CMS to boost Medicaid access to sickle cell gene, cell therapies
With CGT Access Model, states will be reimbursed if patient sees no benefit

The Centers for Medicare & Medicaid Services (CMS) has announced that 33 states, along with the District of Columbia and Puerto Rico, have agreed to participate in the Cell and Gene Therapy (CGT) Access Model, which is intended to boost access to cell and gene therapies for people with sickle cell disease (SCD) on Medicaid.
Under the model, led by the CMS Innovation Center, participating states will receive guaranteed discounts and rebates from manufacturers of cell and gene therapies if they fail to deliver a therapeutic benefit. The participating states represent about 84% of Medicaid recipients with SCD.
“This agreement is a major win for American patients and for Medicaid to provide patients new access to groundbreaking therapies for sickle cell disease,” said U.S. Health and Human Services Secretary Robert F. Kennedy, Jr. in a press release. “I look forward to seeing states lead the charge to improve health outcomes at lower costs for the American people.”
SCD is a genetic condition that causes normally oval-shaped red blood cells to assume a sickle-like shape, which can block blood flow in small blood vessels. These blockages can increase the risk of organ damage, stroke, painful vaso-occlusive crises, and acute chest syndrome, a type of lung injury. People with SCD also have higher rates of emergency department visits and hospitalizations, driving up long-term health care costs.
A new outcomes-based model for Medicaid recipients
Until recently, the sole potential cure for SCD was a stem cell transplant, a procedure that involves replacing a patient’s blood cell precursors with those from a healthy matching donor, typically a healthy sibling. While other approved therapies can treat SCD, they don’t address its underlying genetic cause.
In 2023, two gene therapies were approved for certain patients — Casgevy (exagamglogene autotemcel) by Vertex Pharmaceuticals, at a cost of $2.2 million per patient, and Lyfgenia (lovotibeglogene autotemcel) from Bluebird Bio, listed at $3.1 million per patient.
Because between 50% and 60% of people with SCD have Medicaid coverage, the high cost of these treatments may strain the budgets of state Medicaid agencies.
The new outcomes-based model, developed with input from state Medicaid agencies, patients, and providers, ties payments to the manufacturer to the health outcomes of patients over a certain period. If the gene therapy proves less effective at improving health outcomes than expected, the drug maker will reimburse the healthcare payer a portion of the costs incurred for the treatment. Agreements will also include other concessions, such as guaranteed rebates.
The new CGT Access Model will enable easier access to these therapies for eligible Medicaid recipients. Both Bluebird Bio and Vertex have entered into separate agreements with CMS to participate.
“This model has the potential to improve health outcomes for patients with sickle cell disease, while also ensuring state and taxpayer dollars are being used more effectively,” said Abe Sutton, director of the Innovation Center and CMS deputy director. “By negotiating outcomes-based agreements with drugmakers, we are helping states lead on access, accountability, and affordability simultaneously.”
States will have the option to begin participating at a time of their choosing between January 2025 and January 2026. It includes optional federal support of up to $9.55 million per state to help with implementation, outreach, and data tracking. The duration of the awards is anticipated to be up to 10.5 years.
“This model is a game-changer,” said Mehmet Oz, MD, CMS administrator. “CMS is giving states the tools to deliver lifesaving therapies to patients in need, while holding manufacturers accountable for outcomes and protecting taxpayer dollars.”