The US government is rolling out a new initiative, the Cell and Gene Therapy Access Model, which uses a health outcomes payment model, with Vertex Pharmaceuticals and bluebird bio as the first manufacturers in the program. What’s the market impact?

By Patricia Van Arnum, Editorial Director, DCAT, [email protected]

A new model for funding gene-therapy access
This week (December 4, 2024), the Biden-Harris Administration announced that two drug manufacturers–Vertex Pharmaceuticals and bluebird bio–with US 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, 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 healthcare costs. These outcomes-based agreements will tie payments to whether the therapy improves health outcomes for people with Medicaid (the US government healthcare program for low-income individuals) who receive these treatments.

Having secured agreements with bluebird bio for its gene therapy, Lyfgenia (lovotibegene autotemcel), and Vertex for its gene therapy, Casgevy (exagamglogene autotemcel), CMS will now move forward with engaging US states and territories that participate in the Medicaid Drug Rebate Program to help them decide whether to participate in the model.

The FDA approved bluebird bio’s Lyfgenia and Vertex’s Casgevy n December 2023. CMS negotiated outcomes-based agreements with both manufacturers on behalf of US states and will provide states that choose to participate in the model with the technical assistance and data infrastructure to implement these agreements.

The Cell and Gene Therapy Access Model launches in January 2025, and US states may choose to begin participation anytime between January 2025 and January 2026. The state application portal goes live this month—December 2024—and will remain open through February 28, 2025. In addition, states may apply for optional model funding by responding to the notice of funding opportunity, but they are not required to respond to the notice of funding opportunity to participate in the model. The deadline for responding to the notice of funding opportunity is also February 28, 2025.

The model is part of the Administration’s broader effort to further increase access to novel therapies and lower prescription drug costs. The Center for Medicare and Medicaid Innovation Center developed the model in response to an Executive Order that President Biden issued in October 2022, directing the US Department of Health and Human Services to consider developing models that address access to novel therapies and lower drug costs. After launching in 2025, this model may be expanded to other types of cell and gene therapies in the future, according to CMS.