不适合移植的大 B 细胞淋巴瘤二线使用 axicabtagene ciloleucel：ALYCANTE 最终分析
Second-line axicabtagene ciloleucel in large B-cell lymphoma ineligible for transplantation: ALYCANTE final analysis.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
罕见病先进治疗药品的药物经济学特征：一项系统综述
英文原题：Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review.
查看英文原题
Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review.
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中文摘要
背景与目的
先进治疗药品（ATMPs）是基于基因、组织或细胞的创新药物，针对罕见和严重疾病。ATMPs已显示出有前景的临床结局，但与高成本相关，引发了关于成本效益的问题。因此，本系统综述旨在分析欧洲药品管理局授权的用于治疗罕见疾病的ATMPs的成本效益和成本效用概况。
方法
按照PRISMA指南进行了系统综述。通过检索PubMed、Embase、Web of Science和ProQuest科学数据库识别研究。纳入报告ATMPs增量成本效益/效用比（ICERs/ICURs）的经济学评价。成本标准化为2023年欧元，并构建成本效益平面，以根据每QALY 50,000欧元、100,000欧元和150,000欧元的支付意愿（WTP）阈值评估结果，作为敏感性分析的一部分。
结果
共有61项研究符合纳入标准。用于罕见血液疾病的ATMPs，如tisagenlecleucel和axicabtagene ciloleucel，在大多数研究中被发现具有成本效益，终生时间范围内每例患者的增量QALYs范围为1.5至10。Tisagenlecleucel在治疗急性淋巴细胞白血病中显示出积极的成本效益概况（58%），而axicabtagene ciloleucel在治疗弥漫性大B细胞淋巴瘤中显示出积极概况（85%）。用于脊髓性肌萎缩症（SMA）的onasemnogene abeparvovec显示出不确定的成本效益结果，而用于视网膜疾病的voretigene neparvovec在40%的研究中不具有成本效益，增量QALYs约为1.3，且高成本超过了设定的WTP阈值。
结论
ATMPs 在治疗罕见病方面显示出有前景的经济潜力，但成本效果因适应症而异。政策制定者必须在创新与体系可持续性之间取得平衡，采用更精细的模型并考虑对患者结局的长期影响。
展开英文摘要原文
Background and aim : Advanced Therapy Medicinal Products (ATMPs) are innovative drugs based on genes, tissues, or cells that target rare and severe diseases. ATMPs have shown promising clinical outcomes but are associated with high costs, raising questions about cost-effectiveness. Hence, this systematic review aims to analyze the cost-effectiveness and cost-utility profiles of the European Medicines Agency-authorized ATMPs for treating rare diseases. Methods : A systematic review was conducted following PRISMA guidelines. Studies were identified by searching PubMed, Embase, Web of Science, and ProQuest scientific databases. Economic evaluations reporting incremental cost-effectiveness/utility ratios (ICERs/ICURs) for ATMPs were included. Costs were standardized to 2023 Euros, and a cost-effectiveness plane was constructed to evaluate the results against willingness-to-pay (WTP) thresholds of EUR 50,000, EUR 100,000, and EUR 150,000 per QALY, as part of a sensitivity analysis. Results : A total of 61 studies met the inclusion criteria.
ATMPs for rare blood diseases, such as tisagenlecleucel and axicabtagene ciloleucel, were found to be cost-effective in a majority of studies, with incremental QALYs ranging from 1. 5 to 10 per patient over lifetime horizon. Tisagenlecleucel demonstrated a positive cost-effectiveness profile in the treatment of acute lymphoblastic leukemia (58%), while axicabtagene ciloleucel showed a positive profile in the treatment of diffuse large B-cell lymphoma (85%).
Onasemnogene abeparvovec for spinal muscular atrophy (SMA) showed uncertain cost-effectiveness results, and voretigene neparvovec for retinal diseases was not cost-effective in 40% of studies, with incremental QALYs around 1. 3 and high costs exceeding the WTP threshold set. Conclusions : ATMPs in treating rare diseases show promising economic potential, but cost-effectiveness varies across indications. Policymakers must balance innovation with system sustainability, using refined models and the long-term impact on patient outcomes.
论文信息
- 作者
- Serino M、Krstin M、Mucherino S、Menditto E、Orlando V
- 单位
- Department of Pharmacy, University of Naples Federico II, 80131 Naples, Italy.Italy
- 文献类型
- 综述
- 期刊
- Healthcare (Basel, Switzerland)2025 Aug 2
- 原文标识
- PubMed 40805927 · DOI 10.3390/healthcare13151894
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