Thus, it is important to think not only about how to improve the current state-of-the-art products, but also about

ways in which existing therapies, important to an even larger percentage of the world’s population, can advance as well. The WFH estimates that more than MAPK Inhibitor Library one in 1000 men and women has a bleeding disorder equating to conservatively 6,900,000 worldwide (Table 1). This global estimate includes haemophilia [9] and women with hemophilia (symptomatic carriers) [10–13], von Willebrand disease (VWD) [14], rarer factor deficiencies [15], Glanzmann thrombasthenia and Bernard Soulier Syndrome [7], and is based on established prevalence rates, where known, published or developed by the WFH. The prevalence of VWD is based on those presenting with bleeding symptoms

to primary care physicians and is now thought to be approximately 1 per 1000 [14]. Some incidence estimates for the rarer factor deficiencies may only reflect the severe phenotypes. Additionally, geographical distribution for some of these deficiencies may vary due to consanguineous marriages. Where global prevalence data are not available the actual number of known individuals has been utilized [7]. Although available data, as reflected in the Table 1 indicate a more precise number of one (1) in 880 men and women has a bleeding Selleckchem PD0325901 disorder, given the imprecise nature of many of the estimates, the WFH has adopted a more conservative global prevalence estimate of one (1) in 1000 men and women has a bleeding disorder. More research into the incidence and prevalence of VWD and other inherited platelet disorders is needed. We expect this global estimate to be refined over time. We seek to establish this new global estimate to better reflect the totality of all bleeding disorders, as well as Sucrase to facilitate monitoring progress on patients identified over time as the world population grows and care expands globally. To date, 257,182 individuals with bleeding disorders have been identified worldwide including: 162,781 haemophilia, 65,100

VWD, and 29,301 other bleeding disorders (rarer factor deficiencies and inherited platelet disorders) [7]. Looking just at people with haemophilia, we estimate only about 25% worldwide receive at least minimally adequate treatment. The percentage is far lower for those with VWD and the other bleeding disorders. Adequate treatment means minimum access to episodic therapy with CFCs. The WFH has established that one international unit (IU) of factor (F) VIII CFC per capita should be the target minimum for countries to achieve optimal survival for the haemophilia population [17]. The consensus recommendations of an expert panel assembled by the European Directorate for the Quality of Medicines and HealthCare (EDQM) has concluded that the minimum acceptable national level of CFC use should be 2 IU per capita [18].