In recent years, the chimeric antigen receptor T-cell (CAR-T) therapy Axicabtagene Ciloleucel has attracted sustained attention in the field of hematologic oncology. As the first CAR-T product approved in China, its indications have been expanded to the second-line treatment of relapsed or refractory large B-cell lymphoma (R/R LBCL). While delivering significantly improved response rates and survival benefits, its high single-infusion cost has placed "value for money" at the center of ongoing healthcare debates.

Recently, a research team headed by Professor Zhang Luying from the School of Public Health at Fudan University completed a rigorous cost-effectiveness analysis from the perspective of the Chinese healthcare system, providing quantitative insights via long-term model simulations. These findings were presented as an oral report at the 2026 Annual Meeting of the Health Technology Assessment international (HTAi 2026).

Clinical Evidence: From Deep Remission to Curative Hope

The efficacy data underlying the cost-effectiveness model were predominantly derived from the ZUMA-7 study.

The ZUMA-7 study is a large international, multicenter, randomized controlled Phase III trial that enrolled 359 patients with early relapsed or primary refractory LBCL following first-line treatment. The results demonstrated that compared to standard-of-care (high-dose immunochemotherapy combined with autologous hematopoietic stem cell transplantation [AHSCT]), Axicabtagene Ciloleucel significantly improved survival: 65% of patients achieved a complete response (CR), and the 4-year overall survival (OS) reached 54.6%, solidifying the therapy as a preferred option for this population[1,2]. The study data provided robust efficacy and safety parameters for the economic model.

Cost-Effectiveness Analysis: Incremental Costs in Exchange for Tangible Health Benefits

Adopting a healthcare system perspective, the study constructed a Partitioned Survival Model with a simulated time horizon of up to 30 years, in order to fully capture the long-term health benefits and potential cost sequelae. The analytical strategy compared Axicabtagene Ciloleucel with conventional therapies that rely on immunochemotherapy combined with AHSCT.

The results showed that patients in the Axicabtagene Ciloleucel group gained an average of 3.87 more life-years compared to the conventional treatment group, paired with a higher health-related quality of life during survival. The difference in health outcomes between the two groups was comprehensively evaluated using quality-adjusted life-years (QALYs): Axicabtagene Ciloleucel yielded an additional 3.51 QALYs compared to conventional treatment.

On the cost side, while the Axicabtagene Ciloleucel group generated higher upfront expenses due to the one-time infusion treatment, its subsequent cumulative costs stemming from relapse re-treatments and repeated hospitalizations were substantially lower than those of the conventional treatment group. The total incremental cost was approximately RMB 900,000. The key indicator—the incremental cost-effectiveness ratio (ICER)—was RMB 258,000/QALY, meaning an additional expenditure of RMB 258,000 was required for each extra QALY gained.

Value Judgment: A Rational Choice Below the Threshold

In pharmacoeconomics, three times the per capita gross domestic product (GDP) is often used as a benchmark to determine whether an intervention is cost-effective. According to the latest statistics, three times China's per capita GDP is approximately RMB 287,000. The ICER (RMB 258,000/QALY) of Axicabtagene Ciloleucel is lower than this threshold, indicating that this therapy is a cost-effective strategy within the Chinese healthcare system.

Furthermore, an ICER of RMB 258,000/QALY is not only below the 3x per capita GDP standard, but also closely aligns with the implicit willingness-to-pay (WTP) threshold of several economically developed coastal provinces. This underscores that allocating limited societal medical resources to this therapy can generate significant incremental health returns—resulting in fewer subsequent recurrent hospitalizations, enhanced curative prospects, and a prolonged period of quality-adjusted survival. This represents an optimal allocation of resources where they matter most.

Expert Perspective: Early and Optimal Utilization—From Therapeutic Breakthrough to Value Integration

Professor Zhang Luying remarked: "The value of a high-priced oncological therapy is not determined solely by its upfront acquisition cost. Instead, it must be evaluated comprehensively based on whether it maximizes health utility within finite societal resources. Although Axicabtagene Ciloleucel incurs higher direct costs during the treatment period, its outstanding curative potential brings a prolonged healthy lifespan and better quality of life, while significantly reducing subsequent medical consumption caused by repeated progression or relapse. Crucially, the earlier the line of therapy in which it is administered, the longer the accumulated healthy life expectancy and the better its economic efficiency. Delays in appropriate treatment trigger complications and high-intensity subsequent interventions, which ultimately impose a far greater financial burden. From a health technology assessment perspective, this stands as a high-value choice that benefits patients, families, and the medical insurance system alike."

Conclusion

The clinical introduction of Axicabtagene Ciloleucel extends far beyond climbing response numbers; it provides a panoramic lens through which to examine the dynamics between 'cost' and 'value' in oncology. Rigorous cost-effectiveness analysis establishes that a one-time investment in cellular immunotherapy translates into years of high-quality survival benefits and long-term savings for healthcare resources. A premium drug is not necessarily 'unjustifiably expensive'; evidence-based value assessments are paving a clear, justifiable path for worthwhile innovative therapies to enter clinical practice and reach the patients who need them most.

Expert Introduction

Professor Luying Zhang

School of Public Health, Fudan University

Professor Luying Zhang is a Professor and Doctoral Supervisor at the School of Public Health, Fudan University. She serves as the Deputy Director of the Research and Evaluation Center for Pharmacoeconomics at Fudan University and has been recognized as a Shanghai Pujiang Talent. Her distinguished professional leadership roles include: Vice President of the Healthcare Security Branch of the China Association of Medical Equipment; Vice Chair of the Institutional Management and Evaluation Special Committee of the Shanghai Community Health Association; Standing Committee Member of the Pharmacoeconomics Special Committee of the China Health Insurance Research Association; Board Director of the Shanghai Health Economics Association; Committee Member of the Healthcare Security Professional Committee of the China Association of Social Security; Committee Member of the Medical Insurance Professional Committee, Health Technology Assessment Professional Committee, and Youth Committee of the China Health Economics Association; and Editorial Board Member or Youth Editorial Board Member of the journals China Health Economics, Health Economics Research, and World Clinical Drugs. Her main research domains encompass medical insurance, health economics, policy evaluation, and community health services. She has served as the Principal Investigator for numerous high-level research initiatives funded by the National Science and Technology Major Project, the National Natural Science Foundation of China (NSFC), Major Projects of the National Social Science Fund of China (Sub-projects), the Shanghai Social Science Fund, Key Special Government Decision-Making Consultation Projects of Shanghai, the National Health Commission, and the National Healthcare Security Research Institute. Over the past five years, as first or corresponding author, she has published more than 60 papers in leading Chinese and international peer-reviewed journals, including BMJ, BMJ Global Health, and BMC Medicine. Additionally, she has authored 3 personal monographs and co-authored 4 national planning textbooks, including Health Economics and Medical Security Studies.

Note: The Renminbi (RMB) cost data cited herein have been converted based on the exchange rates utilized within the study. The original academic data were reported in US Dollars (USD), and actual monetary values may fluctuate alongside exchange rate variations.

Literature References:
1. Locke FL, Miklos DB, Jacobson CA, et al. Axicabtagene Ciloleucel as Second-Line Therapy for Large B-Cell Lymphoma. N Engl J Med. 2022;386(7):640-654.
2. Westin JR, Oluwole OO, Kersten MJ, et al. Survival with Axicabtagene Ciloleucel in Large B-Cell Lymphoma. N Engl J Med. 2023;389(2):148-157.
 
[Disclaimer] This article is intended solely for medical academic exchange and professional knowledge sharing. It does not constitute clinical diagnostic or therapeutic evidence, nor does it serve as commercial promotion or clinical guidance for any drug or medical device. The content herein has no direct or indirect conflict of interest with the relevant enterprises mentioned. All insights are based on the expert's personal clinical practice and publicly available academic research, maintaining an objective and neutral stance completely free of commercial bias. The CCMTV platform serves exclusively as a medium for academic communication and disclaims any medical or legal liability arising from the clinical application of this content. Readers must strictly adhere to national clinical guidelines, laws, and regulations prior to making any clinical decisions.