Govt steps to increase access to sickle cell disease treatments in USA

The US Biden-Harris Administration yesterday announced that two drug manufacturers with Food and Drug Administration (FDA)-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, led by the Center for Medicare and Medicaid Innovation, 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. These outcomes-based agreements will tie payments to whether the therapy improves health outcomes for people with Medicaid who receive these drugs.

The FDA approved Lyfgenia (lovotibeglogene autotemcel) from bluebird bio (Nasdaq: BLUE) and Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals (Nasdaq: VRTX) in December 2023. The CMS negotiated outcomes-based agreements with both manufacturers on behalf of states and will provide states that choose to participate in the model with the technical assistance and data infrastructure to implement these agreements.