An Italian Pharmacoutilization Analysis in Patients With Hemophilia A With and Without Inhibitors After Switching to Emicizumab

OBJECTIVES: The burden of lifelong prophylaxis treatments to prevent bleeding events and preserve joint health for patients with haemophilia A is substantial, affecting both patients and the healthcare system. The availability of emicizumab, the first non-factor replacement therapy subcutaneously administered for the treatment of haemophilia A, represented a disruptive innovation in bleeding prevention and patient management. This is a real-world pharmacoutilization analysis evaluating the prophylactic treatment costs in patients with severe haemophilia A, with and without FVIII inhibitors, before and after switching to emicizumab.

METHODS: Starting from an administrative database of Local Health Units (~9 million subjects), the analysis includes male patients with severe haemophilia A, with or without FVIII inhibitors, who began the treatment with emicizumab between January 2018 to September 2023. The prophylaxis regimen was identified using a literature-validated algorithm. FVIII and bypassing agents’ costs were assessed over 12 months before the first prescription of emicizumab. Emicizumab costs were actualized considering its price evolution.

RESULTS: Among 91 patients with at least one prescription of emicizumab and at least 12 months treatment with emicizumab, 24 patients (mean age 26.3±21.2 years) were on prophylaxis with FVIII and 10 patients (mean age 23.0±23.7 years) have FVIII inhibitors. The average prophylaxis annual costs for patients with and without inhibitors, before switching to emicizumab, were €390.751 and €200.699 (VAT included) per patient respectively while the average prophylaxis costs over one year of treatment with emicizumab were €187.943 and €214.271 (VAT included) per patient respectively, by applying a cost actualization adjustment for emicizumab.

CONCLUSIONS: This analysis shows that, considering the emicizumab actual cost, the annual prophylactic treatment decrease of -51,9% after switching to emicizumab for patients with inhibitors while for patients without inhibitors there is a slight difference between emicizumab and FVIII prophylaxis costs (6,8%).