医疗援助计划的新模式只有在起作用时才支付镰状细胞基因疗法的费用,涉及主要药物制造者和34个管辖区。 Medicaid's new model pays for sickle cell gene therapies only if they work, involving major drugmakers and 34 jurisdictions.

医疗援助计划为治疗镰状细胞疾病的基因疗法推出了一个新的基于结果的付款模式,只有治疗成功的国家才支付治疗费用。 Medicaid has launched a new outcome-based payment model for gene therapies treating sickle cell disease, where states only pay if treatments succeed. 该倡议从拜登开始,在特朗普继续推行,涉及Vertex制药公司和青鸟生物公司,如果治疗无法取得成果,它们将提供折扣或折扣。 The initiative, begun under Biden and continued under Trump, involves Vertex Pharmaceuticals and Bluebird Bio, who will provide discounts or rebates if therapies fail to deliver results. 昂贵的治疗费用在每名病人220万至310万美元之间,目的是治愈影响约10万主要是黑人美国人的遗传疾病。 The high-cost treatments, priced between $2.2 million and $3.1 million per patient, aim to cure a genetic disorder affecting about 100,000 primarily Black Americans. 华盛顿特区和波多黎各等33个州也加入了进来,在改善出入便利的同时,设法控制成本。 Thirty-three states, Washington, D.C., and Puerto Rico have joined, seeking to control costs while improving access. CMS没有披露财务条款,引用保密性。 CMS has not disclosed financial terms, citing confidentiality. 诸如Serenity Cole等患者的早期结果显示,尽管长期成效仍在评估之中,但疼痛减轻,生活质量提高。 Early results from patients like Serenity Cole show reduced pain and better quality of life, though long-term effectiveness remains under evaluation. 该模式可为未来的高成本基因和细胞疗法开创先例。 The model could set a precedent for future high-cost gene and cell therapies.