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:

• 擴大患有 SCD 的 Medi-Cal 成員對基因療法的訪問權限。
• 將 SCD 基因療法轉換為 Medi-Cal 服務費藥房福利，簡化覆蓋範圍，從而實現更可預測和可持續的補償流程
• 通過增加歷史上服務不足的社區的治療機會來提高健康公平。
• 通過聯邦協商的回贈，增強 Medi-Cal 計劃的財務可預測性。

「透過擴大這些具變革性療法的可及性，加州正引領潮流，致力解決健康不平等問題、提高預期壽命，並確保罹患鐮狀細胞貧血症的Medi-Cal參保人能獲得最佳的醫療照護，」薩德威斯表示。

關於 CGT 治療模式：基因療法是治療鐮狀細胞貧血症的一種突破性方法。該療法透過修改患者自身的造血幹細胞，協助身體產生健康的紅血球。研究顯示，這項單次治療能減少嚴重疼痛發作的次數，並有望預防併發症，同時改善鐳射細胞性貧血患者的生活品質。

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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通訊辦公室
(916) 440-7660
DHCSPress@dhcs.ca.gov