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A Guide to Handling Missing Data in Cost-Effectiveness Analysis Conducted Within Randomised Controlled Trials


  • Rita Faria


  • Manuel Gomes
  • David Epstein
  • Ian White


Missing data are a frequent problem in cost-effectiveness analysis (CEA) within a randomised controlled trial. Inappropriate methods to handle missing data can lead to misleading results and ultimately can affect the decision of whether an intervention is good value for money. This article provides practical guidance on how to handle missing data in within-trial CEAs following a principled approach: (i) the analysis should be based on a plausible assumption for the missing data mechanism, i.e. whether the probability that data are missing is independent of or dependent on the observed and/or unobserved values; (ii) the method chosen for the base-case should fit with the assumed mechanism; and (iii) sensitivity analysis should be conducted to explore to what extent the results change with the assumption made. This approach is implemented in three stages, which are described in detail: (1) descriptive analysis to inform the assumption on the missing data mechanism; (2) how to choose between alternative methods given their underlying assumptions; and (3) methods for sensitivity analysis. The case study illustrates how to apply this approach in practice, including software code. The article concludes with recommendations for practice and suggestions for future research. Copyright The Author(s) 2014

Suggested Citation

  • Rita Faria & Manuel Gomes & David Epstein & Ian White, 2014. "A Guide to Handling Missing Data in Cost-Effectiveness Analysis Conducted Within Randomised Controlled Trials," PharmacoEconomics, Springer, vol. 32(12), pages 1157-1170, December.
  • Handle: RePEc:spr:pharme:v:32:y:2014:i:12:p:1157-1170
    DOI: 10.1007/s40273-014-0193-3

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    References listed on IDEAS

    1. Andrew R. Willan & Andrew H. Briggs & Jeffrey S. Hoch, 2004. "Regression methods for covariate adjustment and subgroup analysis for non‐censored cost‐effectiveness data," Health Economics, John Wiley & Sons, Ltd., vol. 13(5), pages 461-475, May.
    2. Jan B. Oostenbrink & Maiwenn J. Al, 2005. "The analysis of incomplete cost data due to dropout," Health Economics, John Wiley & Sons, Ltd., vol. 14(8), pages 763-776, August.
    3. David K. Blough & Scott Ramsey & Sean D. Sullivan & Roger Yusen, 2009. "The impact of using different imputation methods for missing quality of life scores on the estimation of the cost‐effectiveness of lung‐volume‐reduction surgery," Health Economics, John Wiley & Sons, Ltd., vol. 18(1), pages 91-101, January.
    4. Paul C. Lambert & Lucinda J. Billingham & Nicola J. Cooper & Alex J. Sutton & Keith R. Abrams, 2008. "Estimating the cost‐effectiveness of an intervention in a clinical trial when partial cost information is available: a Bayesian approach," Health Economics, John Wiley & Sons, Ltd., vol. 17(1), pages 67-81, January.
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    1. Ifigeneia Mavranezouli & Joran Lokkerbol, 2017. "A Systematic Review and Critical Appraisal of Economic Evaluations of Pharmacological Interventions for People with Bipolar Disorder," PharmacoEconomics, Springer, vol. 35(3), pages 271-296, March.
    2. Laura Pirhonen & Kristian Bolin & Elisabeth Hansson Olofsson & Andreas Fors & Inger Ekman & Karl Swedberg & Hanna Gyllensten, 2019. "Person-Centred Care in Patients with Acute Coronary Syndrome: Cost-Effectiveness Analysis Alongside a Randomised Controlled Trial," PharmacoEconomics - Open, Springer, vol. 3(4), pages 495-504, December.
    3. Deidda, Manuela & Geue, Claudia & Kreif, Noemi & Dundas, Ruth & McIntosh, Emma, 2019. "A framework for conducting economic evaluations alongside natural experiments," Social Science & Medicine, Elsevier, vol. 220(C), pages 353-361.
    4. Bernhard Michalowsky & Wolfgang Hoffmann & Kevin Kennedy & Feng Xie, 2020. "Is the whole larger than the sum of its parts? Impact of missing data imputation in economic evaluation conducted alongside randomized controlled trials," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 21(5), pages 717-728, July.
    5. Alexina J. Mason & Manuel Gomes & Richard Grieve & James R. Carpenter, 2018. "A Bayesian framework for health economic evaluation in studies with missing data," Health Economics, John Wiley & Sons, Ltd., vol. 27(11), pages 1670-1683, November.
    6. Abualbishr Alshreef & Allan J. Wailoo & Steven R. Brown & James P. Tiernan & Angus J. M. Watson & Katie Biggs & Mike Bradburn & Daniel Hind, 2017. "Cost-Effectiveness of Haemorrhoidal Artery Ligation versus Rubber Band Ligation for the Treatment of Grade II–III Haemorrhoids: Analysis Using Evidence from the HubBLe Trial," PharmacoEconomics - Open, Springer, vol. 1(3), pages 175-184, September.
    7. Baptiste Leurent & Manuel Gomes & James R. Carpenter, 2018. "Missing data in trial‐based cost‐effectiveness analysis: An incomplete journey," Health Economics, John Wiley & Sons, Ltd., vol. 27(6), pages 1024-1040, June.
    8. Jackie, Yenerall & Wen, You & George, Davis & Paul, Estabrooks, 2015. "Examining Ways to Handle Non-Random Missingness in CEA through Econometric and Statistics Lenses," 2015 AAEA & WAEA Joint Annual Meeting, July 26-28, San Francisco, California 205690, Agricultural and Applied Economics Association.
    9. Janet MacNeil Vroomen & Iris Eekhout & Marcel G. Dijkgraaf & Hein van Hout & Sophia E. de Rooij & Martijn W. Heymans & Judith E. Bosmans, 2016. "Multiple imputation strategies for zero-inflated cost data in economic evaluations: which method works best?," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 17(8), pages 939-950, November.
    10. William Hollingworth & Christopher G. Fawsitt & Padraig Dixon & Larisa Duffy & Ricardo Araya & Tim J. Peters & Howard Thom & Nicky J. Welton & Nicola Wiles & Glyn Lewis, 2020. "Cost-Effectiveness of Sertraline in Primary Care According to Initial Severity and Duration of Depressive Symptoms: Findings from the PANDA RCT," PharmacoEconomics - Open, Springer, vol. 4(3), pages 427-438, September.
    11. Baptiste Leurent & Manuel Gomes & Rita Faria & Stephen Morris & Richard Grieve & James R. Carpenter, 2018. "Sensitivity Analysis for Not-at-Random Missing Data in Trial-Based Cost-Effectiveness Analysis: A Tutorial," PharmacoEconomics, Springer, vol. 36(8), pages 889-901, August.
    12. Adriano Zanin Zambom & Gregory J. Matthews, 0. "Sure independence screening in the presence of missing data," Statistical Papers, Springer, vol. 0, pages 1-29.
    13. Andrea Gabrio & Alexina J. Mason & Gianluca Baio, 2017. "Handling Missing Data in Within-Trial Cost-Effectiveness Analysis: A Review with Future Recommendations," PharmacoEconomics - Open, Springer, vol. 1(2), pages 79-97, June.
    14. 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.
    15. Stephen Rocks & Daniela Berntson & Alejandro Gil-Salmerón & Mudathira Kadu & Nieves Ehrenberg & Viktoria Stein & Apostolos Tsiachristas, 0. "Cost and effects of integrated care: a systematic literature review and meta-analysis," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 0, pages 1-11.
    16. David M. Meads & Adam Martin & Alys Griffiths & Rachael Kelley & Byron Creese & Louise Robinson & Joanne McDermid & Rebecca Walwyn & Clive Ballard & Claire A. Surr, 2020. "Cost-Effectiveness of Dementia Care Mapping in Care-Home Settings: Evaluation of a Randomised Controlled Trial," Applied Health Economics and Health Policy, Springer, vol. 18(2), pages 237-247, April.

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