FDA approves $475,000 CAR-T therapy

The Food and Drug Administration (FDA) approved the first gene therapy released in the U.S., Kymriah, also known as chimeric antigen receptor T-cell (CAR-T) therapy. The $475,000 treatment will be outcomes-based; patients will not be charged by the pharmaceutical company Novartis if the therapy doesn’t work within a month.

The treatment cost includes the initial leukapheresis process to extract the T-cells along with the one-time engineered T-cell therapy. CMS is currently considering payment for the therapy. 

“As part of larger efforts to support the President’s priority, CMS is working actively with all stakeholders, including state officials, on innovative payment arrangements. These arrangements may, for example, include outcome-based pricing for medicines in relation to clinical outcomes,” stated CMS in a press release.

For a patient to qualify for Kymriah, he or she must be no older than 25 with B-cell precursor acute lymphoblastic leukemia that is in second relapse. It’s estimated that 600 patients annually will be eligible for CAR-T treatment, according to The Washington Post. For the first month after treatment, patients are required to stay within two hours of their treatment facility.

To administer the treatment, centers must be certified and have a warning about the risk of neurotoxicity and cytokine release syndrome. However, the results of Kymriah are promising. “In clinical trials in patients treated with CAR-T cells, 69% of patients had complete resolution of CRS within two weeks following one or two doses of Actemra,” stated the FDA. 

The impact the new high-cost drug will have on reimbursement isn't yet clear, but revenue integrity professionals should continue to monitor the situation at their facilities.