The cost-effectiveness of axicabtagene ciloleucel as second-line therapy in patients with large B-cell lymphoma in the United States: An economic evaluation of the ZUMA-7 trial Journal Article


Authors: Perales, M. A.; Kuruvilla, J.; Snider, J. T.; Vadgama, S.; Blissett, R.; El-Moustaid, F.; Smith, N. J.; Patel, A. R.; Johnston, P. B.
Article Title: The cost-effectiveness of axicabtagene ciloleucel as second-line therapy in patients with large B-cell lymphoma in the United States: An economic evaluation of the ZUMA-7 trial
Abstract: Axicabtagene ciloleucel (axi-cel) was found to have superior clinical outcomes compared to standard of care (SOC; salvage chemoimmunotherapy, followed by high-dose therapy with autologous stem cell rescue for responders) for second-line large B-cell lymphoma (2L LBCL) in the pivotal ZUMA-7 trial. The aim of this analysis was to evaluate the cost effectiveness of using axi-cel compared to the current standard 2L LBCL therapy. A 3-state partitioned-survival model estimated the cost effectiveness and budget impact from a payer perspective in the United States. Clinical outcomes were extrapolated based on the pivotal trial. The model calculated expected quality-adjusted life years (QALYs), total costs (in United States dollars [USD], and the incremental cost-effectiveness ratio (ICER), along with the budget impact. Sensitivity and scenario analyses were performed. The proportion alive at 10 years was estimated as 48% for axi-cel and 38% for SOC; median overall survival was estimated at 59 and 24 months for axi-cel and SOC, respectively. Over a lifetime horizon, the model estimated a total of 5.56 and 7.08 QALYs for SOC and axi-cel, respectively, of which 41% and 74% were in the event-free state, respectively. Incremental QALYs and costs were 1.51 and $100,366 USD, resulting in an ICER of $66,381 USD per QALY for axi-cel versus SOC. Despite crossover to subsequent CAR T in the SOC arm, second-line CAR T use was found to improve the quality and length of life compared to SOC. Cost offsets due to subsequent CAR T use led to a limited incremental cost difference. Treatment with axi-cel is a cost-effective option that addresses an important unmet clinical need for patients with LBCL who relapse or are refractory to front-line therapy. © 2022 The American Society for Transplantation and Cellular Therapy
Keywords: cancer survival; clinical article; controlled study; event free survival; overall survival; allogeneic stem cell transplantation; cisplatin; cancer combination chemotherapy; united states; gemcitabine; cytarabine; rituximab; drug megadose; antineoplastic agent; sensitivity analysis; carboplatin; neoplasm recurrence, local; etoposide; dexamethasone; autologous stem cell transplantation; ifosfamide; cost effectiveness analysis; drug cost; health care utilization; health economics; tumor recurrence; large cell lymphoma; lymphoma, large b-cell, diffuse; corticosteroid; cost-effectiveness analysis; adverse drug reaction; quality adjusted life year; cost-benefit analysis; cd19 antigen; antigens, cd19; cost benefit analysis; budget; clinical trial (topic); clinical outcome; diffuse large b cell lymphoma; palliative chemotherapy; large b-cell lymphoma; humans; human; article; median survival time; tisagenlecleucel t; chimeric antigen receptor t-cell immunotherapy; axicabtagene ciloleucel; lisocabtagene maraleucel; receptors, chimeric antigen; chimeric antigen t-cell therapy
Journal Title: Transplantation and Cellular Therapy
Volume: 28
Issue: 11
ISSN: 2666-6375
Publisher: Elsevier Inc.  
Date Published: 2022-11-01
Start Page: 750.e1
End Page: 750.e6
Language: English
DOI: 10.1016/j.jtct.2022.08.010
PUBMED: 35970302
PROVIDER: scopus
PMCID: PMC10314822
DOI/URL:
Notes: Article -- Export Date: 1 December 2022 -- Source: Scopus
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  1. Miguel-Angel Perales
    918 Perales