A literature review of the Costs and Cost-Effectiveness of CAR T-cell therapy in cancer treatment from 2014 to 2024

Abstract

Background: Cancer imposes a substantial and growing global health and economic burden, particularly in low- and middle-income countries. In recent years, chimeric antigen receptor (CAR) T-cell therapy has emerged as a highly innovative immunotherapy with remarkable clinical efficacy in several hematological malignancies. However, the extremely high costs of CAR T-cell therapy raise significant concerns regarding its affordability and cost-effectiveness for healthcare systems. Despite the increasing number of economic evaluations, evidence remains fragmented and has not been comprehensively synthesized.

Objectives: This study aimed (1) to systematically review the published evidence on the costs and cost-effectiveness of CAR T-cell therapy in cancer treatment from 2014 to 2024, and (2) to evaluate the reporting quality of these economic evaluations using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist.

Methods: A literature review was conducted using three major biomedical databases: PubMed, Web of Science, and The Cochrane Library. Studies published in English between 2014 and July 2024 were eligible if they reported cost or cost-effectiveness analyses of CAR T-cell therapy compared with conventional treatments. Data extraction focused on study characteristics, cost components, economic evaluation methods, incremental cost-effectiveness ratios (ICERs), and willingness-to-pay thresholds. The reporting quality of included studies was independently assessed using the CHEERS 2022 checklist.

Results: After screening and eligibility assessment, 25 studies met the inclusion criteria. Most studies were conducted in high-income countries and commonly adopted a healthcare system or payer perspective, using model-based economic evaluations such as Markov models. CAR T-cell therapy was consistently associated with substantially higher upfront costs compared to standard of care, but also demonstrated meaningful gains in life-years and quality-adjusted life years (QALYs). The reported ICERs varied widely across cancer types, countries, and comparator interventions, with some studies suggesting that CAR T-cell therapy may be cost-effective under specific willingness-to-pay thresholds. Quality assessment showed high compliance with CHEERS domains related to title, abstract, introduction, results, and discussion, while lower compliance was observed in the methods domain and reporting of uncertainty and stakeholder engagement.

Conclusions: CAR T-cell therapy offers significant clinical benefits but poses major economic challenges due to its high costs. Evidence on cost-effectiveness varies substantially by context, highlighting the importance of country-specific economic evaluations. Overall reporting quality was moderate to high, though important methodological details were frequently underreported. Future studies should improve transparency and methodological rigor to better inform policy decisions regarding the adoption and reimbursement of CAR T-cell therapy.