OBJECTIVES : In Italy, emicizumab prophylaxis is approved for Haemophilia A with inhibitors and recently this agent has been licensed with a new price for severe Haemophilia A without inhibitors.. Aim of the study is quantifying the budget impact of emicizumab new indication and price on the Hemophilia A management by the Italian National Healthcare Service (NHS) perspective.

METHODS : A budget impact model was developed to compare healthcare costs of two scenarios: Scenario A - emicizumab available only for patients with inhibitors; Scenario B - emicizumab available also for patients without inhibitors and with a new price. Epidemiological and treatment data were retrieved from the literatures and market research. The analysis included only drug cost (>90% of haemophilia cost). Hemophilia A costs were calculated over 3 years (2020-2022) for both scenarios. The budget impact was reported as total difference costs of the two scenarios.

RESULTS : In Scenario A, model estimated a 3-years overall cost of 269.6€ mln in inhibitors patients and 894.5€ mln in non-inhibitors. In Scenario B, model estimated a 3-years overall cost of 229.8€ mln in inhibitors patients and 934.0€ mln in non-inhibitors. In inhibitors patients, the new emicizumab price led to a 3-year overall cost reduction of 39.8€ mln. In non-inhibitor patients, the availability of emicizumab led to an increase of 39.6€ mln, assuming 28% of patients treated with emicizumab instead FVIII after 3 years. The overall budget impact resulted substantially zero (-0.2€ mln).

CONCLUSIONS : Emicizumab prophylaxis for severe Haemophilia A patients could be economically sustainable thanks to the balance between costs saving in inhibitors patients and cost increase in non-inhibitors patients. Clinicians can introduce emicizumab prophylaxis in Italy without increasing the economic pressure on NHS budget. Real word data are needed to confirm these results, considering the variability of dose and regimen associated to rFVIII prophylaxis and the characteristics of patients.