New FDA-approved cancer gene therapy to cost $475,000 for a single course, reports say

Are pharmaceutical companies gouging the public?

Very possibly, as evidenced by a new gene therapy for childhood leukemia that will cost almost half a million dollars for a single course.

Reports yesterday about Federal Drug Administration approval for Novartis’ Kymriah, which is the first gene therapy for cancer that it’s okayed, say it will cost at least $475,000.

Although some oncologists have described the CAR-T drug as revolutionary, critics fear the price tag could pave the way for future drug costs to skyrocket.

According to a story by Catherine Ho in the San Francisco Chronicle, the way the treatment works is this: A patient’s cells are extracted from his or her blood, then sent to a site where they’ll be genetically modified. The altered cells are then re-infused into the patient’s body.

The FDA this week approved Kymriah as a treatment for childhood B-cell acute lymphoblastic leukemia, a cancer of the blood and bone marrow that’s the most common childhood cancer.

The National Cancer Institute has estimated 3,100 patients up to 20 years old are diagnosed with the disease annually. Although the survival rate is 90 percent, that figure drops to as low as 10 percent for those who relapse after a bone marrow transplant. It is that subset of patients who are eligible for cell therapy, whose rate of success is said to be between 70 and 80 percent.

Only a few hundred children and young adults are expected to be treated with Kymriah each year.

Bruno Strigini

A Guardian story, supplemented by Associated Press reporting, said that Bruno Strigini, Novartis’ head of oncology, has explained that the high cost of the treatment is an attempt to balance patient access to the drug with ensuring a return on the company’s investment.

The Guardian piece also cited a Novartis statement to the effect that the company “was collaborating with Medicare on a plan in which the government would only pay for the treatment if patients responded to them by the end of the first month.”

Side effects of the drug include high fevers, diarrhea, vomiting, delirium, loss of balance, and difficulty speaking and understanding.

More information about the exorbitant costs of cancer treatments can be found in  “Rollercoaster: How a man survive his partner’s breast cancer,” a VitalityPress book I, Woody Weingarten, aimed at male caregivers.