BioPharma, Policy

Medicare advisory panel vote could mandate patient-reported outcomes for CAR-T coverage

Non-binding vote by MEDCAC supports use of PROs in CAR-T clinical trials, drawing opposition from industry and oncology groups.

A Centers for Medicare and Medicaid Services expert advisory panel voted Wednesday to support the use of patient-reported outcomes in CAR-T therapy trials. If CMS follows the vote in its coverage decision, it could make PROs a requirement for Medicare coverage of the cell therapies, a move that has drawn opposition from industry and other groups.

The Medicare Evidence Development & Coverage Advisory Committee voted that available evidence supported the ability of four of the seven suggested PRO assessment systems to measure outcomes in future clinical trials. CMS considers MEDCAC’s advice, but is not bound to follow it. The panel’s 13 members include industry, payer, patient-advocacy and academic representatives.

The meeting was convened as part of a national coverage determination analysis requested in February by UnitedHealthcare amid concerns that the effective, but expensive cell therapies for cancers could create financial risks for CMS. The meeting’s purpose was to assess whether scientific evidence supports the various PRO assessment systems, clinical trial design characteristics and durations and suitable controls for applying PROs – patient status reports that come directly from patients themselves – to health outcomes research.

However, MEDCAC’s endorsement of PROs means Medicare coverage could become contingent on their inclusion in trials, a potential policy outcome to which several groups objected in written remarks to the panel, including the Biotechnology Innovation Organization, the American Society for Blood and Marrow Transplantation and the Alliance of Dedicated Cancer Centers.

The NCD analysis and potential for PROs being required drew concern from the manufacturers of currently marketed CAR-Ts as well.

A presentation at the meeting by Kite Pharma – the division of Gilead Sciences that developed the CAR-T Yescarta (axicabtagene ciloleucel) – stated that while Kite recognizes the importance of PROs in clinical trials and has used them in the Phase III ZUMA-7 study of Yescarta, the science supporting them in CAR-T is still evolving. Presentations by Novartis – which markets the CAR-T Kymriah (tisagenlecleucel) – urged CMS to withdraw the national coverage analysis and said mandating PRO data collection is “unnecessary, impractical, and imposes a significant burden on providers and patients outside of clinical trials.”

“Following the MEDCAC vote, Novartis will continue to work with CMS to highlight the importance of these innovative and groundbreaking products and to provide guidance to the MEDCAC on the relevance and applicability of PROs to CAR-Ts and cancer care,” read an emailed statement by Novartis. Gilead did not respond to requests for comment.

Yescarta and Kymriah both carry a $373,000 list price in DLBCL, while Kymriah’s ALL list price is $475,000, not including ancillary service costs estimated to run into the hundreds of thousands of dollars. In connection with the NCD analysis, Dr. Peter Bach, director for health policy and outcomes at New York’s Memorial Sloan Kettering Cancer Center, suggested price competition and randomized controlled trials as a means to ensure even coverage in a New England Journal of Medicine paper last week.

Other CAR-Ts in development include JCAR017 (lisocabtagene maraleucel) from Juno Therapeutics, a Seattle-based company acquired by Celgene, and bb2121 for multiple myeloma by bluebird bio, with which Celgene is partnered. Celgene, along with other organizations, was among the groups presenting at the meeting, and also expressed concerns about mandating PROs.

Photo: nopparit, Getty Images