Setting research priorities to improve cost-effectiveness estimations of primary prophylaxis with clotting factor for people with severe haemophilia

Summary.  Primary prophylaxis with clotting factor is the clinical treatment of choice for people with severe haemophilia, as evidence suggests it can prevent the onset and progression of joint- and muscle-related problems caused by bleeding episodes. However, the major limitation of this approach is that it requires considerably more clotting factor compared with treating on-demand. Thus, there is a need to establish its cost-effectiveness. The aim of this paper is to review the published evidence on the cost-effectiveness of prophylaxis and to highlight areas for future research that would decrease the uncertainty around these findings.