Safety and supply of haemophilia products: worldwide perspectives

Abstract

The survival and well-being of people with haemophilia depends on the supply of safe therapeutic products. Safety and supply are entirely intertwined principles; in the absence of adequate amounts of coagulation products, safety measures may be compromised in order to enhance supply, leading to risks which may result in morbidity and mortality. As haemophilia therapy has emerged through the development of blood transfusion and plasma fractionation, the safety of the blood supply in general has had a strong effect on haemophilia care. Despite the gradual detachment of haemophilia care from blood transfusion through the use of recombinant products, the majority of the world's population with haemophilia in the developing world will be reliant on blood products for the foreseeable future. It is, therefore, important to continue efforts for a safe and sufficient blood supply worldwide. As such a blood supply develops, possibly in tandem with an independent plasma fractionation industry, the level of haemophilia care should improve with the gradual introduction of concentrates for the ultimate goal of covering all aspects of care. Constant vigilance for the threat of blood-borne pathogens should be linked to considerations of how these products are to be manufactured. This should be governed entirely by considerations of safety and pharmaceutical competence. Of equal importance is a governmental capacity to oversee the entry and maintenance of these products on the market. While it is not possible for all countries to have a regulatory authority of the same status as that of the developed countries, it is perfectly feasible to develop a set of basic principles which allow an assessment of basic product safety, quality and efficacy to be made.