The Cell and Gene Therapy (CGT) Access Model, launched by the Centers for Medicare & Medicaid Services (CMS), targets improved health outcomes for Medicaid recipients with rare and severe diseases. It facilitates outcomes-based agreements between states and pharmaceutical manufacturers to lower costs and tie payments to treatment effectiveness, initially focusing on gene therapy for sickle cell disease. Participation is voluntary for states and manufacturers, with funding opportunities available but not mandatory. By addressing historic disparities and poor health outcomes associated with sickle cell disease, the model aims to increase access to transformative therapies and ultimately reduce healthcare spending.
Objective:
- The CGT Access Model aims to improve health outcomes by supporting outcomes-based agreements between states and pharmaceutical manufacturers.
- These agreements provide a framework that lowers prices for states and ties payment to actual treatment outcomes.
Focus Area:
- Initially, the model concentrates on gene therapy treatments for individuals living with sickle cell disease, a genetic blood disorder that disproportionately affects Black Americans.
- Over time, other conditions may be added to the model.
Implementation:
- The CGT Access Model is a multi-year voluntary model for states and manufacturers.
- States can choose to participate by responding to the state Request for Application (RFA).
- Model funding can be applied for through the Notice of Funding Opportunity (NOFO), but it’s not mandatory for participation.
- States can begin participating anytime between January 2025 and January 2026.
Why Cell and Gene Therapies?:
- Cell and gene therapies have high upfront costs, but they hold the potential to reduce overall health care spending over time.
- These therapies address the underlying causes of disease, reduce illness severity, and minimize health care utilization.
Historic Disparities and Sickle Cell Disease:
- By increasing access to transformative therapies, the model aims to address the historic disparities, poor health outcomes, and low life expectancy associated with sickle cell disease.
The model represents a significant step toward ensuring equitable access to cutting-edge treatments for those who need them most.
At Children’s Hospital of Philadelphia, we’re always learning, growing and exploring in our effort to deliver the highest quality, compassionate, family-centered care.
Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use.
Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S.
List of licensed products from the Office of Tissues and Advanced Therapies.
Cell and Gene Therapies (CGTs) are a growing class of transformative, one-time medicines designed to treat previously intractable diseases.