Where people are born, how they are raised, where they go to school … all of these factors have an impact on a person’s health.
Hemophilia is an inherited recessive genetic disorder that causes bleeding that is difficult to control. The blood of people with hemophilia does not coagulate properly because the body does not produce one or more of the plasma proteins required to form clots, or else it does not produce enough of these proteins.
There are approximately 20,000 hemophiliacs in the US.
The American Society of Hematology reports that the cost of treatment is several hundred thousand dollars a year, and far more if patients develop inhibitors that prevent replacement therapy from working. Drugs to treat complications can add more than $800,000 or more to the cost of treatment, placing them among the most expensive drugs for commercially insured patients.
In addition to the risk of bleeding from wounds, internal bleeding is a major threat for people with hemophilia. If the bleeding occurs in joints, it can cause chronic joint damage and pain.
Approximately 15-20% of people with hemophilia develop antibodies (called inhibitors) that prevent the clotting factors from working. This is one of the major complications of hemophilia.
The earliest clotting factor products came on the market in the 1960s. They were created by refining plasma from donated blood. Several thousand people died in the 1980s after HIV-contaminated blood was used to make products for treating hemophilia.
In the 1990s, researchers developed recombinant DNA techniques to make human blood clotting proteins (recombinant blood factors) in animal cells. The Centers for Disease Control (CDC) says that until 1992, all factor replacement products were made from human plasma.
In 1992, the U.S. Food and Drug Administration (FDA) approved genetically engineered recombinant factor concentrate, which does not come from human plasma.
The clotting proteins are then made into a freeze-dried product, which is tested and treated to kill any potential viruses before it is packaged for use.
Human clotting factors and recombinant factors are both used today.
As a pharmacy benefits manager, PharmAvail is acutely aware of the dangers of hemophilia for a small but vulnerable population. We are also aware of the high cost of therapy and regularly collaborate with our clients to ensure members receive the best possible care.