Medicare Moves Closer to CAR-T Coverage Policy

Kerry Dooley Young

February 18, 2019

Medicare has proposed a national coverage policy for chimeric antigen receptor T-cell (CAR-T) therapy with several conditions attached, including monitoring of patients enrolled in an approved registry or clinical trial for at least 2 years after treatment.

The Centers for Medicare & Medicaid Services (CMS) has unveiled a proposed national coverage decision (NCD) memo.

The NCD would be Medicare's first sweeping payment policy for CAR-T, which involves genetically modifying a patient's own T cells to fight against certain leukemias and lymphomas.

At present, local Medicare administrative contractors can decide whether to pay for CAR-T therapy, CMS said. There is hope that a national policy will improve access to these novel therapies.

CAR-T therapies were launched into the US market in 2017 with high prices, about $475,000 for tisagenlecleucel (Kymriah, Novartis) and $373,000 for axicabtagene ciloleucel (Yescarta, Gilead Sciences).

These high costs, as well as problems with reimbursement and insurance coverage, have meant that few patients have received these novel therapies, as reported by Medscape Medical News.

Experts treating hematologic malignancies are enthusiastic about the novel therapies. They point out that clinical responses are far superior to anything that has been seen before. "When you see data like that, it becomes ethically challenging for people to say that we can't do this because of financial issues," one expert told Medscape Medical News last year.

Coverage With Evidence Development

In the proposed decision memo, CMS' medical staff describes CAR-T therapy as "a rapidly evolving field" for which "initial evidence appears promising but inconclusive."

The agency is seeking to require that the therapy be provided at certain well-prepared hospitals. In addition, CMS also said Medicare would require that the CAR-T therapy target a known antigen expressed in the patient's cancer that matches an approved indication for the drug used.

CMS said it intends to use data from registries and studies to help identify the types of patients who benefit from CAR-T therapy. This research could inform a future Medicare decision regarding the types of cases for which the treatment would be covered with no registry or trial requirement, CMS staff said.

Medicare's proposed national CAR-T coverage would use a strategy known as coverage with evidence development (CED). With CED, Medicare pays for emerging therapies while tracking how well they work for patients. CED mandates a flow of detailed information about patients who are given a particular therapy, as well as their outcomes.

"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," said CMS Administrator Seema Verma in a statement on February 15.

CAR-T therapy has been approved in the United States for adults with certain refractory or relapsed lymphomas, CMS said. In the memo, the agency staff notes that there is "very limited" understanding about whether people enrolled in Medicare will experience overall harm or benefit from CAR-T.

"The evidence base for the B-cell lymphoma clinical studies is currently small, especially for older adults where 33 patients who received axicabtagene ciloleucel and fewer than 28 patients who received tisagenlecleucel were 65 years old or older," CMS staff write.

Assessing Quality of Life

In its memo, CMS emphasizes a need for "valid and reliable measurement" of quality of life for those undergoing CAR-T therapy.

"Assessing quality of life and functional status beyond one year following CAR T-cell therapy may therefore be important for a patient deciding whether the benefit from CAR T-cell administration aligns with their care plans and goals," CMS staff write.

CMS initiated work on a national coverage decision on CAR-T therapy last year in response to a request from insurer UnitedHealthcare, which operates Medicare Advantage plans. CMS' Medicare Evidence Development & Coverage Advisory Committee in August 2018 provided feedback to the agency on approaches to monitoring the outcomes for people given CAR-T therapy.

CMS is accepting comments for the proposed NCD until March 17. The agency said it expects to issue a final decision no later than 60 days after the conclusion of the 30-day public comment period.


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