OBJECTIVES: A characteristic feature of the haemophilia A with inhibitors is the efficacy of therapeutic doses of blood factor VIII for the treatment and prevention of bleeding. Emicizumab or activated prothrombin complex concentrate is used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in such patients. This study assessed the cost of emicizumab versus activated prothrombin complex concentrate for children with haemophilia A with inhibitors in Ukraine.

METHODS: The cost analysis was carried out for emicizumab and activated prothrombin complex concentrate over a 1-year horizon per one pediatric patient weighing 30 kg. The analysis included drug manufacturers' prices registry from the Ministry of Health of Ukraine dated 21.10.2019. The direct medical costs were included in the analysis omitting cost of administration.

RESULTS: The direct medical costs per one patient for prophylaxis with emicizumab 1.5 mg/kg once every week was $264,624.74 and for emicizumab 3 mg/kg once every two weeks or 6 mg/kg once every four weeks $205,312.30 for one year. The costs of annual course of prophylaxis with activated prothrombin complex concentrate was $642,390.91 per one patient.

CONCLUSIONS: The introduction of emicizumab can potentially lead to cost savings of $377,766.18 (59%) for emicizumab 1.5 mg/kg once every week and $437,078.62 (68%) for emicizumab 3 mg/kg once every two weeks or 6 mg/kg once every four weeks per one patient for 1-year treatment compared to activated prothrombin complex concentrate. Therefore, the cost analysis shows that emicizumab has lower budget impact per one patient treatment than activated prothrombin complex concentrate. It is worth noting that emicizumab in doses of 3 mg/kg once every two weeks or 6 mg/kg once every four weeks has 25% ($59,312.44) economic advantage over dosage regimen of 1.5 mg/kg once every week for the pediatric population.