In December 2023, the US Food and Drug Administration and the UK Medicines and Healthcare Products Regulatory Agency granted the first regulatory approval for genome therapy for sickle cell disease. This approval brings hope to those suffering from this debilitating genetic disease. However, several barriers may hinder global patient access, including high treatment costs, obtaining informed consent for minors, inadequate public health infrastructure, and insufficient regulatory oversight. These barriers reflect the structural inequalities inherent in global health governance, where patient access often depends on social and institutional arrangements. This article addresses concerns around informed consent, treatment costs, and patient access, and proposes corresponding policy reforms. We argue that these discussions should be framed within a broader global context that considers social and institutional structures, global research priorities, and a commitment to health equity.

中文翻译：

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要在全球获得基因组治疗方面实现公平，扩大获得潜在变革性疗法的机会并造福那些有需要的人，需要改变全球政策

2023年12月，美国食品和药物管理局以及英国药品和保健品监管局首次批准镰状细胞病基因组疗法。这一批准给那些患有这种使人衰弱的遗传病的人带来了希望。然而，一些障碍可能阻碍全球患者获得治疗，包括高昂的治疗费用、未成年人的知情同意、公共卫生基础设施不足以及监管不足。这些障碍反映了全球卫生治理中固有的结构性不平等，患者的获取往往取决于社会和制度安排。本文讨论了有关知情同意、治疗费用和患者准入的担忧，并提出了相应的政策改革建议。我们认为，这些讨论应该在更广泛的全球背景下进行，考虑社会和制度结构、全球研究重点以及对健康公平的承诺。