Abstract

This ancillary analysis of HEMONIS study aims to estimate the annual cost of clotting factors in Sev HA patients in France. HEMONIS is a national multicenter retrospective cohort study describing the treatment patterns of French patients with HA. Sev HA patients were included according to their inhibitor status: current inhibitor (CI) and no history of inhibitor (NHI). Clotting factors consumptions to prevent or manage bleeds were analysed according to treatment regimen (prophylaxis, on-demand (OD) and ITI)). Unit costs were based on the French tariffs excluding VAT (Feiba®: €0.903/U; Novoseven®: €0.608/μg; FVIII: €0.648/UI). Total cost was estimated at individual level and annualized to improve comparability of results. A representative sample of 66 Sev HA patients was analyzed: 36 NHI patients (median age 32; median weight 75.3 kg) and 30 CI patients (median age 22; median weight 66.8 kg). At last visit, the most frequent treatment was FVIII prophylaxis in NHI pts (81%), and ITI in CI pts (40%). In NHI patients treated with FVIII, the annual mean (median) cost per patient was €216,118 (€204,928) for patients under prophylaxis (n=25) and €78,685 (€55,690) for patients under OD treatment (n=9). In CI patients under ITI (FVIII only), mean annual cost per patient was €1,215,799 (€641,520) for background infusions (n=13) and €76,592 (€4,209) for bleeds management (n=11). When treated with prophylaxis (n=6) or OD (n=9) (BPA only), mean annual cost per patient was €811,268 (€795,294) and €184,196 (€140,659) respectively. This study shows the high economic burden of Sev HA patients especially among patients with inhibitor, for which the cost of prophylaxis is estimated above 800,000€ per year. However, this strategy is still associated with limited benefit on bleed reduction and high treatment burden for patients. This study highlights the potential need for more cost-effective treatments in Sev HA.

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