OBJECTIVES: Hemophilia with inhibitor is a rare condition with a significant economic and social burden. The approval of the new therapies could allow physicians to select further options to improve patient’s management and outcome. The purpose of this study was to quantify the potential economic impact of a recently approved therapy, emicizumab, on the overall management of Hemophilia A with inhibitor, in Italy.

METHODS: A budget impact model was built. Adopting the perspective of the Italian National Healthcare Service (NHS), the model compared the healthcare costs of different treatment scenarios: the base-case, where emicizumab is not available for hemophilic patients, vs. the alternative-case, with emicizumab. Drug acquisition costs sustained by the Italian NHS to manage the patients with Hemophilia A and inhibitor have been calculated over 3-years and compared for the two scenario analyses. Emicizumab price was assumed equal to 210.2€/mg in order to have the same annual costs of aPCC prophylaxis. Sensitivity analyses were performed to identify the price scenario where emicizumab reported a neutral budget impact.

RESULTS: Based on actual treatment utilization data in Italy, the model estimated a 3-years overall cost of 368,839€ mln in all hemophilic patients with inhibitor (mean cost per patient/year =723,213€). Assuming a possible switch also from less expensive regimens (on-demand and high dose rFVIII prophylaxis) to emicizumab prophylaxis, a budget impact of +38.5€mln (+10%) was reported over three-years. Changing emicizumb price, a neutral budget impact was obtained at 157.6€/mg (-25% of basecase price).

CONCLUSIONS: The budget estimation is sensitive to emicizumab price. A neutral budget impact can be obtained if emicizumab price was set to 157.6€/mg. Below the neutral price, emicizumab prophylaxis use could save millions of euro. For example, an expected budgetary saving of 39.0€ mln was estimated assuming a price of 105.10€/mg.