Tahanan Balita Nag-aaplay ang California na Sumali sa Groundbreaking Initiative upang Palawakin ang Access sa Lifesaving Gene Therapies para sa Sickle Cell Disease​​ 

California Applies to Join Groundbreaking Initiative to Expand Access to Lifesaving Gene Therapies for Sickle Cell Disease​​ 

Marso 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:​​ 

  • Palawakin ang access sa mga gene therapies para sa mga miyembro ng Medi-Cal na may SCD.​​ 
  • I-streamline ang coverage sa pamamagitan ng paglipat ng mga SCD gene therapies sa Medi-Cal fee-for-service na benepisyo sa parmasya, na nagdudulot ng mas mahuhulaan at napapanatiling proseso ng reimbursement​​ 
  • Isulong ang katarungang pangkalusugan sa pamamagitan ng pagpaparami ng mga pagkakataon sa paggamot para sa mga komunidad na hindi nabibigyan ng kasaysayan.​​ 
  • Pahusayin ang kakayahang mahulaan sa pananalapi para sa programang Medi-Cal sa pamamagitan ng mga rebate na nakipag-usap sa pederal.​​ 

“Sa pamamagitan ng pagpapalawak ng access sa mga transformative therapies na ito, nangunguna ang California sa pagtugon sa mga hindi pagkakapantay-pantay sa kalusugan, pagpapabuti ng inaasahang haba ng buhay, at pagtiyak na ang mga miyembro ng Medi-Cal na may sickle cell disease ay makakatanggap ng pinakamahusay na posibleng pangangalaga,” sabi ni Sadwith.

TUNGKOL SA MODELO NG CGT ACCESS: Ang gene therapy ay kumakatawan sa isang makabagong pamamaraan sa SCD. Binabago nito ang sariling hematopoietic stem cells ng isang pasyente upang matulungan ang katawan na makagawa ng malulusog na pulang selula ng dugo. Ang minsanang paggamot na ito ay naipakita na nakakabawas ng matinding pananakit at may potensyal na maiwasan ang mga komplikasyon at mapabuti ang kalidad ng buhay para sa mga indibidwal na may SCD.​​ 

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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Tanggapan ng Komunikasyon​​ 
(916) 440-7660
DHCSPress@dhcs.ca.gov​​