Abstract
Axicabtagene ciloleucel, a chimeric antigen receptor T-cell therapy, represents a new and potentially curative treatment option for B-cell lymphoma. It is expected to have long-term survival benefits; however, long-term survival data are limited. To estimate the long-term survival and cost-effectiveness of axicabtagene ciloleucel for treatment of relapsed or refractory B-cell lymphoma. Economic evaluation study using a survival analysis that digitized and extrapolated survival curves published in the ZUMA-1 trial (Safety and Efficacy of KTE-C19 in Adults With Refractory Aggressive Non-Hodgkin Lymphoma), which enrolled patients between November 2015 and September 2016 and had a maximum follow-up of 24 months. Five different survival models (standard parametric, flexible parametric, 2 mixture cure models, and a flexible parametric mixture model) were used to extrapolate the survival curves to a lifetime horizon from January through June 2018. A cost-effectiveness analysis, from both a trial-based and lifetime horizon, was also conducted to inform the value of this novel therapy. The model was based on data from 111 patients with B-cell lymphoma who were enrolled in the ZUMA-1 trial. One-time administration of axicabtagene ciloleucel compared with chemotherapy. Undiscounted and discounted life-years (LYs) and quality-adjusted life-years (QALYs), total costs, and incremental costs per LY and QALY gained. The modeled cohort of 111 patients started at 58 years of age. At the end of the trial, treatment with axicabtagene ciloleucel resulted in 0.48 more LYs and 0.34 more QALYs than chemotherapy, producing a cost-effectiveness estimate of $896 600 per QALY for public payers and $1 615 000 per QALY for commercial payers. Extrapolated long-term survival for patients treated with axicabtagene ciloleucel ranged from 2.83 to 9.19 discounted LYs and from 2.07 to 7.62 discounted QALYs. Incrementally, treatment with axicabtagene ciloleucel was associated with 1.89 to 5.82 discounted LYs and 1.52 to 4.90 discounted QALYs vs chemotherapy. With the use of these incremental estimates of survival, cost-effectiveness estimates ranged from $82 400 to $230 900 per QALY gained for public payers and from $100 400 to $289 000 per QALY gained for commercial payers. Treatment with axicabtagene ciloleucel appears to be associated with incremental gains in survival over chemotherapy. The range in projected long-term survival was wide and reflected uncertainty owing to limited follow-up data. Cost-effectiveness is associated with long-term survival, with further evidence needed to reduce uncertainty.
Highlights
Diffuse, large B-cell lymphoma is the most common subtype of non-Hodgkin lymphoma in the United States, accounting for 30% to 40% of all non-Hodgkin lymphoma cases.[1,2] Owing to the aggressive attack on lymph nodes outside of the lymphatic system in patients with diffuse, large B-cell lymphoma, the outlook for those patients whose condition fails to respond to initial chemotherapy cycles is poor
At the end of the trial, treatment with axicabtagene ciloleucel resulted in 0.48 more LYs and 0.34 more qualityadjusted life-years (QALYs) than chemotherapy, producing a cost-effectiveness estimate of $896 600 per QALY for public payers and $1 615 000 per QALY for commercial payers
This can be contrasted with a mean of 0.91 LYs and 0.57 QALYs gained at the end of the trial time horizon for a patient who had been treated with chemotherapy
Summary
Large B-cell lymphoma is the most common subtype of non-Hodgkin lymphoma in the United States, accounting for 30% to 40% of all non-Hodgkin lymphoma cases.[1,2] Owing to the aggressive attack on lymph nodes outside of the lymphatic system in patients with diffuse, large B-cell lymphoma, the outlook for those patients whose condition fails to respond to initial chemotherapy cycles is poor. Even if a patient’s condition responds to second-line chemotherapy and the patient completes autologous stem cell transplantation, 5-year progression-free survival is estimated to be only 10% to 20%.3-5. The recent development and approval of axicabtagene ciloleucel, a chimeric antigen receptor T-cell (CAR-T) therapy, represents a new and potentially curative option.[6] The overall response rate for treatment with axicabtagene ciloleucel in its pivotal trial was 82%, with estimates of overall survival at 6 months of 80%.7. The purpose of the present study was to estimate long-term survival gains for patients treated with axicabtagene ciloleucel using trial evidence and recommended long-term survival extrapolation techniques.[8,9,10,11] Trial-based and projected long-term survival estimates were used to calculate the cost-effectiveness of treatment with axicabtagene ciloleucel to inform the value of this novel therapy
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