商业化 CAR-T 细胞疗法的全球可及性：G20 国家卫生技术评估的横断面研究

Chimeric antigen receptor (CAR) T cells are a major treatment advance for many patients with hematological malignancies, especially those with disease that has relapsed or is refractory to chemotherapy.

However, currently approved commercial autologous CAR T cells require highly specialized manufacturing processes that contribute to high costs and limit their widespread use. In many countries, positive reimbursement recommendations by health technology assessment (HTA) bodies enable patient access to CAR T therapies.

We performed a cross-sectional analysis of HTA evaluations of commercial CAR T therapies among the Group of 20 (G20) member countries plus 3 G20 invitees (Spain, Singapore, and Switzerland) through 1 August 2025. Across the 18 CAR T product-indication pairs with current US Food and Drug Administration (FDA) approval, we analyzed HTA review documentation to ascertain the timing and rationale for a positive or negative recommendation.

Fourteen countries with public HTA data were included in our analysis. Forty-eight percent of CAR T-indication pairs (122/252) are currently recommended for reimbursement by public health systems. The median time from FDA approval to HTA decision was 1. 54 years (interquartile range, 1. 15-2. 59). Common barriers to CAR T cost-effectiveness cited in HTA reports included single-arm trial designs, small study populations, and immature data regarding survival, safety, and quality of life.

Our findings demonstrate substantial global disparities in access to CAR T treatments even among high-income and upper middle-income countries, highlighting the urgent need for both scientific and policy approaches to reduce costs and improve access to these impactful therapies.