Intractable diseases promise long-term relief with new genetic and high-cost cures. Insurers do not plan to access these million-dollar costs of gene therapies. Unlike the vast majority of medications, which only relieve symptoms, these therapies address the underlying cause of the disease. These treatments have alleviated severe cases in patients who would otherwise have eternal or painful treatments.
Pipe, a hematologist at the University of Michigan Health System, spoke about the work being done in major bleeding. For the doctor, “they no longer have to think about their hemophilia,” about the patients. The hematologist understands that “for all intents and purposes, this looks like a cure.” Hemgenix is the name of the therapy that obtained approval from the U.S. Food and Drug Administration. On November 22, it obtained approval to treat patients with severe hemophilia B. CSL Behring, following approval, began marketing the drug. Its value: 3.5 million dollars per dose, making it the most expensive drug in the world.
Genetic cures and their high cost
Pipe made it clear that those who took part in the research study did not have to pay for the therapy. However, both prospective patients and their insurers will have to do so. It is not yet clear whether this single dose will last for life. Pipe has reported that some patients, dosed more than 10 years ago, still keep their bleeding at bay. For gene therapy experts, the price is no surprise.
For Nicole Paulk, assistant professor of gene therapy at the University of California, San Francisco, it is “a very fair price. Several specialists in the field understand that these values, for genetic cures, are within the reasonable and expected range. For Len Valentino, hematologist and executive director of the National Hemophilia Foundation, the price is a milestone for patients. Particularly because this hemophilia B can lead to severe pain and mobility problems over time.
A severe disease and to attack
Hemophilia B is caused by a mutation of a gene called F9. This defect stops the production of an essential protein necessary for blood clotting called Factor IX. Valentino understands that the prevention of these hemorrhages, which the disease produces, can have a great effect on a patient’s life. This treatment would put an end to the regular infusions that patients with this disease receive today.
“A patient with severe hemophilia B might need injections of clotting factor concentrate two or three times a week for a lifetime. That’s a significant burden,” Valentino said. A cost study, carried out in 2016, puts the cost of infusions at more than a quarter of a million dollars a year. By 2021, this value had climbed to over US$300,000 per year.