The FDA just approved the world’s most expensive drug: ScienceAlert

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

Per dose, it will cost $3.5 million, making it the most expensive drug in the world.

At first glance, the price is staggering, but a recent analysis of the drug’s cost-effectiveness suggests it’s a relatively “fair” price for what the treatment gets at least in the US.

The medicine, called Hemgenix, is a gene therapy treatment for haemophilia B, which is a rare genetic disorder that causes impaired blood clotting. More serious symptoms include spontaneous and repeated bleeding episodes that are difficult to stop.

Hemophilia B tends to be more common in men than women, and while it’s hard to come up with an exact number, estimates suggest nearly 8,000 men in the United States currently suffer from the lifelong disease.

The main drug currently used to treat hemophilia B in the United States gives patients much-needed clotting factor, but lifetime treatment costs are 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 cost of adult living for each patient with moderate to severe hemophilia B to be approximately $21-23 million. Treatment costs in the UK are cheaper than in the US or elsewhere in Europe, but still run into the 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 delivered in the body via a viral-based vector, designed to deliver DNA to target cells in the liver. This genetic information is then replicated by cells, spreading instructions for a clotting protein known as Factor IX.

So far, two studies have tested the efficacy and safety of Hemgenix. In a study of 54 participants with severe or moderately severe haemophilia B, researchers found increased levels of factor IX activity, reducing the need for currently available routine replacement therapies for patients.

After receiving the gene therapy, the rate at which patients developed uncontrolled bleeding decreased by more than 50% from their baseline rate.

Side effects included headaches, flu-like symptoms and liver enzyme elevations, all of which should be monitored closely 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, prevention and treatment of bleeding episodes can negatively impact people’s quality of life,” he says Peter Marks, director of the FDA. Center for Biological Evaluation and Research.

“Today’s approval provides a new treatment option for patients with haemophilia B and represents an important advance in the development of innovative therapies for those suffering from the high disease burden associated with this form of haemophilia.”

It’s not yet clear whether this gene therapy treatment is a cure for haemophilia B, but early results are promising.

For severe but rare disorders, such as hemophilia B, the FDA has a special designation to encourage 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 maker, CSL Behring, holds exclusive rights to the US market for the next seven years.

The incentives employed by the US government to stimulate scientific research are a useful way to foster 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 impose restrictions on drug prices, reap the rewards of international research.

Today, the United States pays two to six times more for prescription drugs than other countries in the world.

The previous record holder for most expensive drug was another “one shot” form of gene therapy for the treatment of spinal muscular atrophy. At an estimated $2 million per course, it has also generated heated debate about how drug companies finance their businesses.

While many drugmakers have taken advantage of orphan drug status in recent decades to create medicine monopolies, this latest product could be one case where the policy could work for at least some people.

The upfront costs are certainly immense, but for those who might be lucky enough to have a backing insurance company, Hemgenix could save millions in medical bills, improving lives in immeasurable ways.

The European Medicines Agency and its drug regulatory counterparts in the UK and Australia are also now reviewing gene therapy treatment for use.

It will be interesting to see how much drug makers can charge for Hemgenix in other parts of the world.

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