OBJECTIVES: Hemophilia A is an X-linked hereditary bleeding disorder, caused by a deficiency in factor VIII (FVIII) clotting. Treatment aims at controlling bleeding through FVIII replacement, bypass agents, or emicizumab. FVIII treatments can lead to the development of FVIII inhibitors, a serious complication of the disease. This study aims to analyze the cost of treating bleeding, considering drug treatment from the perspective of the Brazilian public health system.

METHODS: This observational study used annual bleeding rates (ABR), local guidelines regimens and prices. We considered an average adult patient weighing 61.9 kg. The ABR used, was: bypass agents 15.7 in prophylaxis and 23.3 on demand and for emicizumab 2.9. A mean consumption of 270mgc/kg of rFVIIa or 127.5 U/kg of CCPa was considered. The unit cost of CCPa was R$1.73/U and R$3.59/mcg of rFVIIa. The costs of immunotolerance treatment were not computed.

RESULTS: The annual cost of prophylaxis drug treatment of bleeding in an average patient with Bypass was R$214,361.40 with CCPa and R$941,994.82 with rFVIIa. In patients treated with emicizumab, the cost was R$ 39,595.42 when using CCPa and R$ 173,999.04 with the use of rFVIIa to control bleedings. The cost is more than 5 times lower in patients treated with emicizumab, which represents an 81.5% reduction in the average annual cost of drug treatment for bleeding. In the on-demand scenario, the cost is about 8 times lower when emicizumab is used.

CONCLUSIONS: Emicizumab represents an opportunity in terms of effectiveness with a reduction of more than 80% in the number of bleeds/year, with savings that can range from R$174,765.98 (prophylactic CCPa) to R$1,223,993.27 (rFVIIa on demand) for every average patient treated with this technology. These data suggest the opportunity to optimize the allocation of resources to technologies that offer greater sustainability for the public health care Brazilian system.