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

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  • Rita Faria
  • Manuel Gomes
  • David Epstein
  • Ian White

Abstract

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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    2. 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.
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    7. 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.
    8. 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.
    9. Alexina J. Mason & Manuel Gomes & James Carpenter & Richard Grieve, 2021. "Flexible Bayesian longitudinal models for cost‐effectiveness analyses with informative missing data," Health Economics, John Wiley & Sons, Ltd., vol. 30(12), pages 3138-3158, December.
    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. Stephen Rocks & Daniela Berntson & Alejandro Gil-Salmerón & Mudathira Kadu & Nieves Ehrenberg & Viktoria Stein & Apostolos Tsiachristas, 2020. "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. 21(8), pages 1211-1221, November.
    13. Adriano Zanin Zambom & Gregory J. Matthews, 0. "Sure independence screening in the presence of missing data," Statistical Papers, Springer, vol. 0, pages 1-29.
    14. Adriano Zanin Zambom & Gregory J. Matthews, 2021. "Sure independence screening in the presence of missing data," Statistical Papers, Springer, vol. 62(2), pages 817-845, April.
    15. 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.
    16. 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.
    17. Helen A. Dakin & José Leal & Andrew Briggs & Philip Clarke & Rury R. Holman & Alastair Gray, 2020. "Accurately Reflecting Uncertainty When Using Patient-Level Simulation Models to Extrapolate Clinical Trial Data," Medical Decision Making, , vol. 40(4), pages 460-473, May.
    18. Padraig Dixon & William Hollingworth & Jonathan Benger & James Calvert & Melanie Chalder & Anna King & Stephanie MacNeill & Katherine Morton & Emily Sanderson & Sarah Purdy, 2020. "Observational Cost-Effectiveness Analysis Using Routine Data: Admission and Discharge Care Bundles for Patients with Chronic Obstructive Pulmonary Disease," PharmacoEconomics - Open, Springer, vol. 4(4), pages 657-667, December.
    19. 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.
    20. 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.
    21. Becky Pennington & Abualbishr Alshreef & Laura Flight & Andrew Metry & Edith Poku & Philip Hykin & Sobha Sivaprasad & A. Toby Prevost & Joana C. Vasconcelos & Caroline Murphy & Joanna Kelly & Yit Yang, 2021. "Cost Effectiveness of Ranibizumab vs Aflibercept vs Bevacizumab for the Treatment of Macular Oedema Due to Central Retinal Vein Occlusion: The LEAVO Study," PharmacoEconomics, Springer, vol. 39(8), pages 913-927, August.
    22. 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.
    23. 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.
    24. 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.

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