CMS Releases New Rules Overseeing CAR-T Immuno-Oncology Therapy


Up to this point, Medicare’s regional administrators made the decision on whether the government’s healthcare payer for older populations would cover the innovation CAR-T therapies. However, yesterday, the Centers for Medicare and Medicaid Services (CMS) announced the agency would cover CAR-T so patients would have “consistent and predictable access” to the therapy.

Medicare will pay for CAR-T if they are administered in facilities that follow the U.S. Food and Drug Administration (FDA)’s special safety rules, called risk evaluation and mitigation strategies (REMS). It will also reimburse for the therapy when it’s used to treat illnesses that aren’t approved by the FDA.

There are two CAR-T therapies on the market, Gilead Sciences’ Yescarta and Novartis’ Kymriah. Yescarta is approved for non-Hodgkin lymphoma and Kymriah for acute lymphoblastic leukemia.

When the therapies were approved, the FDA required both companies to set up REMS programs, because of the potential for significant side effects, primarily cytokine release syndrome and neurotoxicities. Those REMS programs required hospitals and clinics that provide CAR-T to be specially certified and have on-site access to treatments to handle the side effects, if necessary.

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In a 2018 BioSpace interview with David Maloney, medical director of the Bezos Family Immunotherapy Clinic and director of cellular immunotherapy at Fred Hutchinson Cancer Research Center, Maloney said, “These types of therapies are totally different than any other cancer therapies. They are essentially living cells that actually replicate and grow in the patient as they attack the cancer. It’s exactly the opposite of chemotherapy, which goes in and causes damage, and the damage is slowly resolved over time. CAR-T cells are infused in a relatively small number of cells that actively grow as they engage the target tumor’s cells and kill them. It is a type of living therapy that can expand to eradicate all of the tumor.”

Part of the reluctance on payment is tied to the costs, which run around $375,000 to $475,000, depending on whether the therapies are for patients with advanced lymphoma or pediatric leukemia. And if the patients require hospitalization, which is variable, it can cost hundreds of thousands of dollars more.

The therapies, despite being revolutionary for certain types of cancers, haven’t gained as much traction as expected. This is typically related to confusion over CMS’s rules and reluctance on the part of payers to pay for it.

“Today’s coverage decision provides consistent and predictable patient access nationwide,” stated Seema Verma, CMS Administrator. “CMS will work closely with our sister agencies to monitor outcomes for Medicare patients receiving this in innovative therapy going forward.”

STAT notes that CMS’s decision to write the national coverage determination has been controversial. “The two drug makers already selling CAR-T treatments have argued the national coverage policy is entirely unnecessary, because the contractors that administer Medicare’s hospital claims were already paying for the treatment. Others, including doctors groups, criticized a number of policies in the draft proposal.”

The agency tried to address the concerns in its final rules. It walks back earlier restrictions on CAR-T only being administered in hospitals, which will allow standalone oncology clinics to provide the therapy. Another, perhaps more puzzling change, is CMS gave up on requiring hospitals collect data on how the patients respond to the treatment. Instead, CMS will depend on patient information collected by the FDA and the National Cancer Institute (NCI). The FDA already requires Novartis and Gilead to follow patients for several years and report outcomes. It will be stored in a database supported by NCI.

The Medicare changes don’t guarantee hospitals will break even on CAR-T, however. CMS has struggled to decide on how much to reimburse and Verma told STAT in an earlier interview that it could be years before it is resolved.

Last Friday, CMS indicated it would increase reimbursement for CAR-T, but hospitals indicate those reimbursements are too low and are losing money on Medicare patients.

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