A new and most expensive treatment for a rare disease that causes blood to clot has just been approved by the Food and Drug Administration (FDA) in the United States. The treatment costs 3.5 million dollars for every dose, making it the most costly medication anyplace on the planet.
Initially, the cost is shocking, but a recent cost-effectiveness analysis suggests that, at least in the United States, it is a reasonable cost for what the treatment accomplishes. A gene therapy medication known as “Hemagenix” is used to treat hemophilia B, a rare genetic condition that causes fewer blood clots. The disease’s most severe symptoms include difficult-to-stop bleeding episodes that occur on their own.
Hemophilia B affects more men than women, and approximately 8,000 men in the United States are thought to have the lifelong condition. In the United States, the main medication used to treat the disease gives patients a clotting factor, but the cost of treatment for life is high there.
Patients with moderate to severe hemophilia B, according to the researchers, will incur approximately 21 million to 23 million dollars in lifetime costs. The cost of treatment is significantly lower in the United Kingdom than it is in the United States or elsewhere in Europe.