Another significant current issue in this context is the increased medical cost of conventional treatment due to the higher consumption of concentrates. Biosimilar products may offer advantages in these circumstances and may offer a less expensive alternative. Regulatory issues, BMS-777607 cost however, together with acceptability of biosimilar materials and reimbursement policies as well as supply and demand incentives remain to be considered. Rare bleeding disorders (RBDs) have attracted less attention from the pharmaceutical industry than haemophilia or von Willebrand disease due to the limited number of patients involved. Many cases of this type have

been treated, therefore, using fresh frozen plasma (FFP) or prothrombin

complex concentrates (PCCs) which carry serious risks of infections, allergic reactions and fluid overload. Several specific plasma-derived or recombinant products including fibrinogen, FVIIa, FXI and FXIII have now become available, however, and a phase III clinical study of recombinant FXIIIa has recently been completed demonstrating safety and efficacy of substances of this nature. The introduction of highly purified and recombinant products has facilitated the use of regular prophylaxis PLX4720 as the principal type of haemostasis therapy especially in paediatric and young adult patients. The number of spontaneous and life-threatening bleeds has been remarkably reduced in these individuals compared to those treated on-demand. Furthermore, randomized prospective studies have revealed that primary prophylaxis may protect from the development and progress of haemo-arthropathy. However, several issues still remain unsolved in the treatment of haemophilia. For example, the need for frequent venous access for FVIII or FIX infusions can result in a significant physical and mental burden. Central venous catheters may be helpful, but these involve a risk of

infection and thrombosis. In addition, the development of inhibitors presents the major clinical challenge. Once an inhibitor develops, haemostatic control becomes difficult and complicated. Immune tolerance treatment (ITI) is effective in over half of the patients with inhibitor, but clinical management in the unsuccessful patients is extremely difficult. In such Aldol condensation cases, bypassing therapies with activated prothrombin complex concentrates (APCC) or recombinant factor VII (rFVIIa) are usually used. The haemostatic effects of these materials are limited, however, when compared to replacement therapy with FVIII or FIX concentrates in patients without inhibitor. Economic considerations may also be important due to the increased utilization of FVIII or FIX concentrates. This can cause substantial stress to haemophilia treaters, governments and insurance companies even in developed countries.