Weatherall J, Barnes N, Brown C, Preaud E.
Treatment for hemophilia inhibitor patients has improved substantially since the 1980s and 1990s. However, 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. Hemostatic products alone account for the biggest expense in the care of hemophilia patients. Congenital hemophilia with inhibitors involves lifelong treatment and, with it, substantial long-term costs and lowered HRQOL. To counteract these costs to payers (society) and to improve patient outcomes, improved treatments could increase HRQOL. This could be achieved with factor VIII and factor IX bypassing agents that have greater convenience through subcutaneous administration (less painful injections and avoidance of infusions) and a faster onset through a fast-acting recombinant activated factor VII analog (faster resolution of bleeding episodes leading to faster pain relief, sustained control and less dosing). In short, improved factor VIII/IX bypassing agents for treatment of hemophilia patients with inhibitors would be a cost-effective option for society, improving HRQOL associated with the clinical condition, which further decreases the already limited budget impact because costs per patient are reduced for the 3500 hemophilia inhibitor patients worldwide.