OBJECTIVES The HAVEN 1 trial showed that emicizumab prophylaxis reduced bleeding rates when compared with bypassing agents (BPA) on-demand and prophylaxis, among patients with hemophilia A with inhibitors (HAwI). The objective of this study was to evaluate the budget impact of emicizumab versus BPAs in the Brazilian Public Healthcare System (SUS) perspective.

METHODS Prevalence data for hemophilia from the Ministry of Health was used to estimate the eligible population of patients with HAwI and mean patient weight was extrapolated from general Brazilian male population adjusted by age. Analysis was done in SUS perspective and for a time horizon of 5 years. Drug costs for prophylaxis and breakthrough bleeds treatment were extracted from 2019 official sources (1 BRL = 0.27 USD). Annualized bleeding rates were retrieved from HAVEN 1. In the current scenario, market share was defined as 35% and 65% for BPA prophylaxis and BPA on-demand, respectively. Validity of the results were evaluated on a sensitivity analyses in which the uptake of emicizumab varied: scenario 1 (market-share 35% from Y1-Y5); scenario 2 (35% Y1 to 61% Y5); scenario 3 (6% Y1 to 64% Y5).

RESULTS Mean annual treatment cost is 1.76 million BRL (475.3 thousand USD), 2.00 million BRL (540.1 thousand USD) and 1.16 million BRL (313.7 thousand USD) for emicizumab prophylaxis, BPA prophylaxis and BPA on-demand, respectively. In 5 years, emicizumab can save up to 228 million BRL (61.6 million USD) in scenario 1 and the budget impact is neutral in scenarios 2 and 3. Bleeding rates reduction with emicizumab is the main factor that offsets treatment costs and drives budget savings.

CONCLUSIONS Compared with intravenous BPAs, once-weekly subcutaneous emicizumab prophylaxis can generate savings or at least maintain budget neutrality whilst expanding routine prophylaxis rate among patients with hemophilia A with inhibitors in Brazilian Public Healthcare System perspective.