Economics, Elsevier, vol. 25(6), pages 1015-1028, November.
    10. Christian E. H. Boehler & Joanne Lord, 2016. "Mind the Gap! A Multilevel Analysis of Factors Related to Variation in Published Cost-Effectiveness Estimates within and between Countries," Medical Decision Making, , vol. 36(1), pages 31-47, January.
    11. Pepijn Vemer & Maureen Rutten-van Mölken, 2013. "The Road Not Taken: Transferability Issues in Multinational Trials," PharmacoEconomics, Springer, vol. 31(10), pages 863-876, October.
    12. Aline Gauthier & Andrea Manca & Susan Anton, 2009. "Bayesian Modelling of Healthcare Resource Use in Multinational Randomized Clinical Trials," PharmacoEconomics, Springer, vol. 27(12), pages 1017-1029, December.
    13. Moreno, Elías & Girón, F.J. & Vázquez-Polo, F.J. & Negrín, M.A., 2012. "Optimal healthcare decisions: The importance of the covariates in cost–effectiveness analysis," European Journal of Operational Research, Elsevier, vol. 218(2), pages 512-522.
    14. Jeffrey Hoch & Carolyn Dewa, 2007. "Lessons from Trial-Based Cost-Effectiveness Analyses of Mental Health Interventions," PharmacoEconomics, Springer, vol. 25(10), pages 807-816, October.
    15. Carmen Selva-Sevilla & F Dámaso Fernández-Ginés & Manuel Cortiñas-Sáenz & Manuel Gerónimo-Pardo, 2021. "Cost-effectiveness analysis of domiciliary topical sevoflurane for painful leg ulcers," PLOS ONE, Public Library of Science, vol. 16(9), pages 1-18, September.
    16. Andrew Briggs, 2012. "Statistical Methods for Cost-effectiveness Analysis Alongside Clinical Trials," Chapters, in: Andrew M. Jones (ed.), The Elgar Companion to Health Economics, Second Edition, chapter 50, Edward Elgar Publishing.
    17. Hege Urdahl & Andrea Manca & Mark Sculpher, 2006. "Assessing Generalisability in Model-Based Economic Evaluation Studies," PharmacoEconomics, Springer, vol. 24(12), pages 1181-1197, December.
    18. Xin Sun & Thomas Faunce, 2008. "Decision-analytical modelling in health-care economic evaluations," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 9(4), pages 313-323, November.
    19. Andrew R. Willan & Matthew E. Kowgier, 2008. "Cost‐effectiveness analysis of a multinational RCT with a binary measure of effectiveness and an interacting covariate," Health Economics, John Wiley & Sons, Ltd., vol. 17(7), pages 777-791, July.
    20. Richard Grieve & Richard Nixon & Simon G. Thompson & John Cairns, 2007. "Multilevel models for estimating incremental net benefits in multinational studies," Health Economics, John Wiley & Sons, Ltd., vol. 16(8), pages 815-826, August.
    21. Richard M. Nixon & Simon G. Thompson, 2005. "Methods for incorporating covariate adjustment, subgroup analysis and between‐centre differences into cost‐effectiveness evaluations," Health Economics, John Wiley & Sons, Ltd., vol. 14(12), pages 1217-1229, December.
    22. Peter Makai & Willemijn Looman & Eddy Adang & René Melis & Elly Stolk & Isabelle Fabbricotti, 2015. "Cost-effectiveness of integrated care in frail elderly using the ICECAP-O and EQ-5D: does choice of instrument matter?," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 16(4), pages 437-450, May.

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