The Centers for Medicare & Medicaid Services (CMS) has announced a decision to cover Chimeric Antigen Receptor T-cell, or “CAR T-cell” therapy, which is a form of cancer treatment that uses a patient’s own genetically modified immune cells to fight disease. CAR T-cell therapies are approved by the Food and Drug Administration to treat some people with specific types of cancer – certain types of non-Hodgkin lymphoma and B-cell precursor acute lymphoblastic leukemia.
“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,” said CMS Administrator Seema Verma in her prepared statement. “CMS will work closely with our sister agencies to monitor outcomes for Medicare patients receiving this innovative therapy going forward.”
FDA says it is important to monitor responses to CAR T-cell therapies in the Medicare population, as outcomes data for these patients are relatively limited and the treatment represents a significant change from current practices
CMS will leverage information obtained from the FDA’s required post-approval safety studies for CAR T-cell therapies to the fullest extent possible. Due to the serious risks associated with their use, the FDA required the manufacturers of CAR T-cell therapies to conduct post-marketing observational studies involving patients treated with the therapies.
Medicare will cover CAR T-cell therapies when they are provided in healthcare facilities enrolled in the FDA risk evaluation and mitigation strategies (REMS) for FDA-approved indications (according to the FDA-approved label). In addition, Medicare will cover FDA-approved CAR T-cell therapies for off-label uses that are recommended by CMS-approved compendia. A compendium is used in determining medically accepted uses of drugs and biologicals. Information about CMS-approved compendia is available at https://www.cms.gov/Medicare/Coverage/CoverageGenInfo/compendia.html.
The National Institutes of Health’s National Cancer Institute began supporting the Cellular Immunotherapy Data Resource developed by the Center for International Blood and Marrow Transplantation Research three years ago to allow long-term follow-up and scientific study of patients receiving CAR T-cell therapies. High-quality data, including demographics, tumor characteristics, course of cancer treatment, cellular product manufacturing details, and adverse events and outcomes have been collected for 1,400 patients treated for cancer with CAR T-cell therapies to date.
