Medicaid Faces Costly Frontier With Sickle Cell Gene Therapies

Multimillion-dollar gene therapies promising treatment for sickle cell disease have hit the market, leaving state Medicaid programs confronting how to foot the bill for these breakthrough drugs.

Vertex Pharmaceuticals’ Casgevy and Bluebird Bio‘s Lyfgenia in December 2023 became the first gene therapies to receive FDA approval for the treatment of sickle cell disease. These treatments target the genetic defect causing sickle cell disease, where abnormal hemoglobin causes red blood cells to become crescent-shaped and block blood flow.

Sickle cell disease affects approximately 100,000 Americans, with African Americans making up over 90% of those cases.

Clinical trials have shown ...