RARE Daily

Bluebird Bio Enters First Outcomes-Based Agreement with Medicaid for SCD Gene Therapy

March 11, 2024

Rare Daily Staff

Bluebird Bio said it has signed its first Medicaid outcomes-based agreement with the state of Michigan for its cell-based gene therapy to treat sickle cell disease.

Bluebird said it is in ongoing discussions with more than 15 Medicaid agencies representing 80 percent of Medicaid-insured individuals in the United States. Approximately 50 percent of individuals living with sickle cell disease in the United States are insured by Medicaid.

The company also said it has signed multiple outcomes-based agreements for Lyfgenia with national commercial payer organizations representing dozens of downstream plans and covering approximately 200 million U.S. lives.

Sickle cell disease (SCD) is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” blood cells. People with SCD can experience painful blood vessel blockages, also known as vaso-occlusive episodes that can lead to acute chest syndrome, stroke, jaundice, and symptoms of heart failure. Individuals may also experience anemia, which can result in end-organ damage and premature death.

Lyfgenia is a one-time ex vivo lentiviral vector gene therapy approved for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events. Lyfgenia works by adding a functional β-globin gene to patients’ own hematopoietic stem cells. Durable production of adult hemoglobin with anti-sickling properties is possible following successful engraftment. HbAT87Q has a similar oxygen-binding affinity to wild-type HbA, limits sickling of red blood cells, and has the potential to reduce and vaso-occlusive events. The list price of the therapy is $3.1 million.

Outcomes-based agreements are one way that therapeutic developers are seeking to address concerns payers have about the uncertain long-term benefits of costly one-time treatments with high price tags.

Bluebird said it has designed outcomes-based contract options unique to Lyfgenia that offer payers meaningful risk sharing tied to vaso-occlusive episode-related hospitalizations—a claims-based metric that is directly correlated with clinical benefit and aligned with study endpoints in the Lyfgenia clinical development program—with patients followed for three years.

The company’s outcomes-based contract offering for State Medicaid Agencies was designed with direct input from government payers to specifically address the challenges they face in adapting to provide access to one-time, transformative treatments, and reflects Bluebird’s experience delivering gene therapies in the commercial setting.

“Our commercial approach is built on the principle that people with sickle cell disease insured through Medicaid deserve the same timely access to gene therapy as patients with other forms of insurance,” said Tom Klima, chief commercial and operating officer, Bluebird Bio.

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