Vertex, bluebird agree to Medicaid CGT access model for sickle cell therapies

The Biden-Harris Administration announced that Vertex Pharmaceuticals and bluebird bio, both with FDA-approved gene therapies for sickle cell disease, have entered into agreements with the Centers for Medicare & Medicaid Services to participate in the new cell and gene therapy access model.

The CGT access model, first unveiled in February 2023, is part of a broader government effort to drive down prescription drug costs. Under the model, state Medicaid agencies will assign CMS to coordinate and administer multi-state, outcomes-based agreements with manufacturers for certain cell and gene therapies. These outcomes-based agreements will tie payments to whether the therapy improves health outcomes for people with Medicaid who receive the drugs.

Earlier this year, CMS revealed that that sickle cell disease would be the first focus of the access model. More than 100,000 people in the U.S. live with sickle cell disease, with approximately 50% to 60% enrolled in Medicaid. Hospitalizations and other adverse health episodes related to sickle cell disease cost the health system almost $3 billion annually.

The FDA handed down approvals for bluebird's Lyfgenia, a one-time ex-vivo lentiviral vector gene therapy, and Vertex and CRISPR Therapeutics' Casgevy, a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease in patients 12 years and older on the same day in December 2023. At the time, bluebird said Lyfgenia would come with a $3.1 million price tag; Casgevy was priced at $2.2 million.

Now, having secured agreements with bluebird for Lyfgenia and Vertex for Casgevy, CMS will move forward with engaging states and U.S. territories that participate in the Medicaid Drug Rebate Program to help them decide whether to participate in the model. The model officially launches in January 2025, but states may choose to begin participation anytime between January 2025 and January 2026.