The Food and Drug Administration’s approval of Kymriah was met with excitement for its potential—as well as concerns about the $475,000 cost of this breakthrough gene therapy treatment for a life-threatening form of childhood cancer.

In July an FDA advisory panel recommended approving the one-time treatment for children with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). Many patients with that type of cancer don’t respond to chemotherapy, radiation, and other existing treatments. About 2,500 cases of ALL occur in the U.S. each year, and roughly 600 of them don’t respond to standard treatments.

In a partnership with the Centers for Medicare and Medicaid Services, Novartis, the Swiss drug company that developed Kymriah (tisagenlecleucel), says it will pursue an “innovative pricing approach,” offering Kymriah at no charge for children covered by Medicaid who do not respond by the end of the first month of receiving the treatment. Children from low-income families and children with special needs receive free care through Medicaid.

In other words, if Kymriah works, Medicaid foots the bill. If the treament doesn’t work, Novartis refunds Medicaid. “The individual patient doesn’t receive money back; Medicaid does,” says Stephen Schondelmeyer, Pharm.D., Ph.D., chairman of the department of pharmaceutical care and health systems at the University of Minnesota College of Pharmacy.

Cost for Patients Not Covered by Medicaid

Novartis spokeswoman Julie Masow says that costs for this gene therapy will vary depending on the patient’s health plan, adding that Novartis is working with insurance plans “to ensure they fully understand the value of this therapy and provide coverage for patients accordingly.”

For eligible uninsured or underinsured patients, according to Masow, Novartis will offer co-pay discounts, travel assistance, and even free treatment through its patient access programs.

More on Drug Pricing

Patient-access programs can help families who might have to choose between an expensive medicine or treatment and paying rent and utilities, says Rich Sagall, M.D., president of NeedyMeds, an organization that connects people with patient assistance and other discount programs. 

“Many people are helped by a patient-assistance program or a co-pay-assistance program for expensive treatments,” Sagall adds. “But, unfortunately, for some, there are no appropriate programs.”

(If you have questions about these programs, call the Novartis patient support line at 844-459-6742.)

How Kymriah Works

Kymriah is a customized treatment that uses a patient’s own T-cells, a type of white blood cell known as a lymphocyte. T-cells are extracted from a patient’s bloodstream, genetically modified in a lab to attack cancer cells, and then infused back into the patient. Novartis says the process takes up to 22 days from extraction to infusion. 

The FDA gave Kymriah “breakthrough therapy status,” meaning its approval was fast-tracked. The safety and effectiveness of Kymriah was tested in one small clinical trial of 63 children and young adults with relapsed or refractory B-cell precursor ALL. The study found that the overall remission rate within three months of treatment was 83 percent.

Still, to further evaluate long-term safety, the FDA is requiring Novartis to conduct a study involving patients who are treated with the Kymriah.

For good reason. Though potentially lifesaving, Kymriah can also cause severe side effects, including a life-threatening reaction called cytokine release syndrome—a common immediate complication following T-cell infusions that can result in a range of symptoms from fever and nausea to liver failure and cardiac arrest. The treatment can also cause fever, headaches, low blood pressure, and bacterial infections. Most of these problems appear within one to 22 days after the infusion. 

Novartis says it will first offer Kymriah at 20 treatment centers across the U.S. within the next 30 days; the company says staff members at these facilities have received “extensive training.” This gene therapy will expand to 32 to 35 centers by end of the year. Because limited facilities will offer the gene therapy, families that don’t live near a facility will have to travel for the treatment and live within 2 hours of the facility for the duration of the treatment and monitoring.

“The good news is that 80 to 90 percent of children with B-cell acute lymphoblastic leukemia are cured through chemotherapy or bone marrow transplant,” says Gwen Nichols, M.D., chief medical officer of the Leukemia & Lymphoma Society, a nonprofit organization that helped fund research on the therapy. “But for a small percentage of patients for which traditional therapies are not effective, we are very hopeful about the lifesaving potential of this therapy.” 

Editor’s Note: This article and related materials are made possible by a grant from the state Attorney General Consumer and Prescriber Education Grant Program, which is funded by the multistate settlement of consumer-fraud claims regarding the marketing of the prescription drug Neurontin (gabapentin).