由 jpichiban Tue, 18 Feb 2025 14:52:30 GMT
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从十几岁开始，德肖恩·“DJ”·周（Deshawn “DJ” Chow）就不确定自己能否过上正常生活。镰状细胞病引发的剧烈疼痛发作日益严重。这位19岁的年轻人说：“因为总是缺课和频繁进出医院，生活变得非常艰难。而且……我的头部和腰部下方会感到剧痛。”
一年多前，当美国食品药品监督管理局（FDA）批准了新的镰状细胞基因疗法后，周的养父母联系了洛杉矶的希望之城儿童癌症中心（City of Hope Children’s Cancer Center），希望他能接受这种新疗法。令他们欣慰的是，中心接纳了他作为病人，并迅速从周家的雇主赞助保险中获得了授权。
“他们几乎承担了所有费用，我们几乎不用自掏腰包。所以，我们非常感激这些福利，”DJ的父亲肖恩·周（Sean Chow）说，“我感到很惊讶。”
DJ周是医院少数接受Casgevy治疗的患者之一，这种由Vertex制药公司生产的镰状细胞基因疗法，每位患者的费用超过200万美元。过去一年中，治疗过程包括多次住院和化疗，产生了额外费用。
镰状细胞病是一种血液疾病，患者的红细胞会变形为新月形。该病对黑人的影响尤为严重，常导致剧烈疼痛发作，使患者频繁住院。
周是少数完成新基因疗法治疗的患者之一。今年1月完成Casgevy全部疗程后，他开始梦想做那些一直想做的事情。
“学习滑雪、冲浪，做所有这些事情……因为镰状细胞病，我从未真正体验过这些经历，”他说。
尽管美国有超过10万人患有镰状细胞病，但器官未受疾病损害的年轻患者是最有希望从新疗法中受益的群体。然而，大规模治疗患者的能力提升缓慢。自FDA批准两种镰状细胞基因疗法以来的一年内，仅有100多名患者接受了治疗。
Vertex高管在公司第四季度财报电话会议上表示，截至2023年底，全球已有50名患者接受了首次细胞采集。与此同时，竞争对手Bluebird Bio的高管去年秋天表示，近60名患者接受了其药物Lyfgenia的治疗，该药物每位患者的费用超过300万美元。另有37名患者计划在2025年初开始接受Bluebird的治疗。
对于首批提供新镰状细胞基因疗法的治疗中心来说，与保险公司协调覆盖范围需要一定的学习曲线。
“现在比我们刚开始接收患者时顺畅多了，”华盛顿特区国家儿童医院（Children’s National Hospital）患者准入执行主任詹妮弗·卡梅伦（Jennifer Cameron）说，“很多时候，我们会向他们发送由制造商开发的计费和编码指南，并与支付方分享，如果他们不了解的话。”
希望之城的儿科血液肿瘤学家王利奥（Leo Wang）博士也与周合作，他表示这一过程已经变得更加顺畅，但他担心这些治疗的价格仍会带来覆盖障碍。
“医疗系统面临的挑战是巨大的。这是一种非常昂贵的疗法，”他说，“对于基于雇主的保险计划来说，可能有点难以承担这些费用。”
CVS Health（健康保险公司Aetna的母公司）的首席执行官大卫·乔伊纳（David Joyner）表示，到目前为止，治疗患者的缓慢增长使得早期病例的覆盖管理变得可行。但随着需求的预期增长，情况可能会发生变化。 ramp up, he said many in the industry are looking at developing new payment models for the sickle cell treatments and other gene therapies on the horizon. “There are emerging risk pools being developed … sometimes at the state Medicaid levels, and sometimes collectively across larger payers,” Joyner explained, so that the financial burden of the treatments is spread beyond just one state or one company.”You have to think about a different payment model, because today’s payment model is not constructed to spread the cost,” he said. “But that takes time.”A challenge for MedicaidFor state Medicaid programs, the challenge of affordability for the new sickle cell treatments may be even greater. More than half of sickle cell patients are covered under the federal-state government health plan for low-income Americans.Southern states like Georgia, Florida and Mississippi have some the largest concentrations of sickle cell patients, according to a study by researchers at the University of Chicago.The Biden administration developed a Cell and Gene Therapy payment model under the Centers for Medicare and Medicaid, which will provide states with an outcomes-based discounted price and provide some funding for the new drugs. The deadline for states to apply for the program is Feb. 28, with the first federal grants to help pay for the drugs on track to begin in June, according to CMS officials. Under the new payment model, states could receive up to $9.5 million in federal funding, but even with discounted prices that may not begin to cover the costs of treating Medicaid patients in some cases.Researchers at Oregon Health & Science University calculated that the 10 states with the largest sickle cell populations could see a mean budget impact of $30 million, based on an estimate of treatments priced at just under $1.9 million. Those increased costs would come at a time when the Trump administration and the Republican-controlled Congress are looking for ways to cut federal spending. The administration has already begun to cut staffing at health agencies, and federal funding for state Medicaid programs is expected to be on the table in upcoming budget proposals.Health and Human Services Secretary Robert F. Kennedy Jr. said during his confirmation hearings last month that he is committed to maintaining staffing to provide coordination of sickle cell coverage across the department and other agencies, without specifically discussing funding for the new gene therapies or Medicaid overall.”I have many friends who have sickle cell. I’ve seen the suffering they endure,” Kennedy said. “There are now promising gene therapies. They are very, very expensive, but it’s something that [National Institutes of Health] should be enthusiastically supporting — that kind of research.”Sean Chow said he is grateful to the researchers who developed the gene therapy, which he hopes will allow his son to have a more normal future, without debilitating episodes of pain. He wants other families to have the same opportunity to access the high-priced gene therapies for their loved ones. “Having a child with sickle cell has been heartbreaking,” he said. “I’m hoping as more and more patients get the therapy, the cost can be driven down.”
原文链接：https://www.cnbc.com/2025/02/18/sickle-cell-gene-therapies-push-health-plans-toward-new-payment-models.html