With approximately 6,800 people in North Carolina suffering with sickle cell disease, the North Carolina Department of Health and Human Services (NCDHHS) intends to increase access to new therapies through the Cell and Gene Therapy (CGT) Access Model.

The Cell and Gene Therapy (CGT) Access Model aims to improve the lives of people with Medicaid living with rare and severe diseases by increasing access to potentially transformative treatments.

Cell and gene therapies have high upfront costs but have the potential to reduce health care spending over time by addressing the underlying causes of disease, reducing the severity of illness, and reducing health care utilization.

Initially, the model will focus on access to gene therapy treatments for people living with sickle cell disease, a genetic blood disorder that disproportionately affects Black Americans.

NCDHHS has updated a white paper it issued in December when the U.S. Food and Drug Administration approved the historic therapies named Casgevy and Lyfgenia, which are now available on the market. In clinical trials, both therapies were found to reduce or completely eliminate the extreme pain crises experienced by people living with sickle cell disease.

For additional information on the Cell and Gene Therapy Access Model, see the fact sheet and CGT model page.

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U.S. Set to Approve First Gene-editing Treatment for Sickle Cell Patients