Novartis’ CAR-T therapy for acute lymphoblastic leukemia in pediatric patients appears to significantly increase survival while exerting costs to the healthcare system that are in line with its benefits, a new study has found.

The study, published Monday in JAMA Pediatrics, found that 40 percent of patients treated with Kymriah (tisagenlecleucel) will likely become long-term survivors, meaning alive and still responding to treatment five years later. That compares with the 10-20 percent of younger ALL patients alive and disease-free after five years.

Additionally, Kymriah, whose list price is $475,000, was shown to have a cost per quality-adjusted life year, or cost per QALY, of $46,000, in comparison with clofarabine chemotherapy. Across scenario analyses, the cost per QALY ranged from $37,000-78,000. Cost per QALY measures the cost of a treatment for one year of good health. All in all, the study reported, Kymriah likely provides survival gains and appears priced in alignment with them.

The study collected data from three clinical trials and calculated long-term survival and outcomes using modeling from extrapolation of event-free and overall survival curves. Funding came from the Boston-based Institute for Clinical and Economic Review, or ICER.

Kymriah received Food and Drug Administration approval for pediatric ALL last August, followed the October approval of another CAR-T, Yescarta (axicabtagene ciloleucel), made by Kite Pharma, which Gilead Sciences subsequently acquired for $11.9 billion. Yescarta was approved for diffuse large B-cell lymphoma in adults, an indication for which Kymriah also received approval earlier this year. Both CAR-Ts target CD19, a cell-surface antigen common in ALL and DLBCL, but they also have a number of structural differences.

“Our models estimate that approximately 40 percent of acute lymphoblastic leukemia patients that initiate Kymriah treatment will be long-term survivors and would thus experience a similar life expectancy as a healthy individual, with adjustments made for an increased mortality due to being a survivor of leukemia,” lead study author Melanie Whittington, a research instructor at the University of Colorado School of Pharmacy, wrote in an email.

However, while the study indicated that the cost per QALY indicates that the therapies are cost effective, significant issues remain with respect to how they are reimbursed. In addition to the list prices – Kymriah’s is $475,000 for pediatric ALL, while both it and Yescarta are $373,000 for adult DLBCL – CAR-Ts also carry significant ancillary costs for preparation, administration, hospitalization and treatment for side effects. “Uncertainties remain on how this novel and high-investment treatment will be reimbursed,” Whittington wrote. “CAR-T is unique from any other treatments in that it is a one-time administration and is thus associated with significant upfront costs.” Consequently, she said, there needs to be innovation in payment models and benefit designs to accommodate them.

Kymriah is offered at 68 hospitals around the country, including the University of Colorado, according to its product site. Yescarta is offered at 61.

Pratap Khedkar, managing principal at management consulting firm ZS, had similar concerns about reimbursement. In DLBCL, every hospital that treats CAR-T patients today is losing about $50,000 per patient, in part due in part to the way payments form Medicare are structured. “So reimbursement has to be modified,” he said in an in-person interview. “It’s a one-shot cure that’s extremely expensive, but reimbursement policies have not caught up.” The risk is that hospitals may decide to perform a couple of procedures annually, but as more CAR-Ts enter the market and volume increases, they will decide going beyond that isn’t worth the trouble.

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