21de marzo de 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:​​ 

• Ampliar el acceso a las terapias génicas para los miembros de Medi-Cal con anemia de células falciformes.​​ 
• Optimice la cobertura mediante la transición de las terapias génicas de SCD al beneficio de farmacia de pago por servicio de Medi-Cal, lo que resulta en un proceso de reembolso más predecible y sostenible.​​ 
• Promover la equidad en la salud aumentando las oportunidades de tratamiento para las comunidades históricamente desatendidas.​​ 
• Mejorar la previsibilidad financiera del programa Medi-Cal a través de reembolsos negociados por el gobierno federal.​​ 

"Al ampliar el acceso a estas terapias transformadoras, California está liderando el camino en la lucha contra las desigualdades en salud, la mejora de la esperanza de vida y la garantía de que los afiliados a Medi-Cal con anemia falciforme reciban la mejor atención posible", dijo Sadwith.

SOBRE EL MODELO DE ACCESO CGT: La terapia génica representa un enfoque revolucionario para la SCD. Modifica las propias células madre hematopoyéticas del paciente para ayudar al cuerpo a producir glóbulos rojos sanos. Se demostró que este tratamiento único reduce los episodios de dolor severo y tiene el potencial de prevenir complicaciones y mejorar la calidad de vida de las personas con 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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Oficina de Comunicaciones​​ 
(916) 440-7660
DHCSPress@dhcs.ca.gov​​