In a long-awaited national coverage determination (NCD) decision, CMS said Wednesday that it approved chimeric antigen receptor (CAR) T-cell therapies for Medicare beneficiaries nationwide, and in addition, it approved the NCD without some of the requirements that had some providers concerned.
“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,” said CMS Administrator Seema Verma in a statement.
UnitedHealthcare had requested the national coverage analysis decision last year.
Medicare will cover CAR T-cell therapies when they are provided in healthcare facilities enrolled in the FDA Risk Evaluation and Mitigation Strategies for FDA-approved indications (according to the FDA-approved label); in addition, CMS said Medicare will cover 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 biologics. CMS said
"coverage will be provided consistently on a national level and contractors local policies cannot supersede the NCD. In this final decision, off-label coverage will not be limited to uses recommended by NCCN [National Comprehensive Cancer Network]".
However, CMS decided not to proceed with a model called Coverage With Evidence With Development (CED), which critics feared could overburden providers, causing some to opt out of offering CAR T-cell treatment to Medicare patients.
In its decision memo
, CMS said gave several reasons for removing the CED requirement. It noted that the FDA has required post-marketing studies of the therapy, which is used only in very specific cancer cases where there are no other options, and that this is an area of ongoing research. Routine costs of clinical trials of newer CAR T-cell therapies being studied is covered under their existing policy on trials. "CAR T-cell therapy has been shown to induce remission in carefully identified relapsed or refractory cancer patients in appropriate settings of care," said CMS. "Informed decision making between a physician and patient remains key to determining the best treatment."
That cheered 1 oncologogist, who feared that CMS reporting requirements
would deter cancer centers from offering the treatment and hinder patient access.
"I applaud this determination and believe that this represents an important advance for patients in need of these life-saving treatments," said Joseph Alvarnas, MD, of the City of Hope, in Duarte, California, and editor-in-chief for Evidence-Based Oncology
™. "As we move forward with these new, life-saving treatments we look forward to continued collaboration with the agency to find ways to more effectively bring these treatments to Medicare beneficiaries in need."
CMS said it will use information obtained from the FDA’s required postapproval safety studies “to the fullest extent possible” when examining CAR T-cell therapies, which are approved to treat certain types of non-Hodgkin lymphoma and B-cell precursor acute lymphoblastic leukemia.
There are 2 approved CAR T-cell therapies on the market: Novartis’ product, tisagenlecleucel (Kymriah)—a treatment for children and young adults with B-cell acute lymphoblastic leukemia, and axicabtagene ciloleucel (Yescarta)— for adult patients with relapsed or refractory large B-cell lymphoma.
The Novartis product is priced at about $475,000 for a 1-time treatment and the Kite Pharma/Gilead treatment is priced at $373,000. But it’s estimated that the total cost of treatment can run between $800,000 and $1.5 million. Within the past week
, CMS finalized a payment decision for 2020, boosting the new technology add-on payment to 65%, up from the current 50%; that translates into a maximum add-on of $242,450, up from the current $186,500 payment.
“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 1400 patients treated for cancer with CAR T-cell therapies to date.
To reach its decision, CMS reviewed the evidence and also considered a report
from the Institute for Clinical and Economic Review (ICER), which said the the therapies are priced in alignment with their clinical value. CMS also reviewed evidence-based guidelines from the NCCN.
A year ago, the Medicare Evidence Development and Coverage Advisory Committee also met
, with panelists hearing from CAR T therapy drug makers, health researchers, and policy makers. Much of the meeting focused on the role of patient-reported outcomes and whether that should be included in any NCD. In the decision released Wednesday, CMS acknowledged that it had received a wide range of comments about the topic and said it encouraged patient participation in ongoing research that may include PROs in the future. But incorporating them as part of the NCD was not included.