North Carolina is one of 35 states participating in a federal model that makes it easier for the state to afford gene therapy treatments for Medicaid recipients.
The Cell and Gene Therapy (CGT) Access Model aims to increase Medicaid recipients’ access to gene therapy treatments for rare diseases. CGT lets the Centers for Medicaid and Medicare Services, a federal agency, form contracts with manufacturers of gene therapy treatments on behalf of states to decrease treatment costs paid by state Medicaid agencies.
If the treatments aren’t as effective as expected, the contracts require that manufacturers partially reimburse states for their costs.
CGT is a new pilot program under the Centers for Medicaid and Medicare Services, and this first iteration specifically targets access to gene therapy treatments for sickle cell disease. Sickle cell disease is a genetic blood disorder that changes the shape of blood cells’ oxygen-carrying proteins. Instead of being flexible and circular, the proteins are hard and sickle-shaped, often clogging blood flow and causing debilitating pain.
“This model will increase access to and affordability of gene therapies for sickle cell disease,” a North Carolina Department of Health and Human Services spokesperson told CityView. “These gene therapies can have high upfront costs but can significantly improve health outcomes and quality of life for individuals with sickle cell disease, thus potentially reducing health care utilization and spending over time.”
The model covers the two Federal Drug Administration-approved gene therapy treatments for sickle cell disease, Lyfgenia and Casgevy. Both are one-time treatments that cost over $2 million per patient. Medicaid requires states to cover the gene therapy treatments, placing a heavy burden on state budgets responsible for funding a portion of Medicaid.
At 84%, the majority of the country’s Medicaid beneficiaries with sickle cell disease live in North Carolina and the other 34 states selected to participate in the model.
Out of the 11 states that collect data on babies born with sickle cell disease, North Carolina had the second-highest total between 2016 and 2020. Cumberland County represents over 6% of the state’s total number of babies born with sickle cell disease, according to data from the Centers for Disease Control and Prevention’s Sickle Cell Data Collection Program. The county had 28 babies born with the disease between 2016 and 2020, the fourth-highest county total in the state.
While the Centers for Medicaid and Medicare Services won’t officially announce the list of participating states until June, NCDHHS told CityView on April 30 it is one of them. The federal agency’s website says that one state is already implementing the model, and patients are currently receiving treatment. NCDHHS told CityView it will begin participating in the model on Oct. 1, 2025.
How the Cell and Gene Therapy Access Model makes treatments cheaper
Between May and November 2024, the Centers for Medicaid and Medicare Services negotiated contracts with Vertex Pharmaceuticals Incorporated and Bluebird Bio Inc., the manufacturers of the two FDA-approved sickle cell disease gene therapy treatments. The Centers for Medicaid and Medicare Services consulted with clinicians, patients and their advocates, and state Medicaid experts to create the best contracts with the manufacturers.
Those contracts are called outcomes-based agreements and hold manufacturers accountable for their therapies’ impact on the Medicaid recipients receiving them over time. The agreements include specific outcome measures that track the treatment’s success in improving health outcomes for recipients, NCDHHS told CityView.
Outcomes-based agreements also include coverage standards for fertility preservation services for those receiving gene therapy treatments. Since the treatments require chemotherapy that often results in infertility, manufacturers must cover the cost of services like egg or sperm preservation.
If the manufacturers fail to meet these conditions, they must partially reimburse the state Medicaid agencies for the treatments.
“This means that, if the gene therapies do not meet a set of agreed-upon outcomes, the state will get some money back from the manufacturers in the form of a rebate,” the NCDHHS spokesperson said. “Because these gene therapies are relatively new, this is an important lever to ensure that North Carolina is getting the expected value for its investment in these therapies.”
The Centers for Medicaid and Medicare Services will track the effectiveness of the gene therapy treatments and long-term health outcomes of those receiving them through data submitted by treatment providers. The centers provide technical assistance to help with data collection and the other requirements the model places on participating states.
The centers are also offering up to $9.55 million per state to help administer the model and provide other care to those receiving the gene treatments. NCDHHS said it applied for the funding and is waiting to hear whether it was awarded.
“If awarded, this funding would increase state capacity, through staff and technology support, to successfully implement the model while also allowing NC Medicaid to partner with our in-state treatment centers to offer additional supports to beneficiaries,” NCDHHS said.
NCDHHS said it had already negotiated a reimbursement policy for paying for gene therapies for sickle cell disease. However, it said CGT made it easier to afford the treatments since the contract was negotiated by the Centers for Medicaid and Medicare Services rather than the state and provides rebates if the treatments don’t work as expected.
“Compared to a single state, [the Centers for Medicaid and Medicare Services] has more leverage, expertise and capacity to negotiate these types of agreements due to its national positioning,” NCDHHS said.
Increased access to sickle cell disease treatment through transportation
North Carolina has five comprehensive sickle cell medical centers that can provide specialty care for the disease, like gene therapy treatments. Two are in the Research Triangle, one is in Charlotte, another is in Greenville and the last is in Asheville.
For residents of Cumberland, Guilford and Mecklenburg counties with sickle cell disease, which are among the five counties in North Carolina with the highest number of babies born with sickle cell disease, it can mean long drives for treatment. Cumberland County residents living with and caring for those living with sickle cell disease told CityView last year about their difficulties accessing care, including the hours they spent traveling for it.
Ellozoa McArthur is the Fayetteville regional program director of Piedmont Health Services and Sickle Cell Agency, a federally, state and community-funded case management agency for those with sickle cell disease. McArthur told CityView that transportation is the number one barrier to care for county residents living with sickle cell disease.
The Cell and Gene Therapy Access Model is supporting “ancillary services, including travel expenses,” according to the model’s webpage. Other services include case management by organizations like Piedmont Health Services and Sickle Cell Agency and behavioral health services.
“We are excited to participate in the program, and we are hopeful that the model will be successful,” NCDHHS said. “We look forward to the model expanding in the future to include negation of other disease states for value-based agreements.”
CityView Reporter Morgan Casey is a corps member with Report for America, a national service program that places journalists into local newsrooms. Morgan’s reporting focuses on health care issues in and around Cumberland County and can be supported through the News Foundation of Greater Fayetteville.