Haemophilia is an X-linked genetic disease associated with the dysfunction of the biological cascade leading to blood clotting. Coagulation factors are vital to the functioning of the mechanism that the human body uses to protect and repair itself from physical injury, by the formation of blood clots to seal ruptured blood vessels. Haemophilia patients have a reduced capacity to stop bleeding and are prone to bleeding internally, into joints and organs, and often require hospitalisation 3-4 times per year. Without proper treatment, haemophiliacs typically die from organ failure in their teens.
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Haemophilia A is treated with replacement therapy of a human blood protein, Factor VIII.
Gram for gram this is probably the most expensive compounds in the world and, consequently an expensive treatment. It sells for up to $4m per gram. Many patients, therefore, can simply not afford the treatment.
But there is also a real shortage in the supply of Factor VIII.
World Federation for Hemophilia (WFH) annual reports suggest that there is an annual supply of ~8BN International Units (IU) of both recombinant (manufactured) and plasma derived (collected and processed) Factor VIII. In the developed countries of the world, patients, on average, get around 100,000 IUs per year. That’s enough to treat 80,000 patients.
The WFH report that of the estimated 400,000 suffers of haemophilia A, only 163,000 get treatment (2017). And of these, we estimate only 76,000 get ”adequate” treatment. So there are 240.000 who get no treatment at all.
Haemophilia B is treated with Factor IX. The statistics indicate the same problem. It’s expensive and there just isn’t enough to go around.