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Perspective

Use of the incremental cost-effectiveness ratio for decision-making policies—what is the problem? A perspective paper

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Pages 913-918 | Received 13 Oct 2021, Accepted 07 Apr 2022, Published online: 05 May 2022

References

  • For example, the UK’s NICE, Canada’s CADTH, Australia’s PBAC. New Zealand’s PHARMAC
  • National Institute for Health and Care Excellence. Interim process and methods of the highly specialised technologies programme. Updated to reflect 2017 changes. National Institute for Health and Care Excellence 2017 [cited 2019 Sep 26] Available from 2019 Sep 26: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-highly-specialised-technologies-guidance
  • National Institute for Health and Care Excellence. February 2019 Final evaluation document. Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2. National Institute for Health and Care Excellence [cited 2019 Sep 26] Available from 2019 Sep 26: https://www.nice.org.uk/guidance/gid-hst10008/documents/final-evaluation-determination-document
  • National Institute for Health and Care Excellence. Lumacaftor-ivacaftor for treating cystic fibrosis homozygous for the F508del mutation. National Institute for Health and Care Excellence 2016 Jul 27 [cited 2019 Sep 26] Available from 2019 Sep 26: https://www.nice.org.uk/guidance/ta398
  • Staines R NHS does not value CF patients, says Vertex CEO in letter to PM. Pharmaphorum 2018 July 10 [cited 2019 Sep 26] Available from 2019 Sep 26: https://pharmaphorum.com/news/nhs-does-not-value-cf-patients-says-vertex-ceo-in-letter-to-pm/
  • National Institute for Health and Care Excellence. Appraisal consultation document. Nusinersen for treating spinal muscular atrophy. National Institute for Health and Care Excellence 2018 August [cited 2019 Sep 26] Available from 2019 Sep 26: https://www.nice.org.uk/guidance/ta588/documents/appraisal-consultation-document
  • National Institute for Health and Care Excellence. Nusinersen for treating spinal muscular atrophy. National Institute for Health and Care Excellence 2019 Jul 24 [cited 2019 Sep 26] Available from 2019 Sep 26: https://www.nice.org.uk/guidance/ta588
  • Moro D, Schlander S, Telser H, et al. Evaluating Discrete Choice Experiment Willingness to Pay [DCE-WTP] analysis, and Relative Social Willingness to Pay [RS-WTP] analysis in a Health Technology Assessment of a treatment for an ultra-rare childhood disease [CLN2]. Expert Rev Pharmacoecon Outcomes Res. 2021 doi:10.1080/14737167.2022.2014324.
  • Torrance GW, Thomas WH, Sackett DL. A utility maximization model for evaluation of health care programs. Health Serv Res. 1972;7(2):118–133.
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  • Brouwer WB, Culyer AJ, van Exel NJ, et al. Welfarism vs. extra-welfarism. J Health Econ. 2008 Mar;27:325–338.
  • **Schlander M, Garattini S, Holm S, et al. Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders. J Comp Eff Res. 2014 Jul;3:399–422.
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  • While not discussed here, it should be noted that combining quantity and quality of life into a single measure also creates its own difficulties – should they be given equal weight when combined or should one element have higher weight than the other?
  • It remains unstated in Canada whether or not treatments for rare diseases are also assessed against a different threshold but, again, it is widely considered that they are. The difference between cancer drugs and the rest is, however, less disputable
  • *Schlander M, Schwarz O, Hernandez D, et al. New estimates of the willingness-to-pay for a statistical life year. Value In Health 2018, V21-S1, S111.
  • Berdud M, Drummond MF, Towse A (2018 Accessed 20 November 2020) Establishing a reasonable price for an orphan drug. OHE Research Paper. Available from https://www.ohe.org/publications/establishing-reasonable-price-orphan-drug.
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  • Hansen P, Devlin N. Multi-criteria decision analysis (MCDA) in healthcare decision-making 2019. Oxford Research Encyclopedia of Economics and Finance. Retrieved 2021 Dec 9, Oxford Research Encyclopedia of Economics and Finance https://oxfordre.com/economics/view/10.1093/acrefore/9780190625979.001.0001/acrefore-9780190625979-e-98 doi:10.1093/acrefore/9780190625979.013.98
  • See Thokala P, Devlin N, Marsh K, et al. Multiple criteria decision analysis for health care decision making-an introduction: report 1 of the ISPOR MCDA Emerging Good Practices Task Force. Value Health. 2016;19:1–13. Albeit, the ISPOR task force continues to be equivocal on recommending the use of MCDA
  • Richardson J, Schlander M. Health technology assessment (HTA) and economic evaluation: efficiency or fairness first. J Mark Access Health Policy. 2019;7:1557981.
  • Johnson FR. Why not real economics? Pharmacoeconomics. 2012;30(2):127–131.
  • Linley WG, Hughes DA. Societal views on NICE, cancer drugs fund and value-based pricing criteria for prioritising medicines: a cross-sectional survey of 4118 adults in Great Britain. Health Econ. 2013 Aug;22(8):948–964.

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