Expanding Access to Sickle Cell Gene Therapies for Patients with Medicaid

Connecticut is now participating in a new federal program aimed at expanding treatment access for patients with sickle cell disease who are insured through Medicaid.

Through the Cell and Gene Therapy Access Model established by the Centers for Medicare and Medicaid Services (CMS), costs of cell and gene therapies for sickle cell disease will be tied to how well the drugs work in patients. If the treatments don’t work to their full potential, states participating in the program will receive discounts and rebates from the drug manufacturers.

The U.S. Food and Drug Administration approved two gene therapies for sickle cell disease—exagamglogene autotemcel (Casgevy™) and lovotibeglogene autotemcel (Lyfgenia™)—that while potentially curative are costly, priced at $2.2 million and $3.1 million per patient, respectively. The model is intended to make it easier for participating states to pay for these gene therapies.

Lakshmanan Krishnamurti, MD, professor of pediatrics (hematology /oncology) at Yale School of Medicine and chief of pediatric hematology, oncology, and bone marrow transplant at Yale New Haven Children’s Hospital, worked with Yale Cancer Center, Yale New Haven Hospital (YNHH), and the state of Connecticut to bring this program to the state. Both gene therapies are offered at YNHH.

“This important program will enable patients to have access to this life-changing new therapy for sickle cell disease,” says Clifford Bogue, MD, Waldemar Von Zedtwitz Professor of Pediatrics, chair of the Department of Pediatrics at Yale School of Medicine, and chief of pediatrics at Yale New Haven Health. “We are excited that Connecticut is one of the participating states.”

The Cell and Gene Therapy Access Model is the first of its kind in the United States. Connecticut is among 33 states currently participating along with Washington, D.C. and Puerto Rico, which together represent 84% of Medicaid beneficiaries with sickle cell disease. CMS says other conditions may be added to the model in the future.

In people with sickle cell disease, red blood cells that are typically smooth, round, and flexible are instead hard, crescent-shaped, and brittle. These malformed cells break down faster and can scratch and block blood vessels, leading to severe pain, inflammation, organ damage, infection, and even stroke. Sickle cell disease affects an estimated 100,000 Americans.

Prior to gene therapy, the only potential cure for patients was a bone marrow transplant, an option available to only around 25% of patients. For the other 75%, lifelong management includes medications to reduce blood cell sickling, pain treatment, and blood transfusions.

Both approved gene therapies for sickle cell disease are intended to be one-time treatments. Each takes a different approach, but both ultimately reprogram the body’s blood stem cells, leading them to produce healthy red blood cells in amounts that dilute the sickle cells.

In clinical trials, 93.5% of patients receiving Casgevy and 88% of those receiving Lyfgenia experienced no vaso-occlusion events—in which sickle cells block blood vessels, causing severe pain and, over time, organ damage—in the months following treatment.

“Gene therapies have the potential to be transformative for patients with sickle cell disease,” says Krishnamurti. “Freed from disabling pain, patients have the possibility of normalcy for the first time in their lives.

“CMS has taken a major step forward to make these treatments available at a reasonable cost and to implement these programs in a way that improves access to these complex treatments. The Cell and Gene Therapy Access Model for sickle cell is likely be a forerunner for similar treatments for many diseases in the future.”