CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Cost-effectiveness analysis of axicabtagene ciloleucel as a second-line treatment for diffuse large B-cell lymphoma in China and the United States.
Cost-effectiveness analysis of axicabtagene ciloleucel as a second-line treatment for diffuse large B-cell lymphoma in China and the United States.
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Axi-cel 作为中国 DLBCL 二线治疗的选择不具有成本效益。然而,在美国,Axi-cel 作为 DLBCL 二线治疗显示出成本效益优势。
阿基仑赛注射液(Axi-cel)是中国首个获批用于治疗接受过二线或以上系统性治疗后复发/难治性大B细胞淋巴瘤成人患者的CAR-T 细胞免疫疗法(CAR-T)产品。然而,由于其价格高昂,无法在临床实践中广泛应用。
为在不同经济发展阶段的国家全面评估Axi-cel的经济价值,本文从中国和美国的医疗卫生体系视角,评估了Axi-cel用于弥漫性大B细胞淋巴瘤(DLBCL)二线治疗的成本效果。
Axi-cel治疗复发/难治性大B细胞淋巴瘤(LBCL)的成本效果分析。基于ZUMA-7的临床试验数据,构建短期决策树和长期半马尔可夫分区生存模型,评估两种策略的成本效果。该模型以1个月为周期,循环40年。本文仅考虑直接医疗成本。进行单因素敏感性分析和概率敏感性分析,以评估基础案例结果的稳健性。
在基线成本效果分析中,Axi-cel 与中国标准二线化疗相比,与更多的质量调整生命年(QALY;2.72 对 1.46)和更高的总成本(180,501.55 美元对 123,221.34 美元)相关。此外,Axi-cel 组的增量成本效果比(ICER)为 45,726.66 美元/QALY,高于 37,654.5 美元的阈值。为实现成本效果,Axi-cel 的价格必须适当降低。在美国,Axi-cel 与更多的 QALY(2.63 对 1.74)和更高的总成本(415,915.16 美元对 289,564.34 美元)相关。Axi-cel 的 ICER 为 142,326.94 美元/QALY,低于设定的 150,000 美元阈值。
Axicabtagene ciloleucel (Axi-cel) is the first Chimeric Antigen Receptor T-Cell Immunotherapy (CAR-T) product approved in China for treating adult patients with relapsed or refractory large B-cell lymphoma after receiving second-line or above systemic therapy. However, it cannot be widely used in clinical practice due to its high price.
To evaluate the economic value of Axi-cel fully in countries at different stages of economic development, this article, from the perspective of the medical and health system in China and the United States, evaluated the cost-effectiveness of Axi-cel in the second-line treatment of diffuse large B-cell lymphoma (DLBCL). DESIGN: Cost effectiveness analysis of Axi-cel in the treatment of relapsed or refractory large B-cell lymphoma (LBCL).
Based on the clinical trial data of ZUMA-7, a short-term decision tree and a long-term semi-Markov partitioned survival model were constructed to evaluate the cost-effectiveness of the two strategies. This model was cycled for 40 years in 1-month cycles. In this article, only direct medical costs were considered. One-way sensitivity analysis and probabilistic sensitivity analysis were conducted to assess the robustness of the base-case results.
In the baseline cost-effectiveness analysis, Axi-cel was associated with more quality-adjusted life year (QALY; 2.72 versus 1.46) and greater costs overall ($180,501.55 versus $123,221.34) than standard second-line chemotherapy in China. Moreover, the incremental cost-effectiveness ratio (ICER) of the Axi-cel group was $45,726.66/QALY, which was greater than the threshold of $37,654.5. To achieve cost-effectiveness, the price of Axi-cel must be reduced appropriately. In the United States, Axi-cel was associated with more QALYs (2.63 versus 1.74) and greater costs overall ($415,915.16 versus $289,564.34). The ICER for the Axi-cel was $142,326.94/QALY, below the set threshold of $150,000.
Axi-cel is not a cost-effective option as second-line therapy for treating DLBCL in China. However, In the United States, Axi-cel has shown a cost-effectiveness advantage as a second-line treatment for DLBCL.
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