In patients with
hemophilia A, outcomes have improved dramatically over the last few decades due to several advances in care, including the availability of
factor VIII (FVIII) concentrates. Current research has focused on enhancing the properties of FVIII concentrates and other
coagulation factor products using
recombinant DNA technology. However, there are several challenges to the development of new products for
hemophilia patients, including the relative rarity of the disease, rapidly evolving standards of care, and the varying requirements of regulatory authorities around the world. In the development of two innovative
coagulation factor products (recombinant single-chain
factor VIII [rVIII-SingleChain] and a
recombinant fusion protein linking
coagulation factor VIIa with
albumin [rVIIa-FP]), these issues have been addressed through novel clinical trial designs, including an ongoing three-part study evaluating the safety, efficacy, and pharmacokinetics of rVIII-SingleChain in patients with severe
hemophilia A, and a randomized, placebo-controlled, dose-escalation study of rVIIa-FP in healthy volunteers. The design of these trials is intended to answer as many important clinical questions as possible while limiting the burden on
hemophilia patients.