BioPharma, Policy

CMS delays national coverage determination for CAR-T therapies

In a statement, the agency said that a decision on whether to have Medicare cover the cell therapies would not be issued Friday, but would be forthcoming.

The Centers for Medicare and Medicaid Services is holding off on deciding whether CAR-T cell therapies will be covered under Medicare.

The agency said Friday that it had delayed its national coverage determination for CAR-Ts, quoting an unnamed CMS spokesperson as saying that it would not be issuing the determination for CAR-T cell therapy on Friday, but that a decision was forthcoming. It posted a proposed decision memo in February.

sponsored content

A Deep-dive Into Specialty Pharma

A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.

Two CAR-T therapies currently have Food and Drug Administration approval, namely Gilead Sciences’ Yescarta (axicabtagene ciloleucel), for diffuse large B-cell lymphoma in adults, and Novartis’ Kymriah (tisagenlecleucel), for adult DLBCL and pediatric acute lymphoblastic leukemia. Others are in development, for leukemias and lymphomas, multiple myeloma and solid tumors. Celgene subsidiary Juno Therapeutics is developing another CAR-T for DLBCL and chronic lymphocytic leukemia, lisocabtagene maraleucel. Celgene is also partnered with bluebird bio, whose CAR-T bb2121 is in late-stage development for multiple myeloma.

In August 2018, the Medicare Evidence Development and Coverage Advisory Committee, or MEDCAC, voted to support the use of patient-reported outcomes in CAR-T clinical trials, which could make PROs a requirement for Medicare coverage of the cell therapies. The move drew opposition from industry and other groups. However, while it considers MEDCAC’s advice, CMS is not required to follow votes by the committee, whose 13 members include industry, payer, patient advocacy and academic representatives. The meeting in August stemmed from an NCD analysis requested in February 2018 by UnitedHealthcare amid concerns that CAR-T cell therapies could create financial risks for CMS.

How to pay for CAR-T therapies presents a significant dilemma, especially for CMS – one that will become amplified as the therapies move from relatively rare blood cancers to much more common solid tumors. Both approved CAR-Ts carry list prices of $373,000 for DLBCL and, for Kymriah in ALL, $475,000, on top of supportive care costs that can equal or exceed the list prices. When Kymriah was launched for ALL, Novartis and CMS also piloted a pay-for-performance program, whereby reimbursement would be contingent on the therapy being effective. Although the deal presented little financial risk, given the complete remission rates north of 80 percent that Kymriah produced in that disease, CMS quietly pulled out of the program last year amid concerns of undue industry influence on the government stemming from an inquiry into payments Novartis made to President Donald Trump’s former lawyer and fixer, Michael Cohen.

Photo: CGToolbox, Getty Images