OBJECTIVES

: To estimate the impact on annual costs of the use of Emicizumab in comparison with activated prothrombin complex concentrate – aPCC in prophylactic treatment for pediatric patients with severe Hemophilia A with inhibitors - HAwI from the perspective of the Dominican Republic health system.

METHODS

: A BIA was performed where the current scenario is prophylaxis with aPCC and the future scenario is prophylaxis with Emicizumab. The population was 19 patients with HAwI reported in the Annual Global Survey of the WFH, the time horizon was 3 years. Assumptions: aPCC dose was 50 IU/Kg three times a week, Emicizumab 3 mg/kg loading dose once a week by 4 weeks, then 1.5 mg once a week in maintenance doses. 100% of the cases will switch of therapy in the first year. An average of 3 bleeding events per year per patient was estimated in the current scenario an the results of the Haven II trial for the future scenario as a reference. In both scenarios, a typical case of a 20 kg patient was used as a basis and it was projected to the entire cohort. No discount rate is applied; the costs were obtained of public bidding and are presented in US dollars using the official rate of the Dominican Central Bank. Deterministic analysis is performed regarding the price of each product.

RESULTS

: The annual cost of a pediatric patient in the current scenario with aPCC was USD $ 286,603, in the future scenario it was USD $ 114,650. The cost of three years of care in the current scenario was USD $16,336,424 and in the future scenario USD $ 5,946,191.

CONCLUSIONS

: The BIA shows that Emicizumab could reduce the costs of prophylaxis in pediatric patients with HAwI in the Dominican Republic by USD $ 10,390,233 in a time horizon of three years.