国际细胞与基因治疗学会扩展可及性工作组立场文件：支持研究性细胞和基因干预公平伦理扩展可及的关键考量
International Society for Cell & Gene Therapy Expanded Access Working Group position paper: key considerations to support equitable and ethical expanded access to investigational cell- and gene-based interventions
许多细胞和基因疗法是“量身定制”的，用于治疗严重或无法治愈、有时可治愈的疾病，患者往往无法等到正式获批。本文由国际细胞与基因治疗学会扩展可及性工作组撰写，审视在临床试验之外为无获批替代方案的患者提供研究性疗法的路径。文件讨论患者透明沟通、数据报告、成本回收模式及其对长期商业化的影响，并强调公平与包容，力求在可及性和监管之间取得平衡。
为什么推荐给您：针对细胞与基因疗法扩展可及路径的学会立场文件，提出伦理与操作性框架，属值得关注。
不需要生物学背景，多打比方
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细胞和基因治疗领域发展很快，医生们越来越难在让患者用上药和加强监管之间找平衡。
第一步：有些患者得了严重甚至要命的病，而且没有已经批准的药可用。
患者患有严重或危及生命的疾病，没有已批准的治疗办法，只能考虑研究性疗法，同时要承担治疗风险。
摘要Abstract
This position paper reviews the Expanded Access pathway for cell and gene therapies, examining its critical role at the nexus of patient need, regulatory frameworks, and scientific advancement. Spearheaded by the International Society for Cell & Gene Therapy's Expanded Access Working Group, it explores how investigational therapies are accessed outside of clinical trials for patients with serious or life-threatening conditions when no approved alternatives exist. Access to cell and gene therapy products are of specific interest to patients because many times the products are bespoke, being used to treat serious and/or incurable conditions, and are potentially curative. As the field of cell and gene therapy rapidly progresses, healthcare professionals face mounting challenges in navigating the balance between access and oversight. Key considerations include transparent communication with patients, robust data reporting, and a discussion of cost recovery models and their implications for long-term commercialization strategies. Equity and inclusivity are central themes, highlighting the need to design pathways that are accessible to diverse patient populations while upholding high scientific and ethical standards. This position paper is presented as a resource for clinicians, researchers, and policymakers navigating the evolving landscape of investigational cell and gene therapies. It emphasizes the importance of ethical frameworks and equitable practices in delivering transformative treatments to patients in need.