California applies to join groundbreaking initiative to expand access to lifesaving gene therapies for sickle cell disease
What you need to know: The Department of Health Care Services (DHCS) submitted an application to the Centers for Medicare & Medicaid Services (CMS) to participate in the groundbreaking Cell and Gene Therapy (CGT) Access Model to expand Medi-Cal members’ access to lifesaving gene therapies for sickle cell disease.
Sacramento, California – Governor Gavin Newsom today announced that the Department of Health Care Services (DHCH) submitted an application to the federal 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.
“The opportunities presented by the Cell and Gene Therapy Access Model initiative will be life-changing for people with sickle cell disease. Providing greater access through this groundbreaking care model is an important step for the Medi-Cal program.”
Governor Gavin Newsom
“Sickle cell disease is a chronic, life altering condition that particularly impacts the Black community. The opportunities presented by this initiative are exciting and very promising, and we thank Governor Newsom for his support in getting treatment to California’s Medi-Cal participants.”
Senator Akilah Weber Pierson, M.D., Chair, California Legislative Black Caucus
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:
-
Expand access to gene therapies for Medi-Cal members with SCD.
-
Streamline coverage by transitioning SCD gene therapies to the Medi-Cal fee-for-service pharmacy benefit, ensuing a more predictable and sustainable reimbursement process
-
Advance health equity by increasing treatment opportunities for historically underserved communities.
-
Enhance financial predictability for the Medi-Cal program through federal negotiated rebates.
The Cell and Gene Therapy (CGT) Access Model
Gene therapy represents a groundbreaking approach to SCD by modifying a patient’s own hematopoietic stem cells to help the body produce healthy red blood cells. This one-time treatment has been shown to reduce severe pain episodes and has the potential to prevent complications and improve quality of life for individuals with 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.