A new report published by Boston’s Institute for Clinical and Economic Review (ICER) concludes that two pricey new CAR-T cell therapies, Novartis AG’s Kymriah (tisagenlecleucel) and Gilead Sciences Inc.’s Yescarta (axicabtagene ciloleucel), both provided moderate certainty of a small to substantial net health benefit compared to commonly used chemotherapies or no therapy at all in their respective indications.
“While many medicines have U.S. prices that surpass the value they deliver, our analysis on these two CAR-T therapies underscores that certain medicines can have high prices and still be cost-effective for the health care system,” ICER representative David Whitrap told BioWorld. “Based on ICER’s extrapolation of the current survival curves, both Kymriah and Yescarta appear to be priced in line with the value they deliver to patients.”
A draft of the report, released Dec. 19 ahead of the final report’s arrival Feb. 15, drew mixed reviews from stakeholders, some of whom suggested it was premature considering the limited evidence on the therapies to date or that cost estimates cited might be off-base. Others expressed appreciation for the review and its potential contribution to debates over value-based pricing.
Both Kymriah and Yescarta were approved last year based on relatively small, single-arm designs with limited follow-up. (See BioWorld, Aug. 31, 2017, and Oct. 20, 2017.)
A March 2 meeting of the California Technology Assessment Forum, one of ICER’s three independent evidence appraisal committees, will host a vote on whether the new therapies offer long-term value for money and other issues. After that, the institute will release a summary including the committee’s votes, along with key policy recommendations arising from a roundtable discussion.
Whitrap said that he expects that questions about how to best construct outcomes-based contracts for CAR-T therapies and how indication-specific pricing might be implemented if the therapies are approved for additional indications are likely to draw particular interest during the meeting. Questions about what an appropriate physician markup should be for the administration of the treatments and how payers will cope with the budget impact of both approved and forthcoming CAR-T therapies, are also likely to arise, he said.
ICER’s review found that using Kymriah to treat children with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) instead of the previous standard of care increases health care costs by about $330,000, but extends life by eight years. Using Yescarta instead of chemotherapy to treat adults with relapsed or refractory B-cell lymphoma increases health costs by $460,000 but extends life by four years, the institute said.
The institutes’s value-based price benchmark for Kymriah, including its estimated mark-up to administer the therapy, is between $1,162,563 and $1,688,232. A price above that range would exceed common thresholds for cost-effectiveness, Whitrap said. ICER’s value-based price benchmark for Yescarta, including estimated markup, is between $340,797 and $524,015.
The cost-effectiveness of each therapy fell below or within commonly cited thresholds of $50,000-$150,000 per quality-adjusted life year, ICER said.
The report assumes that a hospital markup for the therapies is not included as part of outcomes-based contracts between the therapies’ manufacturers and the payers, such that payment from the payer to the hospital for the markup would occur regardless of whether or not patients respond. Kymriah was launched with an outcomes-based pricing arrangement with the Centers for Medicare & Medicaid Services, while Gilead has yet to adopt such a model for Yescarta, though has suggested it would be open to entering value- or outcomes-based payment contracts with payers on a case-by-case basis.
Yescarta was developed by Santa Monica, Calif.-based Kite Pharma Inc., which in August attracted an $11.9 billion acquisition deal from Foster City, Calif.-based Gilead. (See BioWorld, Aug. 29, 2017.)
In comments submitted based on the draft report, Kite wrote that “the eligibility and uptake of Yescarta will not lead to a substantial budget impact” and voiced concern that “ICER’s estimate of the eligible population for Yescarta likely overestimates the actual number of patients who will receive treatment.”
In response to a similar complaint from Novartis, ICER recalculated the size of the eligible population of Kymriah to 400 patients annually from an earlier 617.
The Leukemia & Lymphoma Society also weighed in on the report. Gwen Nichols, the group’s chief medical officer, told BioWorld that “CAR-T therapy is clearly costly but particularly in the case of children, the value is very clear. A leukemia diagnosis is incredibly disruptive for the life of a child who must undergo repeated cycles of treatments, infusions, time away from family, home and school, over multiple years.”
When standard therapy doesn’t work, CAR-T therapies can give kids a chance to have a normal life after a one-time infusion, she said. “LLS continues to support research on CAR-T and other immunotherapeutic approaches that will provide less complex (and thus less costly) therapies with even greater safety and efficacy,” she added.
ICER’s final evidence report, accompanying voting questions, public comments and the institute’s response are available on the group’s website. The meeting of the California Technology Assessment Forum is on March 2 in Oakland, Calif.
Feb, 17 2018