A cost evaluation of treatment alternatives for mild‐to‐moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil

Summary.  The first‐line treatment for mild‐to‐moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil is currently activated prothrombin complex concentrate (aPCC), with recombinant activated factor VII (rFVIIa) used as second‐line therapy or as a last resort. The aim of this study was to determine the cost and effectiveness of these treatments from the perspective of the Brazilian National Health Service. A decision analysis model was constructed to assess total direct medical costs (including drug costs, costs of outpatient or inpatient care, ambulance transportation and cost of concomitant medications) of first‐line treatment with aPCC or rFVIIa. Clinical outcome and resource utilization data were obtained both retrospectively and prospectively and validated by the consensus of an expert panel of Brazilian haematologists. A total of 103 bleeds in 25 patients were included in the analysis. rFVIIa resolved bleeds more quickly (4.4 h) than aPCC (62.6 h) and was more effective (100% vs. 56.7% respectively). Mean total direct medical costs (from initiation to cessation of bleed) were estimated to be US$13 500 (aPCC) and US$7590 (rFVIIa). Extensive sensitivity analyses confirmed the cost‐effectiveness of rFVIIa. Compared with aPCC, rFVIIa was more effective and less expensive when used as first‐line treatment for mild‐to‐moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil. rFVIIa should be considered a first‐line treatment for the management of these patients.