Bluebird Bio and Vertex Pharmaceuticals are the first to participate in a scheme to enable people with sickle cell disease to access treatments they would otherwise not be able to afford. Gene and cell therapies are lifechanging treatments but remain some of the world’s most expensive drugs. The developers have agreed to join the Cell and Gene Therapy Access Model, due to launch in early 2025. The model was first announced in February 2023 as part of a Biden administration initiative to lower prescription drug costs.
Sickle cell disease is a relatively common genetic disease: in the United States it affects about 1 out of every 500 Black or African American births. It is caused by a single mutation in the β-globin gene resulting in misshapen, sickle-shaped red blood cells that get stuck in small blood vessels, causing painful vaso-occlusive events. Two potentially curative gene therapies have been approved for sickle cell disease, but without help for access, they may remain out of reach for people on Medicaid.
