CMS to Cover FDA-Approved CAR-T Cell Therapy

The Centers for Medicare & Medicaid Services (CMS) made a long-anticipated announcement Wednesday that chimeric antigen receptor (CAR)-T cell therapy, a cutting-edge immunotherapy for cancer, would be covered by Medicare.

CAR-T cell therapies aim to treat people with specific types of cancer — certain types of non-Hodgkin lymphoma and B-cell precursor acute lymphoblastic leukemia — by genetically modifying a patient’s own immune cells.

The coverage of the U.S. Food and Drug Administration (FDA)-approved CAR-T cell therapy is part of an effort to “strengthen” Medicare by providing access to the latest lifesaving treatments, said Seema Verma, MPH, the CMS Administrator, in a statement.

“As the first type of FDA-approved gene therapy, CAR-T cell therapies are an important scientific advancement in this promising new area of medicine and provide treatment options for some patients who had nowhere else to turn,” added Verma.

The agency had a lot to consider before determining whether it would cover the therapy, as the cost for CAR-T cell therapy is between $373,000 and $475,000. And hospitalization costs associated with CAR-T cell treatment can drive the total cost of care to more than $1 million per patient, according to our sister publication, Targeted Oncology.

To be covered by Medicare, hospitals and health systems need to be enrolled in the FDA risk evaluation and mitigation strategies according to the FDA-approved label. Medicare will also cover the therapies for off-label uses that are recommended by CMS-approved compendia.

The anti-cancer compendia recognizes the American Medical Association Drug Evaluations, U.S. Pharmacopeia-Drug Information and American Hospital Formulary Service-Drug Information as authoritative sources. These sources can be used “in the determination of a ‘medically-accepted indication’ of drugs and biologicals used off-label in an anticancer chemotherapeutic regimen.”

Monitoring responses to these therapies in the Medicare population is necessary because the outcomes data are relatively limited and the treatment represents a big change from current practices, CMS said.

To do this, the agency said it will use information from the FDA’s required post-approval safety studies for these therapies as much as possible. The FDA requires manufacturers of CAR-T cell therapies to conduct post-marketing observational studies involving patients treated with the therapies.

“Our robust post-market surveillance programs will continue to monitor for potential risks, as we do for all licensed and approved medical products,” said Ned Sharpless, M.D., acting commissioner of the FDA. “We will also continue to carefully assess the benefits and risks when considering whether to approve new CAR-T cell products.”

CMS received many comments during a public comment period earlier this year in which experts in the field supported the idea of Medicare coverage for CAR-T cell therapies.

“CAR-T cell therapy represents the best of what American innovation has to offer and it is our goal to ensure that these curative treatments are appropriately accessible to patients they are designed to help,” wrote Karen Bird, executive director of the Alliance of Dedicated Cancer Centers, which aims to advance cancer treatment, among other things.

Providers, however, must be equipped to ensure the safest and most efficacious delivery of such therapies, Bird noted.

CMS announced earlier this year that hospitals administering CAR-T cell therapy need a cellular therapy program, a care area to protect the patient from transmitting infectious agents and written guidelines when administering the therapy for communication with the patient, monitoring and transfer to an intensive care unit.

Providers then would have the opportunity to use a cancer treatment that can be directed specifically to the cancer cells in patients who have failed all other available treatments, officials said.

“(Yesterday’s) coverage decision provides consistent and predictable patient access nationwide,” Verma said.

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