3월 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:

• SCD를 앓고 있는 Medi-Cal 가입자를 위한 유전자 치료에 대한 접근성을 확대합니다.
• SCD 유전자 치료제를 Medi-Cal 행위별 수가 약국 혜택으로 전환하여 보다 예측 가능하고 지속 가능한 환급 프로세스를 통해 보험 적용을 간소화합니다.
• 역사적으로 소외된 커뮤니티의 치료 기회를 늘려 건강 형평성을 개선합니다.
• 연방 정부에서 협상한 리베이트를 통해 Medi-Cal 프로그램의 재정 예측 가능성을 높입니다.

사드위스는“이러한 획기적인 치료법에 대한 접근성을 확대함으로써 캘리포니아주는 건강 불평등을 해소하고, 기대 수명을 높이며, 겸상적혈구병을 앓고 있는 메디칼(Medi-Cal) 가입자들이 최상의 치료를 받을 수 있도록 보장하는 데 앞장서고 있다”고 말했다.

CGT 접근 모델에 대하여: 유전자 치료는 SCD에 대한 획기적인 치료법입니다. 이 치료법은 환자 본인의 조혈모세포를 변형시켜 신체가 건강한 적혈구를 생성하도록 돕습니다. 이 일회성 치료법은 심한 통증 발작을 줄이는 것으로 나타났으며, SCD 환자들의 합병증을 예방하고 삶의 질을 향상시킬 가능성이 있습니다.

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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커뮤니케이션실
(916) 440-7660
DHCSPress@dhcs.ca.gov