The Centers for Medicare & Medicaid Services (CMS), under the leadership of President Trump and Secretary Azar, finalized the decision to cover FDA-approved 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. FDA-approved CAR T-cell therapies are approved to treat some people with specific types of cancer – certain types of non-Hodgkin lymphoma and B-cell precursor acute lymphoblastic leukemia.
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.
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. Therefore, 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.
“We remain committed to supporting the efficient development of safe and effective CAR T-cell therapies. We know there are relatively limited data about the use of these life-saving therapies in the Medicare population. Our robust postmarket surveillance programs will continue to monitor for potential risks, as we do for all licensed and approved medical products. We will also continue to carefully assess the benefits and risks when considering whether to approve new CAR T-cell products,” said Acting FDA Commissioner Ned Sharpless, MD “We will continue working with our partners at CMS and the National Institutes of Health’s National Cancer Institute (NCI) to help advance the development and availability of these therapies to patients in need.”
NCI 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.