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

S. W. Quist (), A. J. Postma, K. J. Myrén, L. A. Jong and M. J. Postma
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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

The European Journal of Health Economics, 2023, vol. 24, issue 9, No 5, 1455-1472

Abstract: 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.

Keywords: Ravulizumab; Eculizumab; C5-inhibitors; Hemoglobinuria; Paroxysmal; Cost–benefit analysis; Costs and cost analysis (search for similar items in EconPapers)
JEL-codes: D61 I19 (search for similar items in EconPapers)
Date: 2023
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DOI: 10.1007/s10198-022-01556-5

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