CMS Proposes That Patients Be Enrolled in Studies to Get Coverage for CAR T-Cell Therapy

CMS has called for enrolling every patient in a clinical trial or registry if they are to get Medicare coverage for chimeric antigen receptor (CAR) T-cell therapy, a decision that may leave some without access to the life-saving cancer treatment.

Called Coverage with Evidence Development (CED), the payment model requires patients to enroll in CMS registries and be tracked for 2 years for institutions to receive Medicare reimbursement for CAR T-cell therapy. According to a CMS statement, policy makers will then use this data to determine who has benefited from the treatment; this will inform a long-term policy for patients not enrolled in studies or registries.

This proposed National Coverage Determination (NCD) would replace the current vacuum that allows regional Medicare Administrative Contractors make payment decisions. This has had the de facto effect of leaving academic institutions on the hook for the therapy portion of this revolutionary cellular treatment, which involves a multistep process that engineers a patient’s T cells and returns them to the body, where they harness the immune system to attack cancer cells.

“CAR T-cell therapy was the first FDA-approved gene therapy, marking the beginning of an entirely new approach to treating serious and even life-threatening diseases,” CMS Administrator Seema Verma said in the statement. “Today’s proposed coverage decision would improve access to this therapy while deepening CMS’s understanding of how patients in Medicare respond to it, so the agency can ensure that it is paying for CAR T-cell therapy for cases in which the benefits outweigh the risks.”

Today’s announcement marks a milestone that major cancer centers approved to administer CAR T-cell therapy have been seeking, but not necessarily the one they wanted. It is unclear how cancer centers will react to the CED model, or whether it will serve as a barometer for commercial payers, as CMS policies often do.

Joseph Alvarnas, MD, a hematologist/oncologist who serves as vice president for Government Affairs and senior medical director for Employer Strategy at City of Hope in Duarte, California, said cancer centers will need time to process what the NCD proposal means. However, he said the CED model adds significant reporting requirements, and that could prove problematic for some institutions.

"It adds to the burden, and anything that adds to the burden increases the risk of disenfranchising some patients," he said in an interview with The American Journal of Managed Care^® (AJMC^®). Already, patients in rural areas have difficulty accessing centers authorized to administer CAR T-cell therapy, he said, and with this proposal, "I think there will be places that will choose not to do it."

On the plus side, Alvarnas said, CMS will gather a large data set that sheds light on how patients are affected by CAR T-cell therapy. He did not think the NCD would offer much guidance for commercial payers, however; even in Medicare, the proposal does not resolve questions about reimbursement.

"In the provider space, our hope is to make sure we are getting treatment to patients who need it without undue delay or the potential for added burdens," said Alvarnas, who is also the editor-in-chief for AJMC^®'s Evidence-Based Oncology™.

Novartis, which received approval for the first CAR T-cell therapy in August 2017, tried to avoid the payment gap that often occurs when novel therapies first reach the market. The pharmaceutical company had reached a value-based agreement with CMS that called for reimbursement only if the treatment worked, which was announced alongside the approval for tisagenlecleucel (Kymriah). But the agreement was quietly withdrawn after UnitedHealthcare asked CMS to pursue a National Coverage Analysis.

For months, institutions have been treating patients without CMS reimbursement for the customized therapies, which have some of highest list prices ever seen in cancer care. Tisagenlecleucel was initially approved to treat pediatric acute lymphoblastic leukemia for a list price of $475,000. A second treatment, axicabtagene ciloleucel (axi-cel, Yescarta), was approved in October 2017 for relapsed or refractory large B-cell lymphoma; it has a list price of $393,000.

In a statement, CMS officials stated that a final plan will be issued no less than 60 days after a 30-day comment period. Of note:

• The proposed NCD has highly specific criteria for what types of institutions can be reimbursed for CAR T-cell therapy, including staff they must employ.
• The plan outlines information that must be tracked, based on whether the patient receives therapy as an inpatient or an outpatient.
• The plan spells out which measurement tools should be used to track patient reported outcomes (PROs) and at what intervals. Manufacturers of the first approved therapies criticized using PROs at an August 2018 hearing of the Medicare Evidence Development & Coverage Advisory Committee (MEDCAC).
• Repeat treatments will not be funded unless a new primary cancer is diagnosed.
• The “furnishing hospital” treating patients must accept “all manufactured products.”

Nathan Kaiser, a spokesman with Gilead, which acquired Kite Pharma, maker of axi-cel, said the company was still reviewing the decision. At the August 2018 hearing about PROs, William Go, MD, PhD, vice president of clinical development at Kite, said PROs are “not quite ready for real-world coverage decisions.”

"We are encouraged though by the CMS statements related to coverage of CAR T-cell therapy. We want to ensure all patients have access to current and future CAR T-cell therapy, including Yescarta. As such, we will continue to work with CMS, including submission of public comments during the comment period," said Kaiser.

UnitedHealthcare, the nation's largest insurer and also the largest provider of Medicare Advantage plans, requested the NCD last year. It did not immediately return an email and phone call about the decision Friday.

Last fall, CMS announced how much it would pay for each CAR T-cell treatment—a maximum of $186,500 per case starting this year—but academic medical centers that treat patients with blood cancers could not be paid without the NCD in place.

Tisagenlecleucel has received a second indication for large B-cell lymphoma, and axi-cel has been approved in 3 other conditions. CAR T-cell therapy is being studied in non-Hodgkin lymphoma and multiple myeloma, among other diseases. FDA is expected to approve both new therapies and new indications for existing therapies in the near term.