CMS Expands Access to Lifesaving Gene Therapies Through Innovative State Agreements

Participating states to test outcomes-based payments for sickle cell disease treatments, improving care while lowering long-term costs

The Centers for Medicare & Medicaid Services (CMS) announced today that 33 states, plus the District of Columbia and Puerto Rico, will participate in the Cell and Gene Therapy (CGT) Access Model, a bold new approach to delivering cutting-edge treatments for people on Medicaid living with sickle cell disease. Participating states represent approximately 84% of Medicaid beneficiaries with the condition, significantly expanding access to transformative care.

“This agreement is a major win for American patients and for Medicaid to provide patients new access to groundbreaking therapies for sickle cell disease,” said U.S. Health and Human Services Secretary Robert F. Kennedy, Jr. “Thanks to the leadership of Dr. Oz, CMS is making this model a reality. I look forward to seeing states lead the charge to improve health outcomes at lower costs for the American people.”

Led by the CMS Innovation Center, the model is the first time the federal government has negotiated outcomes-based agreements with CGT manufacturers on behalf of state Medicaid agencies. Under the model, participating states receive guaranteed discounts and rebates from participating CGT manufacturers if the therapies fail to deliver their promised therapeutic benefits.

“This model is a game changer,” said CMS Administrator Dr. Mehmet Oz. “CMS is giving states the tools to deliver lifesaving therapies to patients in need, while holding manufacturers accountable for outcomes and protecting taxpayer dollars.”

Sickle cell disease can be an extremely painful condition that significantly impacts overall quality of life for those affected. Many long-term health complications from sickle cell disease—including stroke, acute chest syndrome, and chronic end-organ damage—can lead to higher rates of emergency department visits and hospitalizations, and higher long-term health care expenditures.

“This model has the potential to improve health outcomes for patients with sickle cell disease while also ensuring state and taxpayer dollars are being used more effectively,” said Abe Sutton, Director of the Innovation Center and CMS Deputy Director. “This is CMS delivering innovation with purpose. By negotiating outcomes-based agreements with drugmakers, we are helping states lead on access, accountability, and affordability simultaneously.”

The model includes several key features:

• CMS-negotiated outcomes-based contracts with manufacturers, developed with input from state Medicaid agencies, patients, and providers.
• Optional federal support of up to $9.55 million per state to help with implementation, outreach, and data tracking.
• Flexible start dates between January 2025 and January 2026 for participating states.
• Potential future expansion to cover other diseases with high-cost, high-impact therapies.

The model is voluntary for states and manufacturers. Participation is open to manufacturers that responded to CMS’ Request for Applications and agreed to negotiate terms based on clinical outcomes.

This initiative underscores CMS’ commitment to accelerating access to innovative therapies, improving patient health, and ensuring the smart use of Medicaid resources.

The following states and territories are participating in the CGT Access Model: Arizona, Arkansas, California, Colorado, Connecticut, Delaware, Florida, Illinois, Kansas, Kentucky, Louisiana, Maine, Maryland, Michigan, Mississippi, Missouri, New Jersey, New York, North Carolina, Ohio, Oklahoma, Oregon, Pennsylvania, Rhode Island, South Carolina, Tennessee, Texas, Utah, Vermont, Virginia, Washington, West Virginia, and Wisconsin, as well as the District of Columbia and Puerto Rico.

To learn more about the Cell and Gene Therapy Access Model, visit: www.cms.gov/priorities/innovation/innovation-models/cgt.