Medicare will begin covering the growing collection of immunotherapy drugs referred to as CAR T-cell therapy for cancer patients being treated in some healthcare facilities, the Centers for Medicare & Medicaid Services announced Wednesday.
A growing number of drug companies have begun seeking and achieving regulatory approval for chimeric antigen receptor (CAR) T-cell therapy, in which oncologists use a patient’s own immune system to attack certain cancers. Food and Drug Administration-approved CAR T-cell therapies have been OK'd to treat some people with specific types of cancer including certain types of non-Hodgkin lymphoma and B-cell precursor acute lymphoblastic leukemia.
"In the absence of a national coverage decision, basically, hospitals and providers are going to our Medicare contractors, or MACs, and are making these decisions. There's a lot of confusions about 'Is this covered? How is it covered?'" CMS Administrator Seema Verma said in a call with reporters. "Today's decision makes it very clear that 'Yes, this is covered.' We're paying that not only for CAR T, but all the related services. The administration of the drug. The collection of the cells. The manipulation of the cells and then putting it back into the patient. And then any of their outpatient or inpatient care as well."
Medicare will cover the therapies when they are provided in healthcare facilities enrolled in the FDA risk evaluation and mitigation strategies (REMS) for FDA-approved indications, officials said. In addition, Medicare will cover the FDA-approved therapies for off-label uses that are recommended by CMS'-approved compendia.
The announcement comes on the heels of a CMS announcement last week that, among other things, increased what it would pay new technology add-on payments from 50% to 65% in FY2020.
RELATED: CMS announces delay of CAR T-cell national coverage determination
It also comes after CMS announced a delay to its national coverage determination in May after being inundated with comments from industry. CMS first initiated a national coverage analysis for CAR T-cell therapy for cancers in May 2018 and began considering public comments. In February, CMS posted a proposed decision memo saying it intends to allow Medicare to pay for CAR T.
Groups like the American Society for Transplantation and Cellular Therapy (ASTCT) have said making a national coverage determination was "premature" citing concerns that it would create barriers to providing current and future CAR-T therapies. Patients who receive CAR T have typically exhausted all other available therapies.
However, manufacturers backed a coverage determination, pointing out the growing evidence supporting CAR T-cell therapy.
Among them was Gilead's Kite Pharma, which manufacturers Yescarta, the first FDA-approved CAR T-cell therapy for adults with relapsed or refractory large B-cell lymphoma. "We believe that the existing and growing body of evidence strongly supports that CAR T-cell therapy is reasonable and necessary for Medicare beneficiaries," wrote Michael Amoroso, senior vice president and head of worldwide commercial cell therapy at Kite Pharma earleir this year.
"This is important for our beneficiaries to have access to the latest treatments and therapies," Verma said. "In the case of CAR T, this is a life-saving treatment for people that really don't have any other options," Verma said. "We're making sure that that access is available while we are also monitoring the outcomes-related data."
Future monitoring and costs
In CAR T-cell therapy, a patient’s white blood cells are extracted and modified with a receptor that drives the cells to seek out the cancer and attack it. The ultimate goal of the therapy is to not only kill existing tumors but provide an internal line of defense against future cancers.
But the still-emerging technology is pricey and it comes with risks.
CMS said it will be important to closely monitor responses to CAR T cell therapies in the Medicare population, since outcomes data for these patients are relatively limited and the treatment represents a significant change from current practices.
"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, M.D., in a statement.
Verma declined to offer specifics on what the change would ultimately cost the Medicare system.
"I don't know that we've had an overall study to tell you this is going to be the cost to the overall Medicare program and when we are making coverage decisions, we are making that separate from how much this is going to cost the program," Verma said. "That being said, I do think the whole issue of CAR T is really bringing up the issue of how expensive the new therapies that are coming out. They're life-saving and they're transforming and we haven't seen this type of development before in terms of curative treatments. But I think it's sort of begging the question, 'How is the system going to pay for this over the long term?'"
Kite won FDA approval in October 2017 for Yescarta which has a list price of $373,000. Yescarta’s sole challenger in the field, Novartis’ Kymriah, lists for $475,000.
"At a time when the Medicare trustees have warned us that we're having problems paying for the Medicare program in six or seven years, this is something we're deeply concerned about," Verma said. "At the same time, we're committed to strengthening the program and want to make sure our beneficiaries have access to life-saving treatments. That's our first priority."
She also declined to give estimates of how much this would cost hospitals. She also said it's important to wait to collect cost data because the market will adjust.
"A lot of times hospitals are able to negotiate with the manufacturers on the pricing. That's why it's premature for us to set a price or put anything out there because we also want to give time for the market to respond and to allow that negotiation to occur. So after some time, we can see what they've negotiated and we're better able to determine that pricing."