Selecting therapeutic products for the treatment of haemophilia follows the process of obtaining market approval of products submitted to the scrutiny of a regulatory agency. In well-resourced countries, key decisions on whether a product is sufficiently safe and of high quality are made by highly expert and well-resourced agencies, such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). In countries lacking such agencies, well-informed decisions can still be made through an appreciation of the key issues affecting the quality, safety and efficacy of haemophilia products. A number of well-established principles may then be applied in order to make a choice. In this review, reflecting principles outlined by the World Federation of Hemophilia, we outline the key features in determining the acceptability of therapeutic products for haemophilia in order to ensure an optimal choice in all the environments providing haemophilia care.
Farrugia, A., Liumbruno, G., Candura, F., Profili, S., & Cassar, J. (2018). Factors affecting the quality, safety and marketing approval of clotting factor concentrates for haemophilia. Blood Transfusion, 16, 525-534. https://doi.org/10.2450/2018.0150-18