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Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands

Author

Listed:
  • S. W. Quist

    (Asc Academics)

  • A. J. Postma

    (Asc Academics)

  • K. J. Myrén

    (Alexion, AstraZeneca Rare Disease)

  • L. A. Jong

    (University Medical Center Groningen (UMCG), University of Groningen)

  • M. J. Postma

    (University Medical Center Groningen (UMCG), University of Groningen
    University of Groningen)

Abstract

Objectives The aim of this study was to evaluate the cost-effectiveness of ravulizumab compared with eculizumab for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) in the Netherlands. Methods A cost-effectiveness analysis was conducted based on a Markov cohort model simulating the course of patients with PNH with clinical symptom(s) indicative of high disease activity, or who are clinically stable after having been treated with eculizumab for at least the past six months. Costs, quality of life, and the incremental cost-effectiveness ratio (ICER) were estimated over a lifetime horizon from a Dutch societal perspective. Several additional analyses were performed, including a one-way sensitivity analysis, a probabilistic sensitivity analysis, and scenario analysis. Results When compared with eculizumab, ravulizumab saves €266,833 and 1.57 quality adjusted life years (QALYs) are gained, resulting in a dominant ICER. Drug costs account for the majority of the total costs in both intervention groups. Cost savings were driven by the difference in total treatment costs of ravulizumab compared with eculizumab caused by the reduced administration frequency, accounting for 98% of the total cost savings. The QALY gain with ravulizumab is largely attributable to the improved quality of life associated with less frequent infusions and BTH events. At a willingness-to-pay threshold of €20,000/QALY, there is a 76.6% probability that ravulizumab would be cost-effective. Conclusions The cost reduction and QALY gain associated with the lower rates of BTH and less frequent administration make ravulizumab a cost-saving and clinically beneficial substitute for eculizumab for adults with PNH in the Netherlands.

Suggested Citation

  • S. W. Quist & A. J. Postma & K. J. Myrén & L. A. Jong & M. J. Postma, 2023. "Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 24(9), pages 1455-1472, December.
  • Handle: RePEc:spr:eujhec:v:24:y:2023:i:9:d:10.1007_s10198-022-01556-5
    DOI: 10.1007/s10198-022-01556-5
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    References listed on IDEAS

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    1. Don Husereau & Michael Drummond & Stavros Petrou & Chris Carswell & David Moher & Dan Greenberg & Federico Augustovski & Andrew Briggs & Josephine Mauskopf & Elizabeth Loder, 2013. "Consolidated Health Economic Evaluation Reporting Standards (CHEERS) Statement," PharmacoEconomics, Springer, vol. 31(5), pages 361-367, May.
    2. John Devin Peipert & Austin G Kulasekararaj & Anna Gaya & Saskia M C Langemeijer & Susan Yount & F Ataulfo Gonzalez-Fernandez & Emilio Ojeda Gutierrez & Christa Martens & Amy Sparling & Kimberly A Web, 2020. "Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria," PLOS ONE, Public Library of Science, vol. 15(9), pages 1-12, September.
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    More about this item

    Keywords

    Ravulizumab; Eculizumab; C5-inhibitors; Hemoglobinuria; Paroxysmal; Cost–benefit analysis; Costs and cost analysis;
    All these keywords.

    JEL classification:

    • D61 - Microeconomics - - Welfare Economics - - - Allocative Efficiency; Cost-Benefit Analysis
    • I19 - Health, Education, and Welfare - - Health - - - Other

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