CGT Access Model Aims to Expand Access to Sickle Cell Treatments

Thirty-three states, the District of Columbia, and Puerto Rico will participate in CMS’ voluntary Cell and Gene Therapy (CGT) Access Model, which aims to increase access to cutting-edge sickle cell disease treatments for Medicaid beneficiaries.1

States participating in the multiyear, voluntary model will receive guaranteed discounts and rebates from CGT manufacturers if therapies do not lead to improved outcomes.2 The model, led by the CMS Innovation Center, marks the federal government’s first time negotiating outcomes-based payments with CGT manufacturers for state Medicaid agencies.1

The first gene therapies for sickle cell disease were FDA approved in 2023, but high upfront costs have been barriers to patient access to these life-changing treatments. | Image credit: RFBSIP - stock.adobe.com

“This agreement is a major win for American patients and for Medicaid to provide patients new access to groundbreaking therapies for sickle cell disease,” HHS Robert F. Kennedy, Jr, said in a press release. “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.”

The CGT Access Model includes treatments for sickle cell disease, a blood disorder that can manifest as debilitating pain crises and a range of systemic complications that can lead to emergency department visits and hospitalizations.3 Approximately 84% of patients covered by Medicaid reside in states participating in the model, according to the press release from CMS.1. The new framework lowers the prices of CGTs for sickle cell disease for states and ties payment to outcomes, making it easier for states to pay for these potentially life-changing therapies.2

“This model is a game changer,” CMS Administrator Mehmet Oz, MD, said in a statement.1 “CMS is giving states the tools to deliver lifesaving therapies to patients in need, while holding manufacturers accountable for outcomes and protecting taxpayer dollars.”

The first gene therapies for sickle cell disease were FDA approved in 2023, but high upfront costs have been barriers to patient access to these life-changing treatments.4 Sickle cell disease primarily affects non-Hispanic Black or African American individuals, and patients face an estimated life expectancy more than 20 years shorter than the US average.5 Many people with the disease have difficulty accessing appropriate care.

Several key aspects of the CGT Access Model aim to increase access to care, including CMS-negotiated outcomes-based contracts with manufacturers that have been developed with input from state Medicaid agencies, patients, and providers; optional federal support of up to $9.55 million for each participating state to help with implementation, outreach, and data tracking; flexible start dates between January 2025 and January 2026; and the potential for expansion to other diseases that have high-cost, high-impact therapies.1 Participation is optional for both states and manufacturers.

States and territories participating in the model are 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, Wisconsin, the District of Columbia, and Puerto Rico.

“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, in a statement. “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.”

References

1. CMS expands access to lifesaving gene therapies through innovative state agreements. News release. CMS. July 15, 2025. Accessed July 18, 2025. https://www.cms.gov/newsroom/press-releases/cms-expands-access-lifesaving-gene-therapies-through-innovative-state-agreements

2. Cell and Gene Therapy Access Model. CMS.gov. Accessed July 18, 2025. https://www.cms.gov/priorities/innovation/innovation-models/cgt

3. How sickle cell disease may affect your health. National Heart, Lung, and Blood Institute. Updated August 22, 2024. Accessed July 18, 2025. https://www.nhlbi.nih.gov/health/sickle-cell-disease/health-effects

4. Kaltwasser J. High costs of sickle cell therapies highlight paradox of drug development. AJMC®. August 9, 2024. Accessed July 18, 2025. https://www.ajmc.com/view/high-costs-of-sickle-cell-therapies-highlight-paradox-of-drug-development

4. Data and statistics on sickle cell disease. CDC. May 15, 2024. Accessed July 18, 2025. https://www.cdc.gov/sickle-cell/data/index.html