CMS Launches Value-Based Gene Therapy Program for Sickle Cell Disease

A new federal payment model launching today will let states offer gene therapies for sickle cell disease at a lower cost by having the federal government negotiate prices with drugmakers.

CMS has launched the Cell and Gene Therapy (CGT) Access Model, making this the first time CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., and Puerto Rico, have signed on, collectively covering about 84% of Medicaid enrollees with the condition, according to a news release.

"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., in a statement.

Participating states gain access to CMS-negotiated pricing contracts that include guaranteed rebates if a treatment fails to meet clinical expectations. In exchange, states agree to adopt uniform access policies that support coverage and care coordination for eligible patients.

Mehmet Oz, M.D.

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

Gene therapies for conditions like sickle cell disease can exceed $2 million per treatment. These one-time treatments aim to correct the underlying cause of disease but carry substantial upfront costs, making them a challenge for state Medicaid budgets that operate under year-to-year constraints. By pooling negotiating power and standardizing contracts, CMS aims to create sustainable payment models that help expand access.

Under the new program, manufacturers of FDA-approved gene therapies for sickle cell disease launched before May 2024 were invited to negotiate outcomes-based agreements with CMS. These contracts include supplemental rebates, volume-based pricing discounts, and predefined metrics to assess treatment success over time.

States, meanwhile, receive technical and financial support to implement the model. This includes up to almost $10 million per state to cover costs associated with provider contracting, data collection and patient support services

Those receiving gene therapy for sickle cell disease require extended inpatient hospital stays and potentially multiple visits to specialized treatment centers. The preparatory process involves chemotherapy, which typically renders patients infertile. The model includes coverage for fertility preservation services and support for ancillary services, including travel expenses, case management and behavioral health services.

The Cell and Gene Therapy Access Model represents the first time the federal government has negotiated outcomes-based agreements with manufacturers on behalf of state Medicaid agencies. Under the program, participating states receive guaranteed discounts and rebates from manufacturers if the therapies fail to deliver their promised therapeutic benefits.

"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.

CMS said it is already in discussions with stakeholders about future conditions the model may include.

The participating states 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 and Wisconsin.