CMS launches cell and gene therapy access model for Medicaid patients with sickle cell disease

The Centers for Medicare & Medicaid Services (CMS) announced last week that 33 states along with the District of Columbia and Puerto Rico have committed to joining the Cell and Gene Therapy (CGT) Access Model.

The initiative represents the first time the federal government has negotiated outcomes-based agreements with gene therapy manufacturers on behalf of state Medicaid agencies. Under these agreements, states will receive guaranteed discounts and rebates if the therapies fail to deliver their promised therapeutic benefits, according to CMS.

The model will cover about 84% of Medicaid beneficiaries with sickle cell disease within the participating states and territories. Between 50% and 60% of people living with sickle cell disease have Medicaid coverage.

According to the CDC, sickle cell disease, a hereditary blood disorder, affects about 100,000 people in the US.

CMS administrator Mehmet Oz called the model “a game changer,” noting that it gives “states the tools to deliver lifesaving therapies to patients in need, while holding manufacturers accountable for outcomes and protecting taxpayer dollars.”

Meanwhile, the Sickle Cell Disease Association of America (SCDAA) has welcomed the announcement.

In a separate statement that organization said, “For a long time, the only disease-modifying therapy for sickle cell disease was hydroxyurea, which doesn’t work for many sickle cell patients and leaves the community with limited options for care. The new gene therapies that were approved by the FDA in 2023 will allow individuals the opportunity for transformative therapy with potential cure,” said Edward Donnell Ivy, SCDAA chief medical officer.

“However, access to this new therapy might be limited by the cost of the treatment, so it is important for CMS and other stakeholders to get involved in ensuring access to this therapy, particularly since it has been demonstrated that a large portion of the sickle cell population relies on Medicaid for access to care,” he stated.

The model is voluntary for both states and manufacturers. Only companies that responded to CMS’s Request for Applications and agreed to negotiate terms based on clinical outcomes are eligible to participate.

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