Cost-effectiveness of axicabtagene ciloleucel for adult patients with relapsed or refractory large B-cell lymphoma in the United States

Abstract

Purpose: Axicabtagene ciloleucel (axi-cel) was recently approved for treatment of relapsed or refractory (R/R) large B-cell lymphoma (LBCL) following two or more prior therapies. As the first CAR T-cell therapy available for adults in the US, there are important questions about clinical and economic value. The objective of this study was to assess the cost-effectiveness of axi-cel compared to salvage chemotherapy using a decision model and a US payer perspective.

Materials and methods: A decision model was developed to estimate life years (LYs), quality-adjusted life years (QALYs), and lifetime cost for adult patients with R/R LBCL treated with axi-cel vs salvage chemotherapy (R-DHAP). Patient-level analyses of the ZUMA-1 and SCHOLAR-1 studies were used to inform the model and to estimate the proportion achieving long-term survival. Drug and procedure costs were derived from US average sales prices and Medicare reimbursement schedules. Future healthcare costs in long-term remission was derived from per capita Medicare spending. Utility values were derived from patient-level data from ZUMA-1 and external literature. One-way and probabilistic sensitivity analyses evaluated uncertainty. Outcomes were calculated over a lifetime horizon and were discounted at 3% per year.

Results: In the base case, LYs, QALYs, and lifetime costs were 9.5, 7.7, and $552,921 for axi-cel vs 2.6, 1.1, and $172,737 for salvage chemotherapy, respectively. The axi-cel cost per QALY gained was $58,146. Cost-effectiveness was most sensitive to the fraction achieving long-term remission, discount rate, and axi-cel price. The likelihood that axi-cel is cost-effective was 95% at a willingness to pay of $100,000 per QALY.

Conclusion: Axi-cel is a potentially cost-effective alternative to salvage chemotherapy for adults with R/R LBCL. Long-term follow-up is necessary to reduce uncertainties about health outcomes.

Keywords:

• Axicabtagene ciloleucel
• CAR T-cell therapy
• cost-effectiveness
• lymphoma

JEL Classification:

• C44
• C53

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Declaration of funding

This study was funded by Kite, a Gilead Company.

Declaration of financial/other relationships

JAR, SDS, AB, and SDR are consultants for Kite, a Gilead Company. VWL, AGP, LN, and PC are employees of Kite, a Gilead Company. Peer reviewers on this manuscript have received an honorarium from JME for their review work. One reviewer discloses their role as an investigator for the Canadian Cancer Trials Group, which receives research funding from Kite Pharma Inc., Gilead, Novartis, Janssen, Roche, AbbVie, Takeda, Amgen, Seattle Genetics, and Pfizer. The remaining reviewers have no other relevant financial relationships to disclose.

Acknowledgements

None reported.