OBJECTIVES: To estimate the impact on annual costs of the use of Emicizumab compared to activated prothrombin complex - aPCC in prophylactic treatment for pediatric patients with severe Hemophilia A and inhibitors in the health system of the Dominican Republic.

METHODS: A BIA is performed where the current scenario is prophylaxis with aPCC and the future scenario is prophylaxis with emicizumab. The population is 19 patients with Hemophilia A and inhibitors reported by the Dominican Republic in the WFH global annual survey, the time horizon was 3 years. Assumptions: aPCC dose 50 IU/kg 3 times per week, emicizumab 3mg/kg loading dose once per week for 4 weeks, then 1.5 mg once per week in maintenance dose. 100% of the cases will make the switch in the first year. An average of 3 annual bleeding events per patient is estimated in the current scenario and the results of the Haven II trial for the future scenario as a reference. In both scenarios, a standard case of a 20-kg patient is used as the basis and the entire cohort is projected. No discount rate is applied, a deterministic analysis is performed regarding the price of each product.

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

CONCLUSIONS: The BIA shows that emicizumab could reduce prophylaxis costs in pediatric patients with Hemophilia A in the Dominican Republic by USD $10,390,233 over a 3-year time horizon, reducing annual health care costs by 67.5%. It is recommended in future analyzes to include indirect costs associated with less frequent application and a decrease in the annual rate of bleeding.