The United States Federal Drug Administration (FDA) has just approved a new treatment for a rare blood clotting disease, one with a heavy price tag.
Per dose, it will cost $3.5 million, making it the most expensive drug anywhere in the world.
At first glance, the price is shocking, but a recent analysis on the drug’s cost-effectiveness shows that it is a relatively “fair” price for what the treatment achieves – at least in the US.
The drug, called Hemgenix, is a gene therapy treatment for hemophilia B, which is a rare genetic disease that causes impaired blood clotting. More severe symptoms include spontaneous and recurrent bleeding episodes that are difficult to stop.
Hemophilia B tends to be more common in men than women, and while an exact number is hard to come by, estimates suggest that nearly 8,000 men in the U.S. currently suffer from the lifelong disease.
The main drug currently used to treat hemophilia B in the US provides patients with a much-needed clotting factor, but the lifetime cost of treatment is high. In those with severe symptoms, a routine and expensive treatment regimen is required, which over time can begin to diminish in effectiveness.
Today, researchers estimate the adult lifetime cost of each patient with moderate to severe hemophilia B to be approximately 21 to 23 million US dollars. Treatment costs in the UK are cheaper than in the US or elsewhere in Europe, but still run into tens of millions of dollars per patient over their lifetime.
Hemgenix, on the other hand, is a single-dose intravenous product at a fraction of the price. The product is delivered into the body via a virus-based vector that is engineered to deliver DNA to target cells in the liver. This genetic information is then copied by cells, spreading the instructions for a clotting protein known as Factor IX.
Two studies have so far tested the effectiveness and safety of Hemgenix. In a study among 54 participants with severe or moderate hemophilia B, researchers found increased levels of Factor IX activity, reducing the need for routine replacement therapies currently available to patients.
After receiving the gene therapy, the rate at which patients developed uncontrolled bleeding dropped by more than 50 percent compared to their baseline rate.
Side effects included headaches, flu-like symptoms and liver enzyme elevations, which should be carefully monitored by doctors in the future.
“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advances in the treatment of hemophilia, the prevention and treatment of bleeding episodes can negatively impact people’s quality of life,” says FDA Director Peter Marks . Center for Biological Evaluation and Research.
“Today’s approval provides a new treatment option for patients with hemophilia B and represents significant progress in the development of innovative therapies for those facing a high disease burden associated with this form of hemophilia.”
It is not yet clear whether this gene therapy treatment is a cure for hemophilia B, but initial results are promising.
For serious, but rare, disorders such as hemophilia B, the FDA has special designation to incentivize medical research. Hemgenix, for example, is classified as an “orphan drug” because it will only treat a small number of patients.
As part of this designation, Hemgenix’s manufacturer, CSL Behring, has exclusive rights to the US market for the next seven years.
The incentives the US government uses to promote scientific research are a useful way to stimulate innovation in rare diseases that would otherwise fall by the wayside, but this policy presents a double-edged sword.
It also means that the US market bears the cost of supporting drug monopolies, while other nations, which place restrictions on drug prices, reap the benefits of international research.
Today, the US pays between two and six times more for prescription drugs than other countries around the world.
The previous record holder for the most expensive drug was another form of ‘one shot’ gene therapy to treat spinal muscular atrophy. At an estimated US$2 million per course, it also sparked heated debate about how pharmaceutical companies finance their businesses.
While many drugmakers have taken advantage of orphan drug status in recent decades to create drug monopolies, this latest product could be a case where the policy might work for at least some people.
The upfront cost is certainly huge, but for those who may be lucky enough to have a supportive insurance company, Hemgenix could save millions in medical costs, improving lives in countless ways.
The European Medicines Agency and its equivalent drug regulators in the UK and Australia are now also reviewing gene therapy therapy for use.
It will be interesting to see how much drug makers are allowed to charge for Hemgenix in other parts of the world.