Medicaid May Cover Million‑Dollar Sickle Cell Gene Therapies — Connecticut Joins National Pilot

Connecticut Joins Federal Pilot To Expand Medicaid Coverage For Sickle Cell Gene Therapies

Advances in cell and gene therapy now offer the potential for transformative, one‑time treatments for sickle cell disease — but high prices and low awareness have kept most patients from accessing them. Connecticut has joined 33 states, Washington, D.C., and Puerto Rico in a new federal pilot that will allow Medicaid to cover these therapies under an outcomes‑based payment model.

Why This Matters

Scale and need: The Centers for Disease Control and Prevention estimates roughly 100,000 people in the United States live with sickle cell disease. The condition disproportionately affects people of color: more than 90% of cases are among Black Americans and an estimated 3%–9% are Latino.

Current access: Fewer than 100 people nationwide have received gene therapy for sickle cell disease so far, according to Dr. Lakshmanan Krishnamurti, chief of pediatric hematology, oncology and bone marrow transplant at Yale New Haven Children’s Hospital.

How Sickle Cell Disease Works

Sickle cell disease is an inherited mutation of hemoglobin, the protein in red blood cells that carries oxygen. The mutation causes cells to become sticky and crescent‑shaped, making them fragile and prone to breaking down early. These misshapen cells can obstruct blood flow and cause anemia, severe pain crises, organ damage, infections, acute chest syndrome, stroke and other complications.

"It's like putting a lot of water in a balloon and putting it in the freezer. It becomes hard and jagged; it breaks the balloon," Krishnamurti explained.

Treatment Options And Gene Therapy

Longstanding curative options such as bone marrow transplant carry substantial risks, including infertility and immune rejection. Daily oral therapies can reduce symptoms but generally require lifelong use and may not prevent premature death.

Gene therapy offers an alternative: clinicians harvest a patient’s blood stem cells, reprogram them to produce healthy hemoglobin, and then transplant the modified cells back. The U.S. Food and Drug Administration has approved two cell‑based gene therapies for sickle cell disease — Casgevy (Vertex) and Lyfgenia (Bluebird Bio) — both authorized in 2023 for patients aged 12 and older.

Casgevy works by editing a patient’s stem cell DNA to boost production of fetal hemoglobin, which binds oxygen more tightly. Lyfgenia introduces a functional hemoglobin gene using a modified viral vector so engineered stem cells produce functional hemoglobin. Both treatments are available at Yale, and the full care pathway can take up to a year, including stem cell collection, high‑dose chemotherapy, transfusions, close monitoring and psychological support during recovery.

Costs And The CMS Pilot

List prices at Yale place Casgevy at about $2.2 million and Lyfgenia at about $3.1 million. While these are large upfront costs, clinicians note that the lifetime medical costs for many people with severe sickle cell disease can reach several million dollars, so a successful one‑time therapy could be cost‑effective over time.

The Centers for Medicare & Medicaid Services (CMS) has launched the multiyear Cell and Gene Therapy Access Model to test a federally coordinated, outcomes‑based payment approach. Under the model, payments for cell and gene therapies are tied to real‑world patient outcomes; if a therapy fails to deliver expected results, participating states can receive discounts or rebates from manufacturers. CMS says this is the first time the federal government has negotiated outcomes‑based agreements on behalf of state Medicaid programs.

Connecticut is also one of seven states receiving additional federal funds to help cover administrative costs such as provider readiness and data collection. A state Department of Social Services spokesperson said agreements are being finalized and that the program could launch by Jan. 1 if approvals are in place.

What Patients Should Know

Gene therapy could offer the chance to live without daily medications and severe activity restrictions. However, the process is intensive, requires strict medical follow‑up and is not appropriate for every patient. Patients interested in exploring gene therapy should speak with their hematologist about eligibility, risks, expected outcomes and the timeline for the pilot program.

Bottom line: The CMS pilot could expand access to expensive but potentially curative therapies for sickle cell disease by aligning payment with outcomes and reducing financial barriers through federal‑state collaboration.