This article has been updated with additional information from CMS.
NEW YORK – The US Centers for Medicare & Medicaid Services on Wednesday announced that Vertex Pharmaceuticals and Bluebird Bio have agreed to participate in its Cell and Gene Therapy (CGT) Access Model, which it expects to launch in January 2025.
CMS earlier this year announced plans for the CGT Access Model, which will initially focus on sickle cell disease. Under this model, the agency will partner with state Medicaid programs and drug manufacturers and establish outcomes-based agreements to price sickle cell treatments tied to health outcomes.
Vertex's Casgevy (exagamglogene autotemcel), a gene-editing therapy it codeveloped with CRISPR Therapeutics, and Bluebird's gene therapy Lyfgenia (lovotibeglogene autotemcel), both received the US Food and Drug Administration's approval as treatments for sickle cell disease in late 2023. These were the first gene therapies approved for this condition.
Now that CMS has secured agreements with Vertex and Bluebird, the agency will engage states and US territories that participate in the Medicaid Drug Rebate Program as potential partners in the CGT Access Model. The application for states to join the program will launch this month and close in February. States will be able to choose to begin participating between January 2025 and January 2026.
As part of the CGT Access Model, CMS negotiated separate outcomes-based agreements with Vertex and Bluebird on behalf of the participating states. The agency will also provide participating states with technical assistance and data infrastructure to implement the outcomes-based agreements.
"Cell and gene therapies hold significant potential to improve patient outcomes and transform lives, ultimately reducing long-term healthcare spending," Liz Fowler, director of the Center for Medicare and Medicaid Innovation (CMMI) at CMS, said in a statement. "However, due to the high costs, these therapies can pose challenges to state budgets. This model will afford state Medicaid agencies more budget predictability while helping improve access to these innovative therapies for people with Medicaid with sickle cell disease."
CMS has shared "key terms" of the agreements that CMS has negotiated with Vertex and Bluebird with states and territories, a spokesperson said in an email. The spokesperson said CMS is unable to disclose additional details about the agreements "due to their highly confidential and proprietary nature."
"CMS believes that these agreements hold manufacturers accountable for the impact their therapies have on people with Medicaid in a meaningful way, and we are optimistic that states will find the terms of these agreements attractive and choose to join the model," the spokesperson said.
CMMI, also known as the Innovation Center, unveiled the CGT Access Model in January as a voluntary program aiming to improve access to novel therapeutics for Medicaid patients with rare and severe illnesses. Within the program, drug companies will be required to pay for certain supportive services to address barriers to accessing gene therapy, such as fertility preservation services. Drugmakers typically aren't allowed to pay for such services due to prohibitions in the federal Anti-Kickback Statute and the federal Beneficiary Inducement Statute.
CMMI will evaluate whether the initiative lowers healthcare costs by balancing high upfront costs against the potential to reduce healthcare spending over time with a one-time gene therapy, as well as whether participation in the CGT Access Model improves health outcomes for Medicaid patients.
The CGT Access Model may expand to other types of cell and gene therapies in the future, CMS said.
"This is a new frontier in providing access for people with sickle cell disease to potentially transformative treatments," CMS Administrator Chiquita Brooks-LaSure said in a statement. "CMS is pleased that these two drug manufacturers have agreed to participate in the model and work with the agency and states in providing access to potentially curative treatments that might otherwise be out of reach."