Verma said the agency, which had scheduled this coverage decision originally for late May, has been struggling to figure out how to cover and pay for the treatment, called CAR T-cell therapy. The treatment costs $375,000 or $475,000, depending on whether it is used for advanced lymphoma or pediatric leukemia. Hospital stays can add hundreds of thousands of dollars to the cost of care.
Drugmakers note CAR T-cell therapy is designed to be given just once and to be a potential cure for patients who have run out of other options. But not all patients benefit from it and because it is so new, it’s too soon to know whether it will deliver long-term cures.
Such expensive treatments are “begging the question of how the system is going to pay for this over the long term,” Verma said, especially given Medicare’s considerable financial strains. “This is something we are extremely concerned about,” she added.
The coverage decision announced Wednesday is different in some important aspects from an agency proposal from February. That proposal called for “coverage with evidence development” — which would have required hospitals to collect and report data on patient outcomes over a long period. Hospitals said the data collection would be overly burdensome.
CMS’ final decision dropped that requirement. Instead, the agency said it will rely on patient information collected by the Food and Drug Administration and the National Cancer Institute. The FDA is requiring the two manufacturers of the therapy — Novartis and Gilead Sciences — to follow patients for years and report on outcomes. Such data will be put in a registry supported by NCI.
The Medicare decision also said it will cover the therapy when it is administered at health care facilities enrolled in an FDA-mandated safety program requiring special training on handling side effects. That means the treatment could be given on an outpatient basis, which would be much less costly.
The American Society of Hematology praised the decision, especially the elimination of the “coverage with evidence development” requirement. It said the proposed mandate would have prompted some hospitals not to provide CAR T-cell therapy.
The group also praised the decision for saying Medicare would cover all FDA-approved uses for CAR T-cell therapy and off-label uses recommended in CMS-approved compendiums, which are used to determine medically accepted uses of drugs and biologics.
The Medicare decision was the second step in a week taken by CMS to bolster CAR T-cell therapy. Last Friday, the agency said it would increase the reimbursement for CAR T-cell therapy, but didn’t go as far as many hospitals had wanted. Medical centers have complained they are losing money on Medicare patients because reimbursements have been too low.
The FDA approved two versions of CAR T-cell therapy, which stands for chimeric antigen receptor T-cell, in 2017. Kymriah is made by Novartis for certain types of lymphoma and childhood leukemia, and Yescarta is manufactured by Gilead Sciences for lymphoma. Other CAR T-cell therapy products are in development.
The complicated treatment involves extracting and genetically altering the patient’s T cells to attack a protein on the surface of cancer cells. The cells are then infused back into the patient.