The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene Therapy Access Model.
Participants include the District of Columbia, Puerto Rico and 33 states such as California, Florida, Illinois, New York, Pennsylvania and Texas. Collectively, the states’ footprints represent 84% of Medicaid beneficiaries with sickle cell disease.
It is the first time the federal government has negotiated “outcomes-based agreements” with cell and gene therapy manufacturers for state Medicaid agencies, a news release said.
“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 Department of Health and Human Services Secretary Robert F. Kennedy Jr. in a statement. Kennedy said in a speech in April that there would be 35 participants in the model.
Should drug manufacturers promise—and fail to deliver—clinical benefits, states will receive discounts and rebates from the manufacturers. The feds can support up to nearly $10 million per state to help with implementation and data tracking.
Participants have until January 2026 to begin. The CMS again stressed that other diseases may be addressed in the future.
“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,” Abe Sutton, director of the Innovation Center and deputy director of CMS, said. “By negotiating outcomes-based agreements with drugmakers, we are helping states lead on access, accountability and affordability simultaneously.”
Cell and gene therapies result in expensive upfront costs but have the potential to reduce long-term spending through disease prevention.
The feds named two drug manufacturers, Vertex Pharmaceuticals and bluebird bio, as model participants in December. It’s expected that more than 100,000 people have sickle cell disease across the country, mostly impacting Black and Hispanic Americans.
Employers, health plans and unions overwhelmingly view the affordability of cell and gene therapies as a major or moderate challenge, a report from the Pharmaceutical Strategies Group found. Most respondents also said they don’t participate in value-based contracts or warranties through risk-sharing programs with manufacturers.