ON AUGUST 7, the Centers for Medicare & Medicaid Services (CMS) finalized the decision to cover U.S. Food and Drug Administration (FDA)-approved chimeric antigen receptor (CAR) T-cell therapy among recipients of Medicare benefits. FDA-approved CAR T-cell therapies are approved to treat patients with 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. “[This] coverage decision provides consistent and predictable patient access nationwide. CMS will work closely with our sister agencies to monitor outcomes for Medicare patients receiving this innovative therapy going forward.”
Medicare will cover CAR T-cell therapies when they are provided in health-care facilities enrolled in the FDA Risk Evaluation and Mitigation Strategies 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.
CMS also mentioned the importance of monitoring 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 postapproval 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 postmarketing observational studies involving patients treated with the therapies.
Ned Sharpless, MD
“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 3 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.
Read the full CMS decision memo on CAR T-cell therapy at www.cms.gov/medicare-coverage-database/details/nca-decision-memo.aspx?NCAId=291. ■