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Tisagenlecleucel (Kymriah)
Tisagenlecleucel has the potential to be cost-effective for pediatric B-cell acute lymphoblastic leukemia (B-ALL) patients in the United States, according to researchers.
The group found evidence to suggest the chimeric antigen receptor (CAR) T-cell therapy—which has a list price of $475,000—may prove cost-effective if long-term survival benefits are realized.
An analysis indicated that the incremental cost-effectiveness ratio for tisagenlecleucel compared to clofarabine ranged from $37,000 to $78,000 per quality-adjusted life year (QALY) gained.
Melanie D. Whittington, PhD, of the University of Colorado at Denver, Aurora, and her colleagues described this work in JAMA Pediatrics.
For this study, the researchers used a decision analytic model that extrapolated the evidence from clinical trials over a patient’s lifetime to assess life-years gained, QALYs gained, and incremental costs per life-year and QALY gained. The researchers compared tisagenlecleucel to the antineoplastic agent clofarabine.
While tisagenlecleucel has a list price of $475,000, researchers discounted the price by 3% and added several additional costs, such as hospital administration, pretreatment, and potential adverse events, to get to a total discounted cost of about $667,000.
The team estimated that 42.6% of B-ALL patients would be long-term survivors with tisagenlecleucel, 10.34 life-years would be gained, and 9.28 QALYs would be gained.
In comparison, clofarabine had a total discounted cost of approximately $337,000, which included an initial discounted price of $164,000 plus additional treatment and administrative costs.
With clofarabine, 10.8% of B-ALL patients were long-term survivors, 2.43 life-years were gained, and 2.10 QALYs were gained in the model.
Overall, the mean incremental cost-effectiveness ratio was about $46,000 per QALY gained in this base-case model.
In analyses of different scenarios, such as a deeper discount, a different treatment start, or a different calculation of future treatment costs, the cost-effectiveness ratio varied from $37,000 to $78,000 per QALY gained.
The researchers noted that clinical trial evidence for tisagenlecleucel came from single-arm trials, which made the selection of a comparator challenging. Clofarabine was chosen because it had the most similar baseline population characteristics, but the researchers acknowledged that blinatumomab is also frequently used as a treatment for these patients.
“We suspect that tisagenlecleucel would remain cost-effective compared with blinatumomab,” the researchers wrote in JAMA Pediatrics. “A study conducted by other researchers found the incremental cost-effectiveness ratio of tisagenlecleucel versus blinatumomab was similar to the incremental cost-effectiveness ratio of tisagenlecleucel versus clofarabine [i.e., $3,000 more per QALY].”
The researchers suggested that uncertainties in the evidence should be considered as payers are negotiating coverage and payment for tisagenlecleucel.
This study was funded by the Institute for Clinical and Economic Review, which receives some funding from the pharmaceutical industry. Four study authors are employees of the Institute for Clinical and Economic Review.