A groundbreaking gene therapy has been found to cure patients battling cancer by at least 79 percent. It has just been approved by the U.S. Food and Drug Administration (FDA). The cost of the treatment? A whopping $475,000.
Known as CAR-T therapy, the FDA said it was the “first gene therapy” that has been made available in the U.S. and could cure patients with lethal-type blood and bone marrow cancer that has been affecting children and young adults.
The FDA-approved CAR-T therapy administers a customized treatment that uses a patient’s own T cells, which plays an important role for a person’s immunity system. The T cells would be extracted from the patient and would be cryogenically frozen in order to be transported to Novartis’ manufacturing center in New Jersey.
Novartis would genetically alter the T cells to “have a new gene that codes for a protein – called a chimeric antigen receptor, or CAR,” which fights off and kills leukemia cells with a certain antigen on their surface. These genetically modified T cells would then be infused back into the patient, MIT Technology Review reported.
The new gene therapy has shown remarkable results in a clinical trial of 63 children and young adults battling severe lymphoblastic leukemia. Out of the 63 patients administered with Novartis’ CAR-T therapy, 83 percent have remained in remission within three months. At six months, 89 percent of the patients were still living and at 12 months, 79 percent survived.
It would take an average of 22 days to create the therapy, from the time the T cells were extracted from the patient to the day the cells are infused back into the patient, Novartis said.
The rights to the new cancer treatment were given to them and they would sell it under the label Kymriah for $475,000. Previous estimates for the treatment were predicted to be around $600,000 to $725,000. Novartis said if patients do not respond to the therapy within a month, they do not need to pay for the treatment.
Novartis’ price tag for the treatment was met with criticism from David Mitchell, Founder and President of Patients For Affordable Drugs. According to him, a price of $475,000 per treatment was “excessive.”
Mitchell believed Americans should be able to afford the groundbreaking treatment because the funding for the research was drawn from “American taxpayers” who “invested over $200 million in CAR-T’s discovery.”
“But let’s remember, American taxpayers invested over $200 million in CAR-T’s discovery. To date, Novartis has not acknowledged the significance of taxpayers’ investment, and the company declined to detail its own investment,” he said. “While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive. Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more.”
Mitchell said they would be closely watching the issue with the intention to know if Novartis would lower the price if more patients become eligible for Kymriah and if U.S. taxpayers would be charged more than citizens from other regions when the drug is approved in other countries.