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Handling Missing Data in Within-Trial Cost-Effectiveness Analysis: A Review with Future Recommendations


  • Andrea Gabrio

    () (University College London)

  • Alexina J. Mason

    (London School of Hygiene and Tropical Medicine)

  • Gianluca Baio

    (University College London)


Abstract Cost-effectiveness analyses (CEAs) alongside randomised controlled trials (RCTs) are increasingly designed to collect resource use and preference-based health status data for the purpose of healthcare technology assessment. However, because of the way these measures are collected, they are prone to missing data, which can ultimately affect the decision of whether an intervention is good value for money. We examine how missing cost and effect outcome data are handled in RCT-based CEAs, complementing a previous review (covering 2003–2009, 88 articles) with a new systematic review (2009–2015, 81 articles) focussing on two different perspectives. First, we provide guidelines on how the information about missingness and related methods should be presented to improve the reporting and handling of missing data. We propose to address this issue by means of a quality evaluation scheme, providing a structured approach that can be used to guide the collection of information, elicitation of the assumptions, choice of methods and considerations of possible limitations of the given missingness problem. Second, we review the description of the missing data, the statistical methods used to deal with them and the quality of the judgement underpinning the choice of these methods. Our review shows that missing data in within-RCT CEAs are still often inadequately handled and the overall level of information provided to support the chosen methods is rarely satisfactory.

Suggested Citation

  • 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.
  • Handle: RePEc:spr:pharmo:v:1:y:2017:i:2:d:10.1007_s41669-017-0015-6
    DOI: 10.1007/s41669-017-0015-6

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    References listed on IDEAS

    1. Andrew Briggs & Taane Clark & Jane Wolstenholme & Philip Clarke, 2003. "Missing.... presumed at random: cost-analysis of incomplete data," Health Economics, John Wiley & Sons, Ltd., vol. 12(5), pages 377-392.
    2. 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.
    3. Gerald Richardson & Andrea Manca, 2004. "Calculation of quality adjusted life years in the published literature: a review of methodology and transparency," Health Economics, John Wiley & Sons, Ltd., vol. 13(12), pages 1203-1210.
    4. 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.
    5. Nicholas Graves & Damian Walker & Rosalind Raine & Andrew Hutchings & Jennifer A. Roberts, 2002. "Cost data for individual patients included in clinical studies: no amount of statistical analysis can compensate for inadequate costing methods," Health Economics, John Wiley & Sons, Ltd., vol. 11(8), pages 735-739.
    6. 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.
    7. 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.
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    Blog mentions

    As found by, the blog aggregator for Economics research:
    1. James Altunkaya’s journal round-up for 3rd September 2018
      by jamesaltunkaya in The Academic Health Economists' Blog on 2018-09-03 11:00:24
    2. Thesis Thursday: Andrea Gabrio
      by Chris Sampson in The Academic Health Economists' Blog on 2019-09-19 06:00:59


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