Cost-effectiveness of rFVIIa and CCPa in the on-demand treatment of congenital haemophilia with inhibitors in the Brazilian public setting

Abstract

Objective: To provide economic evidences that support the use of recombinant activated factor VII (rFVIIa) in comparison to activated prothrombin complex concentrate (aPCC) for the treatment of episodes of mild to moderate bleeding due to haemophilia with inhibitors in Sistema Único de Saúde (SUS). Methods: Studies that explored the clinical efficacy of aPCC and rFVIIa treatments have shown differences between them in their capacity for controlling bleeding episodes. The cost-effectiveness analysis was developed based on two decision tree models proposed by You et al. (2009) and Jiménez-Yuste et al. (2013). The clinical outcome was the percentage of patients with controlled bleeding, and the economic outcome was direct medical costs. The time horizon varied according to hemorrhage severity. Drugs unit costs were obtained from the Diário Oficial da União (1 µg of rFVIIa R$ 2.09 and 1U of aPCC R$ 2.28). In You model, also considered the costs with tranexamic acid and treatment center, extracted from CMED (Câmara de Regulação do Mercado de Medicamentos) and SIGTAP (Sistema de Gerenciamento da Tabela de Procedimentos), respectively. Results: You model showed a higher percentage of patients that controlled bleeding with rFVIIa compared to aPCC (89.2% vs. 75.1%) and with lower treatment cost (R$ 18,921 vs. R$ 28,691). Considering Jiménez-Yuste, the percentage of patients that controlled bleeding was also greater with rFVIIa (79.0% vs. 61.0%) with lower treatment cost (R$ 63,446 vs. R$ 68,952). Conclusion: rFVIIa is a cost-saving alternative for the treatment of patients with congenital haemophilia with inhibitors compared to aPCC.