Two Drug Manufacturers Agree to Participate in Sickle Cell Disease Therapy Access Model with CMS

The Biden-Harris Administration has announced that “two drug manufacturers with Food and Drug Administration-approved gene therapies for sickle cell disease have entered into agreements with the Centers for Medicare & Medicaid Services (CMS) to participate in the Cell and Gene Therapy Access Model.”

The voluntary model will “test outcomes-based agreements for cell and gene therapies, with the aim to improve health outcomes, increase access to cell and gene therapies, and lower health care costs.”

Sickle cell disease is a potentially “extremely painful condition that significantly impacts overall quality of life for those affected. This disease disproportionately impacts Black and Hispanic Americans and has historically had limited treatment options. In the United States, more than 100,000 people live with sickle cell disease, with approximately 50% to 60% enrolled in Medicaid. Individuals with the disease have a shorter life expectancy, by more than 20 years on average. Hospitalizations and other adverse health episodes related to sickle cell disease cost the health system almost $3 billion annually.”

Cell and gene therapies for sickle cell disease typically have high costs. The model aims to give state Medicaid agencies “more budget predictability while helping improve access to these innovative therapies for people with Medicaid with sickle cell disease.”

The access model launches in January 2025, and “states may choose to begin participation anytime between January 2025 and January 2026.”