Medicare will provide nationwide coverage for CAR T-cell therapies, an innovative anti-cancer treatment, for certain types of lymphoma and leukemia, the Centers for Medicare & Medicaid Services (CMS) has announced.
Medicare will pay for this costly therapy to all cancer patients falling under the labels approved by the U.S. Food and Drug Administration (FDA) as well as off-label uses under certain conditions specified by CMS.
CMS will also be working closely with other agencies to monitor the treatment’s safety and outcomes in Medicare beneficiaries, according to its news release.
Chimeric antigen receptor T-cell therapy, or CAR T-cell therapy, is a type of anti-cancer treatment that involves collecting a patient’s own immune T-cells and genetically modifying them in the lab to produce a chimeric antigen receptor, or CAR, that targets a specific cancer marker. The modified cells are then expanded in the lab and infused back into the patient, where they help fight the cancer.
So far, two such therapies have been FDA-approved to treat some patients with certain types of lymphoma and leukemia.
Kymriah (tisagenlecleucel), sold by Novartis, is indicated for the treatment of children and adults up to 25 years old who have B‑cell precursor acute lymphoblastic leukemia or adults with certain types of B-cell non-Hodgkin’s lymphoma that failed prior treatments.
The other CAR T-cell therapy approved is Yescarta (axicabtagene ciloleucel), which is sold by Kite Pharma/Gilead, and is indicated for use in adults with diffuse large B-cell lymphoma that failed conventional treatment.
Both therapies target CD19, a protein on the surface of lymphoma and leukemia cancer cells.
Medicare will cover CAR T-cell therapies that are provided in healthcare facilities enrolled in the FDA risk evaluation and mitigation strategies for approved indications.
It can also cover the therapies for off-label uses in circumstances recommended by the CMS compendia, which determines medically-accepted uses of medications and biologicals.
“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,” CMS administrator Seema Verma said in the release.
“Today’s 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,” she said.
Due to the serious risks associated with CAR T-cell therapies, the FDA required companies producing such treatments to conduct post-marketing observational studies to monitor safety events. But data is relatively limited, and the treatment represents a significant change from current practices.
Therefore, monitoring the responses among Medicare population will help build the current collection of data on CAR T-cell therapies.
“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 Ned Sharpless, MD, acting FDA commissioner. “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.”
The first successful cancer treatment with CAR T-cells was developed at NCI, which joined efforts with the Center for International Blood and Marrow Transplantation Research three years ago to perform long-term follow-up studies in patients receiving CAR T-cell therapies. To date, high-quality data, tumor characteristics, course of cancer treatment, adverse events, and outcomes have been collected for 1,400 patients.
“NCI looks forward to continuing to support the expansion of this registry to include more cancer patients in the Medicare program and others being treated with CAR T-cell therapies,” said Douglas R. Lowy, MD, acting director of NCI.
“We’ve recently seen dramatic progress in the development of CAR T-cell therapies and other treatments that harness patients’ own immune cells to treat their cancers, and we continue to learn more in clinical studies. By partnering with CMS and FDA, we have an opportunity to increase our understanding … ultimately allowing us to better understand who will benefit from these therapies.”
Read CMS’s full decision memo here.