Abstract

A US Real-World study found the median recombinant coagulation factor VIII (rFVIII) international units (IUs) dispensed were between 10% and 45% higher with extended half-life (EHL) versus standard half-life (SHL) products. The objective was to evaluate the cost of SHL versus EHL rFVIII in Spain. A probabilistic model (second order Monte Carlo simulation) in a hypothetical cohort of 1,000 patients with hemophilia A and a time horizon of 1 year was carried out. The mean IUs dispensed quarterly with each rFVIII were taken from a Real-World study, conducted in two US databases, which included 776 patients with hemophilia A. The frequency of use of each recombinant rFVIII was obtained from sales estimation (IMS) of a 12-months period in Spain (BOE, 2018). rFVIII prices (€ 2019) were obtained from the Reference Price Order by the National Health System. A base case analysis, considering the use of rFVIII in the clinical practice in Spain and two sensitivity analyzes were performed. In the base case analysis, an average annual saving per patient treated with SHL of € 11,227 was obtained, with a 75.5% saving probability. The results were stable for sensitivity analyzes performed. In the event that there was no change in treatment from the SHL to the EHL, there would be a greater annual saving per patient (€ 53,078) with a 99.9% saving probability. When applying the rFVIII frequency of use in the clinical practice in the US, the annual savings per patient treated with SHL instead of EHL would amount to € 16,350, with a saving probability of 79.9%. The probabilistic model found higher expenditures over one year for hemophilia A patients using EHL versus SHL products. The use of SHL instead of EHL, would amount to cost savings to the National Health System in Spain.

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