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The United States Federal Drug Administration (FDA) has just approved a new treatment for a rare blood clotting disease, one with a hefty price tag.

Per dose, it'll cost US$3.5 million, making it the most expensive drug anywhere in the world.

At first glance, the price is gobsmacking, but a recent analysis on the cost-effectiveness of the drug suggests that's a relatively 'fair' price for what the treatment achieves – at least in the US.

The medicine, called Hemgenix, is a gene therapy treatment for hemophilia B, which is a rare genetic disease that causes reduced clotting of the blood. The most serious symptoms include spontaneous and repeated bleeding episodes that are difficult to stop.

Hemophilia B tends be more common in men than women, and while an exact number is hard to come by, estimates suggest nearly 8,000 men in the US currently suffer from the lifelong disease.

The main medication currently used to treat hemophilia B in the US bestows patients with a much-needed clotting factor, but its lifetime treatment costs are steep. In those with severe symptoms, a routine and expensive treatment regime is required, one that over time can begin to wane in effectiveness.

Today, researchers estimate the adult lifetime cost for every patient with moderate to severe hemophilia B is around US$21 to $23 million. Treatment costs in the UK are cheaper than in the US or elsewhere in Europe, but still add up to tens of millions of dollars per patient over their lifetime.

Hemgenix, on the other hand, is a one-time intravenous product given in a single dose at a fraction of the price. The product is carried into the body via a viral-based vector, which is engineered to deliver DNA to target cells in the liver. This genetic information is then replicated by cells, spreading the instructions for a clotting protein, known as Factor IX.

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Originally posted by QuirtEvans:
It's expensive, but the long-term view is simple.

This isn't the only disease with a limited population. It's expensive to find these sorts of treatments. If the pharma company is told, tough luck, that's too expensive, what's their incentive to develop the next orphan drug? Wouldn't the pharma company say, next time, I'll make sure only to invest in research that has the potential to help millions? I'll leave those with rare diseases to fend for themselves?