Cost-effectiveness of axicabtagene ciloleucel versus tisagenlecleucel for the treatment of 3L + relapsed/refractory large B-cell lymphoma in the United States: incorporating longer survival results

Abstract

Aims

To provide an update on the cost-effectiveness of the chimeric antigen receptor (CAR) T-cell therapies axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) for the treatment of relapsed/refractory (r/r) large B-cell lymphoma (LBCL) among patients who have previously received ≥2 lines of systemic therapy using more mature clinical trial data cuts (60 months for axi-cel overall survival [OS] and 45 months for tisa-cel OS and progression-free survival [PFS]).

Methods

A partitioned survival model consisting of three health states (pre-progression, post-progression and death) was used to estimate quality-adjusted life years (QALYs) and costs associated with axi-cel and tisa-cel over a lifetime horizon. PFS and OS inputs for axi-cel and tisa-cel were based on a previously published matching-adjusted indirect treatment comparison (MAIC). Long-term OS and PFS were extrapolated using parametric survival mixture cure models (PS-MCMs). Costs of CAR-T cell therapy drug acquisition and administration, conditioning chemotherapy, apheresis, CAR T-specific monitoring, stem cell transplant, hospitalization, adverse events, routine care, and terminal care were sourced from US cost databases. Health state utilities were derived from previous publications. Model inputs were varied using a range of sensitivity and scenario analyses.

Results

Compared with tisa-cel, axi-cel resulted in 2.51 additional QALYs and $50,185 additional costs (an incremental cost-effectiveness ratio [ICER] of $19,994 per QALY gained). In probabilistic sensitivity analysis (PSA), the ICER for axi-cel versus tisa-cel was ≤$50,000/QALY in 99.4% of simulations and ≤$33,500 in 99% of simulations. Axi-cel remained cost-effective versus tisa-cel (assuming a willingness-to-pay threshold of $150,000 per QALY) across a range of scenarios.

Conclusions

With longer-term survival data, axi-cel continues to represent a cost-effective option versus tisa-cel for treatment of r/r LBCL among patients who have previously received ≥2 lines of systemic therapy, from a US payer perspective.

Keywords:

• Cost-effectiveness
• large B-cell lymphoma
• anti-CD19
• chimeric antigen receptor, T-cell therapy
• axicabtagene ciloleucel (axi-cel)
• tisagenlecleucel (tisa-cel)
• matching-adjusted indirect comparison
• iCER

JEL Classification Codes:

• C01
• C
• O51
• O5
• O
• I10
• I1
• I

Transparency

Declaration of funding

Funding for this study was provided by Kite Pharma, a Gilead Company.

Declaration of financial/other relationships

OOO has undertaken consultancy and advisory boards for: Pfizer, Kite, Gilead, AbbVie, Janssen, TGR therapeutics, ADC, Novartis, Epizyme, Curio science, Nektar, Cargo, Caribou and has received institution funding from: Kite, Pfizer, Daichi Sankyo, Allogene and honoraria from: Pfizer, Gilead. MDR is an employee of Kite Pharma, who funded the study. ND, RB and CJ are employees of Mtech Access, who have received consultancy fees for the development of the economic model and manuscript. FLL performed a consulting/advisory role for Allogene, Amgen, Bluebird Bio, Bristol-Myers Squibb, Calibr, Cellular Biomedicine Group, Cowen, ecoR1, Emerging Therapy Solutions Gerson Lehman Group, GammaDelta Therapeutics, Iovance, Janssen, Kite, a Gilead Company, Legend Biotech, Novartis, Umoja, and Wugen; received research funding from Allogene, Kite, and Novartis; and holds patents, royalties, other intellectual property from several patents held by the institution in author’s name (unlicensed) in the field of cellular immunotherapy.

Acknowledgements

We thank Rongzhe Liu, Ibrahim Diakite and Marc F. Botteman (Pharmerit) who developed the original cost-effectiveness model, Julia Snider, Madhu Palivela and Jeff Shafiroff (Kite Pharma) who provided input on the analysis design and Helen Smethurst (Mtech Access) for medical writing services in preparation of the manuscript, all funded by consultancy payments from Kite Pharma.

Reviewer disclosures

Peer reviewers on this manuscript have received an honorarium from JME for their review work but have no other relevant financial relationships to disclose.

Previous presentations

Results of an earlier version of the model were presented at the European Hematology Association (EHA) 2023 congress.

Results of a previous version of this model using an earlier clinical data cut have previously been published in Journal of Medical Economics (Rongzhe Liu, et al. (2021) Journal of Medical Economics, 24:1, 458-468, DOI: 10.1080/13696998.2021.1901721).