三月21 ， 2025​​ 

SACRAMENTO — The Department of Health Care Services (DHCS), under the direction of Governor Gavin Newsom, this month applied to the Center for Medicaid and Medicaid Innovation (CMMI) at the Centers for Medicare & Medicaid Services (CMS) to participate in the groundbreaking Cell and Gene Therapy (CGT) Access Model. If approved, this multi-year initiative will expand Medi-Cal members’ access to lifesaving gene therapies for sickle cell disease (SCD), a severe genetic blood disorder that disproportionately affects people of African descent.​​ 

“California’s application to participate in this federal model reflects our commitment to expanding access to groundbreaking treatments for Medi-Cal members with sickle cell disease,” said State Medicaid Director Tyler Sadwith. “These therapies have the potential to transform lives, and this initiative helps ensure that cost is not a barrier for Californians who need them most.”​​ 

WHY THIS MATTERS: More than 50 percent of individuals with SCD are covered by Medicaid in the United States. By participating in the CGT Access Model, California will:​​ 

• 扩大患有 SCD 的 Medi-Cal 会员获得基因治疗的机会。​​ 
• 通过将 SCD 基因疗法转换为 Medi-Cal 按服务收费的药房福利来简化保险范围，从而实现更可预测和可持续的报销流程​​ 
• 通过增加历史上服务不足的社区的治疗机会来促进健康公平。​​ 
• 通过联邦政府协商的回扣提高 Medi-Cal 计划的财务可预测性。​​ 

“通过扩大这些变革性疗法的可及性，加利福尼亚州正在引领解决健康不平等问题、提高预期寿命，并确保患有镰状细胞病的Medi-Cal参保人获得最佳护理，”萨德威斯表示。

关于CGT治疗模式：基因疗法是治疗镰状细胞病的一种开创性方法。该疗法通过改造患者自身的造血干细胞，帮助机体产生健康的红细胞。研究表明，这种一次性治疗可减少严重疼痛发作，并有望预防并发症，从而改善镰状细胞病患者的生活质量。​​ 

However, these therapies come with extraordinary costs, making access difficult for patients and Medicaid programs, including Medi-Cal. The CGT Access Model addresses this challenge by using a negotiated rebate system that ties payments for these treatments to patient outcomes. If the therapy does not meet expected health improvements, drug manufacturers will provide rebates to Medicaid programs, helping ensure financial sustainability while expanding access to care.​​ 

Initially, the model will focus on gene therapies for SCD, a condition affecting more than 100,000 individuals nationwide, including more than 8,000 Medi-Cal/Children’s Health Insurance Program (CHIP) members in California.​​ 

If California is approved, eligible Medi-Cal members will have access to gene therapy treatment, case management, travel assistance, behavioral health support, and fertility preservation services, as the treatment process involves chemotherapy, which can impact fertility. CMS anticipates testing the model over an 11-year performance period, beginning on January 1, 2025.​​ 

BIGGER PICTURE: In addition to applying for the CGT Access Model, California was selected earlier this year for the Transforming Maternal Health (TMaH) Model, which aims to improve maternal health outcomes and reduce costs, and is transforming Medi-Cal through its California Advancing and Innovating Medi-Cal (CalAIM) waiver, which focuses on whole-person care, care management, and addressing social drivers of health—strengthening efforts to create a more inclusive and effective health system.​​ 

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通讯办公室​​ 
(916) 440-7660
DHCSPress@dhcs.ca.gov​​