Elshiekh Cleopatra, Rudà Roberta, Cliff Edward R Scheffer, Gany Francesca, Budhu Joshua A
Immigrant Health and Cancer Disparities Service, Department of Psychiatry, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
Division of Neuro-Oncology, Department of Neuroscience, University and City of Health and Science Hospital, Turin, Italy.
Neurooncol Pract. 2024 Dec 3;12(Suppl 1):i49-i58. doi: 10.1093/nop/npae098. eCollection 2025 Feb.
The isocitrate dehydrogenase (IDH) inhibitor, vorasidenib, may offer a promising new treatment option for patients with IDH-mutant gliomas. However, the indefinite nature of this targeted therapy raises significant financial concerns. High costs of targeted cancer therapies, often exceeding $150 000 annually, contribute to financial toxicity, characterized by medical debt, income loss, and psychological stress, and place stress on health systems. This review analyzes the drug approval and pricing mechanisms in various countries and their impact on healthcare costs and patient access, focusing specifically on the impacts in neuro-oncology. The United States employs a market-driven approach resulting in higher drug prices, while most countries, such as the United Kingdom, Germany, France, Italy, Japan, South Africa, and Brazil, use negotiated pricing and health technology assessment to manage costs. The financial burden of expensive medications affects patient adherence and quality of life, with many cancer patients facing substantial out-of-pocket expenses and potential treatment abandonment, and many more unable to access these drugs altogether. Vorasidenib's introduction, while potentially improving patient outcomes, may exacerbate financial toxicity unless mitigated by patient access programs and cost-management strategies. As neuro-oncology treatment paradigms evolve, understanding the economic implications of new therapies is essential to ensure equitable access and optimize patient care.
异柠檬酸脱氢酶（IDH）抑制剂伏立西尼可能为IDH突变型胶质瘤患者提供一种有前景的新治疗选择。然而，这种靶向治疗的不确定性引发了重大的经济担忧。靶向癌症治疗的高昂成本，通常每年超过15万美元，导致了经济毒性，其特征包括医疗债务、收入损失和心理压力，并给卫生系统带来压力。本综述分析了各国的药物审批和定价机制及其对医疗成本和患者可及性的影响，特别关注神经肿瘤学领域的影响。美国采用市场驱动的方法，导致药品价格更高，而大多数国家，如英国、德国、法国、意大利、日本、南非和巴西，采用谈判定价和卫生技术评估来控制成本。昂贵药物的经济负担影响患者的依从性和生活质量，许多癌症患者面临大量自付费用并可能放弃治疗，还有更多患者根本无法获得这些药物。伏立西尼的推出虽然可能改善患者预后，但除非通过患者可及计划和成本管理策略加以缓解，否则可能会加剧经济毒性。随着神经肿瘤学治疗模式的演变，了解新疗法的经济影响对于确保公平可及性和优化患者护理至关重要。
