Words by Isabel O’Brien
The US government has announced a major step towards increasing access to potentially life-changing treatments for sickle cell disease, with the Centers for Medicare and Medicaid Services (CMS) securing agreements with Bluebird Bio and Vertex Pharmaceuticals for participation in its new 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 made by the two companies, aiming to improve health outcomes, increase access to these therapies and reduce healthcare costs. Under this model, payments will be tied to whether the therapies improve health outcomes for people with Medicaid.
“Cell and gene therapies hold significant potential to improve patient outcomes and transform lives, ultimately reducing long-term health care spending,” said Liz Fowler, Deputy Administrator and Director, CMS Innovation Center. “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.”
The two gene therapies now included in the model were both approved by the FDA in early 2024 for the treatment of sickle cell disease. Outcomes-based agreements negotiated by CMS will provide participating states with technical assistance and the necessary data infrastructure to implement these agreements.
Sickle cell disease, which disproportionately affects Black and Hispanic Americans, leads to significant pain, a reduced life expectancy and high healthcare costs. More than 100,000 people in the US live with the disease, with approximately 50% to 60% of them enrolled in Medicaid. The disease costs the healthcare system nearly $3bn annually.
“This is a new frontier in providing access for people with sickle cell disease to potentially transformative treatments,” said Chiquita Brooks-LaSure, Administrator, CMS. “[We are] 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.”
The Cell and Gene Therapy Access Model is set to launch in January 2025, with states able to begin participation anytime between January 2025 and January 2026. This move aligns with broader efforts by the Biden-Harris Administration to expand access to novel therapies and lower prescription drug costs.
