Medicare Coverage of CAR-T Cell Therapy Opens Patient Access to Life-Saving Treatment

The Centers for Medicare and Medicaid Services (CMS) announced recently that it will cover chimeric antigen receptor (CAR)-T cell therapy nationwide — offering patients more consistent and predictable access to this lifesaving therapy, according to Dr. Roy Silverstein.

“One of the problems right now in our field is that we’ve developed these exciting new drugs and cellular therapies, but because of the reimbursement issues and expense, they are not as widely available as we would like for many patients who could benefit from them and don’t have access to them,” Silverstein, who is the president of the American Society of Hematology (ASH), explained in an interview with CURE. “So, what we are hoping is that by CMS changing their rules, it will open up more sites to deliver these therapies.”

CAR-T Cell Therapy Costs

The Food and Drug Administration (FDA) has already approved two CAR-T cell therapies: Kymriah (tisagenle­cleucel) and Yescarta (axicabtagene ciloleucel), for some adults and children with certain types of blood cancers, including:

• B-cell precursor acute lymphoblastic leukemia, in people up to 25 years of age
• Diffuse large B-cell lymphoma
• Primary mediastinal large B-cell lymphoma
• Large B-cell lymphoma transformed from follicular lymphoma
• High grade B-cell lymphoma
• Aggressive B-cell lymphoma not otherwise specified (NOS)

CAR-T cell therapy works by removing a patient’s T cells, re-engineering and genetically modifying them in a lab and then putting them back into the patient’s body to fight the individual’s cancer by attacking their malignant cells.

Due to the extensive process that goes into engineering CAR-T cell therapies, these drugs are highly expensive, ranging from an estimated $375,000 or $475,000 in cost.

“It is a very complicated and lengthy process that is individualized for every patient. It takes weeks and weeks from beginning to end to make the drug for that patient, and it is actually a living drug so there is a lot of quality control and a lot of complicated steps along the way,” Silverstein explained, adding that this type of therapy is very different than any other kind of drug that currently exists.

“The manufacturing is truly individualized for every patient. There is not a factory that can make it and send it for hospitals to store for when it is ready,” he said. “And also, it has taken many years of research and development, so there are costs associated with that to develop the drug.”

Outside of the drug itself, the care associated with treatment is just as expensive. When a patient is treated with CAR-T cell therapy, Silverstein noted, there are often lengthy hospital stays as well. “When you add it all up, it can be over $1 million for each patient.”

Medicare Coverage

With this decision (which was originally intended to be made by late May), the CMS also announced that it changed certain aspects from its original proposal that was issued in February.

The agency’s original proposal called for “coverage with evidence development” — meaning hospitals would have been required to collect and report data on patient outcomes over a long period of time. A burdensome task, the CMS removed this requirement and announced it would instead rely on patient information collected by the FDA and the National Cancer Institute (NCI).

With this, the FDA is now requiring Novartis and Gilead — the manufacturers of the currently approved CAR-T cell therapies – to follow patients and report on their outcomes. In turn, this data will be entered into an NCI registry.

In addition, the CMS announced it would cover CAR-T cell therapy when it is administered at health care facilities enrolled in an FDA-mandated safety program requiring special training on handling side effects. The plus side to this is that patients may have access to treatment on an outpatient basis — in turn, offsetting some of the therapy’s costs, Silverstein said.

“Hopefully as we learn more about (CAR-T cell therapies) and the CMS learns more about them, we will work together to help them develop more modern methods for reimbursing for these new kinds of drugs,” he added. “We hope that this will continue to improve.”

Patient Benefits

Of note, this also means the CMS modified its requirements for patients to be eligible for CAR-T cell therapy, including FDA-approved uses as well as off-label uses recommended in CMS-approved compendiums (which determine medically-accepted uses of drugs and biologics in the agency).

“Initially they were only talking about patients with FDA-approved indications, and now they have loosened that to allow physicians who believe their patients would benefit from this, and where there is some evidence from clinical research that patients could benefit from this, to allow them to also get the therapy,” said Silverstein, who is also the Linda and John Mellowes Professor of Medicine and an associate director of clinomics at the Genomic Sciences and Precision Medicine Center at Medical College of Wisconsin.

Even better, this decision will not affect patients’ out-of-pock costs, he added.

“These therapies are so exciting and they’ve been a true breakthrough for treating patients with blood cancers,” Silberstein said. “In the future, we hope that they develop these drugs for other kinds of cancers as well. We are extremely excited about these therapies and really hopeful that CMS will continue to work with the professional organizations, like ASH, and the health systems to develop more modern and creative ways to reimburse the health systems for the care of these patients that receive these treatments.”