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Estimating mean QALYs in trial-based cost-effectiveness analysis: the importance of controlling for baseline utility

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  • Andrea Manca

    (Centre for Health Economics, University of York, UK)

  • Neil Hawkins

    (Centre for Health Economics, University of York, UK)

  • Mark J. Sculpher

    (Centre for Health Economics, University of York, UK)

Abstract

In trial-based cost-effectiveness analysis baseline mean utility values are invariably imbalanced between treatment arms. A patient's baseline utility is likely to be highly correlated with their quality-adjusted life-years (QALYs) over the follow-up period, not least because it typically contributes to the QALY calculation. Therefore, imbalance in baseline utility needs to be accounted for in the estimation of mean differential QALYs, and failure to control for this imbalance can result in a misleading incremental cost-effectiveness ratio. This paper discusses the approaches that have been used in the cost-effectiveness literature to estimate absolute and differential mean QALYs alongside randomised trials, and illustrates the implications of baseline mean utility imbalance for QALY calculation. Using data from a recently conducted trial-based cost-effectiveness study and a micro-simulation exercise, the relative performance of alternative estimators is compared, showing that widely used methods to calculate differential QALYs provide incorrect results in the presence of baseline mean utility imbalance regardless of whether these differences are formally statistically significant. It is demonstrated that multiple regression methods can be usefully applied to generate appropriate estimates of differential mean QALYs and an associated measure of sampling variability, while controlling for differences in baseline mean utility between treatment arms in the trial. Copyright © 2004 John Wiley & Sons, Ltd.

Suggested Citation

  • Andrea Manca & Neil Hawkins & Mark J. Sculpher, 2005. "Estimating mean QALYs in trial-based cost-effectiveness analysis: the importance of controlling for baseline utility," Health Economics, John Wiley & Sons, Ltd., vol. 14(5), pages 487-496.
  • Handle: RePEc:wly:hlthec:v:14:y:2005:i:5:p:487-496 DOI: 10.1002/hec.944
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    References listed on IDEAS

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    1. Daniel Polsky & Henry A. Glick & Richard Willke & Kevin Schulman, 1997. "Confidence Intervals for Cost-Effectiveness Ratios: A Comparison of Four Methods," Health Economics, John Wiley & Sons, Ltd., vol. 6(3), pages 243-252.
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    3. Elisabeth Fenwick & Karl Claxton & Mark Sculpher, 2001. "Representing uncertainty: the role of cost-effectiveness acceptability curves," Health Economics, John Wiley & Sons, Ltd., vol. 10(8), pages 779-787.
    4. Jeffrey S. Hoch & Andrew H. Briggs & Andrew R. Willan, 2002. "Something old, something new, something borrowed, something blue: a framework for the marriage of health econometrics and cost-effectiveness analysis," Health Economics, John Wiley & Sons, Ltd., vol. 11(5), pages 415-430.
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    Cited by:

    1. David Meads & Andrea Marshall & Claire Hulme & Janet Dunn & Hugo Ford, 2016. "The Cost Effectiveness of Docetaxel and Active Symptom Control versus Active Symptom Control Alone for Refractory Oesophagogastric Adenocarcinoma: Economic Analysis of the COUGAR-02 Trial," PharmacoEconomics, Springer, vol. 34(1), pages 33-42, January.
    2. David M. Meads & Andrea Marshall & Claire T. Hulme & Janet A. Dunn & Hugo E. R. Ford, 2016. "The Cost Effectiveness of Docetaxel and Active Symptom Control versus Active Symptom Control Alone for Refractory Oesophagogastric Adenocarcinoma: Economic Analysis of the COUGAR-02 Trial," PharmacoEconomics, Springer, vol. 34(1), pages 33-42, January.
    3. McCarthy, Ian M., 2016. "Eliminating composite bias in treatment effects estimates: Applications to quality of life assessment," Journal of Health Economics, Elsevier, vol. 50(C), pages 47-58.
    4. Darren K. Burns & Edward C. F. Wilson & Paula Browne & Sandra Olive & Allan Clark & Penny Galey & Emma Dix & Helene Woodhouse & Sue Robinson & Andrew Wilson, 2016. "The Cost Effectiveness of Maintenance Schedules Following Pulmonary Rehabilitation in Patients with Chronic Obstructive Pulmonary Disease: An Economic Evaluation Alongside a Randomised Controlled Tria," Applied Health Economics and Health Policy, Springer, vol. 14(1), pages 105-115, February.
    5. Matthew Franklin & Sarah Davis & Michelle Horspool & Wei Sun Kua & Steven Julious, 2017. "Economic Evaluations Alongside Efficient Study Designs Using Large Observational Datasets: the PLEASANT Trial Case Study," PharmacoEconomics, Springer, vol. 35(5), pages 561-573, May.
    6. Francisco Jódar-Sánchez & Amaia Malet-Larrea & José Martín & Leticia García-Mochón & M. López del Amo & Fernando Martínez-Martínez & Miguel Gastelurrutia-Garralda & Victoria García-Cárdenas & Daniel S, 2015. "Cost-Utility Analysis of a Medication Review with Follow-Up Service for Older Adults with Polypharmacy in Community Pharmacies in Spain: The conSIGUE Program," PharmacoEconomics, Springer, vol. 33(6), pages 599-610, June.
    7. Nadia Yakhelef & Martine Audibert & Bruno Peirera & Antoine Mons & Emmanuel Chabert, 2015. "Cost-utility Analysis of Vertebroplasty versus Thoracolumbosacral Orthosis in the Treatment of Traumatic Vertebral Fractures," Working Papers halshs-01241824, HAL.
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    9. Theodoros Mantopoulos & Paul M. Mitchell & Nicky J. Welton & Richard McManus & Lazaros Andronis, 2016. "Choice of statistical model for cost-effectiveness analysis and covariate adjustment: empirical application of prominent models and assessment of their results," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 17(8), pages 927-938, November.
    10. Nadine Berndt & Catherine Bolman & Lilian Lechner & Wendy Max & Aart Mudde & Hein Vries & Silvia Evers, 2016. "Economic evaluation of a telephone- and face-to-face-delivered counseling intervention for smoking cessation in patients with coronary heart disease," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 17(3), pages 269-285, April.
    11. Melina Dritsaki & Felix Achana & James Mason & Stavros Petrou, 2017. "Methodological Issues Surrounding the Use of Baseline Health-Related Quality of Life Data to Inform Trial-Based Economic Evaluations of Interventions Within Emergency and Critical Care Settings: A Sys," PharmacoEconomics, Springer, vol. 35(5), pages 501-515, May.
    12. Nadia YAKHELEF & Martine AUDIBERT & Bruno PEIRERA & Antoine MONS & Emmanuel CHABERT, 2015. "Cost-utility Analysis of Vertebroplasty versus Thoracolumbosacral Orthosis in the Treatment of Traumatic Vertebral Fractures," Working Papers 201534, CERDI.
    13. Danielle Brunenberg & Gwenn Wetzels & Patricia Nelemans & Carmen Dirksen & Johan Severens & Henri Stoffers & Jan Schouten & Martin Prins & Peter Leeuw & Manuela Joore, 2007. "Cost Effectiveness of an Adherence-Improving Programme in Hypertensive Patients," PharmacoEconomics, Springer, vol. 25(3), pages 239-251, March.
    14. Ian M. McCarthy, 2015. "Putting the Patient in Patient Reported Outcomes: A Robust Methodology for Health Outcomes Assessment," Health Economics, John Wiley & Sons, Ltd., vol. 24(12), pages 1588-1603, December.
    15. Henderson, Catherine & Knapp, Martin & Fernández, José-Luis & Beecham, Jennifer & Hirani, Shashivadan P. & Beynon, Michelle & Cartwright, Martin & Rixon, Lorna & Doll, Helen & Bower, Peter & Steventon, 2014. "Cost-effectiveness of telecare for people with social care needs: the Whole Systems Demonstrator cluster randomised trial," LSE Research Online Documents on Economics 57270, London School of Economics and Political Science, LSE Library.
    16. Ian M. McCarthy, 2014. "Eliminating Aggregation Bias when Estimating Treatment Effects on Combined Outcomes with Applications to Quality of Life Assessment," Emory Economics 1409, Department of Economics, Emory University (Atlanta).
    17. Rachael Hunter & Gianluca Baio & Thomas Butt & Stephen Morris & Jeff Round & Nick Freemantle, 2015. "An Educational Review of the Statistical Issues in Analysing Utility Data for Cost-Utility Analysis," PharmacoEconomics, Springer, vol. 33(4), pages 355-366, April.
    18. repec:spr:pharmo:v:1:y:2017:i:3:d:10.1007_s41669-017-0023-6 is not listed on IDEAS
    19. Lars Oddershede & Simon Walker & Wolfgang Stöhr & David T. Dunn & Alejandro Arenas-Pinto & Nicholas I. Paton & Mark Sculpher, 2016. "Cost Effectiveness of Protease Inhibitor Monotherapy Versus Standard Triple Therapy in the Long-Term Management of HIV Patients: Analysis Using Evidence from the PIVOT Trial," PharmacoEconomics, Springer, vol. 34(8), pages 795-804, August.
    20. Wanrudee Isaranuwatchai & Maureen Markle-Reid & Jeffrey Hoch, 2015. "Adjusting for Baseline Covariates in Net Benefit Regression: How You Adjust Matters," PharmacoEconomics, Springer, vol. 33(10), pages 1083-1090, October.
    21. Paddy Gillespie & Eamon O’Shea & Andrew Murphy & Susan Smith & Mary Byrne & Molly Byrne & Margaret Cupples, 2012. "Relative cost effectiveness of the SPHERE intervention in selected patient subgroups with existing coronary heart disease," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 13(4), pages 429-443, August.

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