Abstract

Treatment for haemophilia inhibitor patients has improved substantially since the 1980's and 1990's. But from a health economic perspective, an unmet need persists for better treatment options in terms of health-related quality of life (HRQoL) and cost savings. Haemostatic products alone account for the biggest expense in the care of haemophilia patients. Congenital haemophilia with inhibitors involves lifelong treatment, and with it, substantial long-term costs and lowered HRQoL. A systematic review on cost and HRQoL findings for haemophilia treatments in economic terms was conducted on more than 280 scientific studies. Fifty-five articles were retained for the execution of the project analysis. The analysis shows that to counteract costs to payers (society) and to improve patient outcomes, improved treatments could increase HRQoL. This could be achieved with FVIII and FIX bypassing agents that have: greater convenience through subcutaneous administration (less-painful injections, avoidance of infusions), faster onset through a fast-acting rFVIIa analogue (faster resolution of bleeding episodes leading to faster pain relief, sustained control, less dosing), and longer duration through a long-acting rFVIIa derivative (fewer injections and rebleeds, arthropathy prevention). Improved FVIII/IX bypassing agents for treatment of haemophilia patients with inhibitors would be a cost-effective option for society, improving HRQoL associated with the clinical condition.

Full Text
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