Miracle of haemophilia drugs: Personal views about a few main players.

Abstract

In the second decade of the third millennium there have been dramatic developments pertaining to the availability of highly innovative drugs for hemophilia care, notwithstanding a satisfactory previous scenario. I am going to emphasize the role of 2 main categories of players: scientist physicians who produced important translational research and the pharmaceutical industry, who developed, produced and made commercially available so many improved treatment weapons stemming from the translational research of the forementioned scientist physicians. Pertaining to the role of scientist physicians, I chose to mention first those who were successful in the 1980 in the production of recombinant coagulation factors. In addition, those who more recently helped to produce new non substitutive therapies given by the subcutaneous route, and recombination coagulation factors with an extended half-life. Current miraculous progress in hemophilia therapy is stemming from the research work of outstanding scientist physicians who acted in close collaboration with small biotechnology companies, leading to the early development of innovative therapeutic products, subsequently taken to the market place by the so called Big Pharma. I shall briefly provide my views to explain the fact that large pharmaceutical companies show more and more interest in such a rare disease as the hemophilias.