The expansion of coverage will make the treatment available to more people. However, experts say reimbursements to hospitals might be a problem.

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Two CAR-T treatments already have FDA approval. Others are in the clinical trial phase. Getty Images

CAR-T cell therapy, a new kind of cancer treatment that harnesses the body’s immune system to recognize and attack cancer cells, is the talk of the oncology world.

In the CAR-T process, a sample of a patient’s T cells are taken from the blood and modified to produce special structures called chimeric antigen receptors (CARs) on their surface.

When these cells are re-infused into the body, the new receptors enable them to target a specific antigen on the patient’s tumor cells and kill them, all without generally harming normal cells.

There are numerous CAR-T treatments in clinical trial, and two that have already been approved by the Food and Drug Administration (FDA): Yescarta and Kymriah.

Despite the positive results in clinic and in trials and declarations by cancer researchers that CAR-T could be a cure for certain blood cancers and some solid-tumor cancers as well, the immunotherapy has been in a sort of regulatory limbo.

There has been uncertainty about what the Centers for Medicare and Medicaid Services (CMS) would decide as far as CAR-T coverage for enrollees.

But that changed this month when CMS officials announced their agency will cover CAR-T for Medicare recipients, beginning October 1.

In a call with reporters, CMS Administrator Seema Verma said, “Today’s decision makes it very clear that, ‘Yes, this is covered.’”

The coverage only applies to Medicare, the federal insurance program for people 65 years and older. It does not apply to Medicaid, the insurance program for low-income individuals and families.

Before announcing its decision, CMS also dropped a proposal introduced in February that would have required hospitals to collect and report data on patient outcomes over a long period.

Some cancer hospital officials complained the requirement would be unnecessarily burdensome.

Health officials tell Healthline that the CMS decision is a significant milestone that will likely broaden access of this kind of treatment to thousands more people with cancer nationwide.

It could also positively influence many private insurers. Many already cover CAR-T, but typically private insurance companies are obliged to follow Medicare’s lead.

In a statement, Dr. Roy Silverstein, president of the American Society of Hematology (ASH), said:

“The American Society of Hematology commends CMS for issuing a broad coverage policy for this innovative, potentially life-saving treatment for Medicare beneficiaries and resolving to move forward in a way that limits additional burden on provider institutions and allows for coverage that can include future indications of CAR-T.”

But the coverage decision is tempered by a related CMS decision regarding the reimbursement percentage Medicare will provide to hospitals for the costs of CAR-T.

CMS officials announced earlier this month in its “final inpatient rule” that it was raising CAR-T reimbursement costs from 50 percent to 65 percent.

That disappointed many in the cancer field, who hoped the percentage would be bumped to 80 percent or even 100 percent.

“It cannot be overlooked that Medicare has now put providers in a difficult position,” said Silverstein in his statement.

Silverstein added that the CMS decision “does not do enough to adequately reimburse institutions for the cost of care or the cost of the product.”

He added that while this increase is an improvement, “it still leaves institutions to cover the remaining 35 percent of the product cost. Institutions will now be reimbursed $242,450 for the CAR-T product, which has a total cost of $373,000.”

Dr. Dimitrios Tzachanis, an associate clinical professor of medicine at the UC San Diego Moores Cancer Center, said the CMS decision on reimbursement is hopefully just a first step rather than the final say in the matter.

“I think some steps in the right direction have been made, but the decision to raise the reimbursement payment from 50 percent to 65 percent means that a potential amount of care will still not be covered,” Tzachanis told Healthline. “That leaves the institutions with a six-figure deficit with CAR-T therapy. That type of gap in reimbursement is unsustainable.”

Tzachanis said this decision is not an optimal one for people with cancer.

“It incentivizes cancer centers to do more of the care as outpatient rather than inpatient. For this treatment, that is not ideal. These decisions should not be based on financials,” he said.

Officials tell Healthline that the CMS decision on reimbursement to hospitals for CAR-T will force some institutions to deny CAR-T to their patients.

“They (CMS) are engaged in making this right. It is a complex issue, but this decision is not enough to convince those institutions that have been reticent to administer CAR-T,” Dr. Joseph Alvarnas, an oncologist at City of Hope in California who specializes in CAR-T, told Healthline.

Jayson Slotnik, a partner at the consulting firm Health Policy Strategies in Washington, D.C., told Healthline that the CMS decision on reimbursement was made for the wrong reasons.

“This was a political decision based on concern around high drug prices,” Slotnik said. “The question is: What are we not learning about these therapies, what is the opportunity lost here because of this near-sightedness related to drug pricing?”

But Slotnik said the decision to cover CAR-T is a good first step.

“As a result of this decision, the percentage of patients who will have access to CAR-T will be higher. But it’s not ideal,” Slotnik said.

“The ideal would be if CMS enacted a policy that made the reimbursement for these drugs more in line with acquisition costs, that would provide better service and better economics,” he added.

Chuck Fata is alive today because of CAR-T.

Nearly four years ago, Fata, a restaurant owner, avid athlete, and family man from Los Angeles, was diagnosed with diffuse large B-cell lymphoma (DLBCL), the most common type of non-Hodgkin’s lymphoma.

Fata immediately began a chemotherapy regimen, but his remission only lasted a few months.

In late 2015, the cancer recurred and Fata underwent an autologous bone marrow stem cell transplant.

“They did the stem cell. I went through it and got back to my life, and it was looking good,” he told Healthline.

But just a few months later, his doctors found another tumor in his stomach, which began to bleed. Half of Fata’s stomach was removed, along with the tail of his pancreas and his spleen.

But the cancer had spread to the lymph nodes in Fata’s neck and other areas. He had run out of viable treatment options. Fata’s future looked bleak.

“That’s when my doctors told me about CAR-T,” said Fata, who qualified for a CAR-T clinical trial because he had failed chemo and an autologous stem cell transplant.

Fata was sent to City of Hope in the Los Angeles area, where in February 2016 he enrolled the ZUMA-1 phase 1 CAR-T trial sponsored by Kite Pharma, now a subsidiary of Gilead Sciences.

When Fata returned to City of Hope a month after his trial to see how he was doing, he was not his typical confident self.

“I was skeptical because nothing else had worked for me,” he said.

But Dr. Tanya Siddiqi, the City of Hope hematologist/oncologist who was the principal investigator of the trial and Fata’s doctor, told Fata that he was in complete remission.

And today, Fata remains cancer-free.

“I am still amazed when I think of all the treatments I went through, from CHOP chemo to surgery, the recovery time, the stem cell transplant, and how complicated it all was,” he said.

“The CAR-T saved my life, and it was so quick. I went in and they took my T cells, two weeks later I did the rounds of chemo, I was in the hospital for about a week, and two weeks later I am in remission,” he added.

Fata is now back to snow skiing, playing racquetball, and running his restaurants.

But most of his time this summer has been dedicated to his family. Specifically, to planning not one but two weddings. Both of his daughters are getting married.

“CAR T was a miracle for me,” Fata said.

Fata’s clinical trial and others are what led the CAR-T with which he was treated, Yescarta, to be approved by the FDA.

Yescarta is the first FDA-approved CAR T-cell therapy for adult patients with certain types of B-cell lymphoma.

The FDA has approved this treatment for patients with the following conditions that have either not responded to or have relapsed following two or more lines of systemic therapy:

  • DLBCL, primary mediastinal B-cell lymphoma
  • high grade B-cell lymphoma
  • DLBCL that results from follicular lymphoma

“We applaud CMS’ continued actions to close the significant gap that exists in providing coverage for CAR-T cell therapy to Medicare beneficiaries, which will allow more patients access to this potentially life-saving therapy,” a representative from Kite Pharma, the drug’s manufacturer, told Healthline.

Kymriah, from Novartis, is an FDA-approved CAR T-cell therapy for adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) as well as young adult patients up to age 25 with relapsed or refractory acute lymphoblastic leukemia (ALL).

“Novartis applauds CMS for its decision to allow Medicare coverage nationally for CAR-T therapies,” Julie Masow, a Novartis representative, told Healthline.

“Instituting nationwide coverage will help this critically ill patient population [that has] few alternative options, and we appreciate that CMS recognizes the need to provide access to this groundbreaking therapy,” Masow said. “Novartis also continues to encourage CMS to develop reimbursement rates in the inpatient hospital setting that fully reflect the value of these innovative and groundbreaking products.”

Masow added that Novartis is committed to “doing everything we can to help patients who can benefit from Kymriah have access to the therapy. In the U.S., more than 90 treatment centers are qualified to administer Kymriah. We have manufactured Kymriah for more than 1,000 patients worldwide in the clinical and commercial setting.”

Masow explains that in the United States, the large national insurers have coverage policies for Kymriah.

“Many commercial payers require a prior authorization to ensure the patient meets their policy criteria, which for most payers aligns with the approved label and/or clinical guidelines like NCCN,” she said.

Novartis CAR-T pipeline is focused on continuing to develop treatments for B-cell malignancies and other blood cancers as well as solid tumors. It also focuses on researching next-generation CAR-Ts that focus on new targets and utilize new technologies.

There are clinical trials for multiple CAR-T treatments in the United States, China, Europe, and other regions.

Celgene plans to file for FDA approval of its CAR-T, liso-cel, for DLBCL during the fourth quarter of this year.

The company also anticipates filing for approval of its CAR-T, ide-cel, for multiple myeloma during the first half of next year.

A spokesperson for Celgene told Healthline, “Celgene supports CMS’s final coverage decision, which will help ensure Medicare patients can access CAR-T cell therapies. We are pleased the final decision includes modifications that ensure current and future cancer patients will be able to access these innovative, targeted and personalized therapies.”

When Alan Izumigawa, 51, an Army veteran from Riverside County in California, was diagnosed with DLBCL, he tried chemotherapy.

But it failed.

He was then denied a bone marrow transplant.

“I was supposed to have the bone marrow transplant in Seattle, but I still had lymphoma tumors, so I did not qualify,” he told Healthline.

Izumigawa was out of options. Then his doctors told him about Yescarta.

“I really wanted to do it,” said Izumigawa, who was sent by the Department of Veterans Affairs to the UC San Diego Moores Cancer Center to do the procedure in late April.

“It was an FDA-approved treatment, but we were told that this procedure would cost close to half a million dollars. But the VA coordinated and paid for treatment,” he said. “They told me I was among the first patients at that particular VA who was covered for CAR-T.”

Izumigawa, who grew up in Okinawa, Japan, and served in the U.S. Army from 1987 to 1991, is now in complete remission, his doctors say.

He is on 100 percent service-connected disability but is enjoying time with his family.

He did have some side effects from the treatment, including mental confusion and fever, both of which are common. But they only lasted about three days, said Izumigawa.

“I would recommend it. It’s a good therapy. It really works,” he said. “When we were denied the bone marrow transplant, there were no other options available for me. CAR-T was a lifesaver. I can now help out my mom.”

Slotnik said that with game-changing medicines such as CAR-T, there should be “no compromise” in terms of patient access or coverage.

“My life experience has been watching us go from toxic chemotherapy to monoclonal antibodies to using the immune system to fight cancer,” he said. “These treatments are turning what was a death sentence to an acute disease. Look at what Gleevec has done. It gives a lot of people hope, and not just for cancer but for all diseases.”

Slotnik said that perhaps it’s time for the public to get involved.

“Consumers in this country still have a lot of power and rights, including the right to speak up, their voices carry a lot of weight as taxpayers and voters,” Slotnik said.

“If they don’t like what they are seeing and hearing, they should make their voice be heard in a constructive way by calling their representatives and reaching out to CMS,” he added.