Dhia mus rau cov ntsiab lus​​ 
Tsev Xov xwm California Thov Koom Nrog Txoj Haujlwm Tshiab Los Nthuav Kev Nkag Mus Rau Kev Kho Mob Gene Uas Cawm Tau Txoj Sia Rau Kab Mob Sickle Cell​​ 

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

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:​​ 

  • Nthuav nkag mus rau kev kho noob rau Medi-Cal cov tswv cuab nrog SCD.​​ 
  • Txhim kho qhov kev pab cuam los ntawm kev hloov SCD noob kev kho mob mus rau Medi-Cal tus nqi-rau-kev pab cuam lub khw muag tshuaj, ua kom muaj kev kwv yees ntau dua thiab muaj txiaj ntsig zoo.​​ 
  • Ua ntej kev noj qab haus huv kev ncaj ncees los ntawm kev nce kev kho mob rau cov zej zog uas tsis tau txais kev pab yav dhau los.​​ 
  • Txhim kho kev kwv yees nyiaj txiag rau qhov kev pab cuam Medi-Cal los ntawm tsoomfwv cov nyiaj rov qab sib tham.​​ 

"Los ntawm kev nthuav dav kev nkag mus rau cov kev kho mob hloov pauv no, California tab tom ua tus thawj coj hauv kev daws teeb meem kev tsis sib luag ntawm kev noj qab haus huv, txhim kho lub neej ntev, thiab xyuas kom meej tias cov tswv cuab Medi-Cal uas muaj kab mob sickle cell tau txais kev kho mob zoo tshaj plaws," Sadwith hais.

TXOG TUS QAUV CGT ACCESS: Kev kho mob gene sawv cev rau txoj hauv kev tshiab rau SCD. Nws hloov kho tus neeg mob cov hlwb hematopoietic kom pab lub cev tsim cov qe ntshav liab noj qab haus huv. Kev kho mob ib zaug no tau pom tias yuav txo tau qhov mob hnyav thiab muaj peev xwm tiv thaiv cov teeb meem thiab txhim kho lub neej zoo rau cov tib neeg uas muaj 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.​​ 

###​​ 

Office of Communications​​ 
(916) 440-7660
DHCSPress@dhcs.ca.gov​​