Pharmaco-economic aspects of inhibitor treatment

The management of bleeding episodes and surgery in haemophilia patients who develop inhibitors is especially difficult. Haemostasis is typically achieved using high doses of factor VIII (FVIII) or FIX to overcome the inhibitor, or by using activated products, or by inhibitor eradication by immune tolerance induction. We assessed the costs of coagulation factors for non-inhibitor haemophilia A and B patients (ACneg; n = 104), patients with low responding inhibitors (LR; n = 24) and patients with high responding inhibitors (HR; n = 17) in our centre between 1988 and 1995. In ACneg and LR patients, > 90% of costs were attributed to home treatment, compared with 54% for HR patients. The costs of treatment were $US 36.8 m total, $US 41,000 mean per patient for ACneg patients; $US 10 m total, $US 46,000 mean per patient for LR patients; and $US 7.6 m total, $US 59,000 mean per patient for HR patients. The mean cost of hospital treatment per HR patient was more than 10 times that for ACneg or LR patients and this was influenced by the occurrence of 4 very severe bleeding episodes in these patients. This analysis highlights the different costs and treatment patterns for inhibitor patients and should enable us to evaluate the impact of new treatments such as recombinant activated rFVII (rFVIIa; NovoSeven, Novo Nordisk, Bagsvaerd, Denmark) on treatment at our centre in the future.