A new treatment for a rare blood clot disease has just been approved by theFDA.
It's the most expensive drug in the world at US$ 3.5 million per dose.
The price is gobsmacking, but a recent analysis on the cost-effectiveness of the drug suggests that is a relatively 'fair' price for what the treatment accomplishes.
Hemgenix is a medicine that is used to treat a rare genetic disease that causes reduced clotting of the blood. Bleeding episodes that are difficult to stop are the most serious symptoms.
Estimates suggest that over 8,000 men in the US suffer from hemophilia B, which is a lifelong disease.
The main medication used to treat hemophilia B in the US gives patients with a much needed clotting factor, but its lifetime treatment costs are steep. A routine and expensive treatment regime is required for people with severe symptoms.
The lifetime cost for every patient with moderate to severe hemophilia B is estimated by researchers. Over the course of a patient's lifetime, treatment costs in the UK can add up to tens of millions of dollars.
Hemgenix is a one-time product that can be given in a single dose. The product is carried into the body via a viral-basedVector which is engineered to deliver DNA to target cells in the liver. This genetic information is copied by cells and used to make Factor IX.
The efficacy and safety of Hemgenix have been tested. Researchers found that increased Factor IX activity levels reduced the need for replacement therapies in one study.
The rate at which patients developed bleeds decreased after receiving the gene therapy.
All of the side effects should be monitored by physicians going forward.
More than two decades have passed since gene therapy for the disease was first proposed. Peter Marks is the director of the FDA's Center for Biologics Evaluation and Research.
Today's approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies for those with this form of hemophilia.
The results of the treatment are promising, but it's not clear if it's a cure for the disease.
The FDA has a special designation for rare and serious diseases. Hemgenix is classified as an orphan drug because it will only treat a small group of patients.
The exclusive rights to the US market for the next seven years have been granted to the manufacturer of Hemgenix.
The incentives employed by the US government to spur on scientific research is a useful way to boost innovation into rare diseases, but it also presents a double-edged sword.
The US market shoulders the cost of drug monopolies while other nations get the benefits of international research.
The US pays more for prescription drugs than any other country.
Another form of gene therapy was the previous record holder for most expensive drug. It generated a lot of debate over the way drug companies fund their businesses.
While many drug manufacturers have taken advantage of the orphan drug status over the past few decades to create medicine monopolies, this latest product could be one example where the policy just might work for some people.
Hemgenix could save millions of dollars in medical costs for those who have a supportive insurance company.
The European Medicines Agency and its drug regulators in the UK and Australia are looking at the treatment.
It will be interesting to see how much Hemgenix costs in other countries.
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