Sickle Cell disease - 3D Illustration of sickled blood cells on a dark background
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Sickle cell disease (SCD) will be the first focus of the U.S.’s new Cell and Gene Therapy (CGT) Access Model, which aims to increase access to cell and gene therapies for vulnerable populations. Gene therapies are some of the most expensive treatments in the world. The Centers for Medicare & Medicaid Services (CMS) will partner with participating states and manufacturers to find ways to expand access to gene therapies for SCD. 

The CGT Access Model will be launched in January 2025, and states may choose to begin participation between January 2025 and January 2026.

Early last year, CMS announced three new models for testing to “…help lower the high cost of drugs, promote accessibility to life-changing drug therapies, and improve quality of care,” including the CGT Access Model. This program allows state Medicaid agencies to assign CMS to coordinate and administer multi-state, outcomes-based agreements with manufacturers for certain cell and gene therapies.

As more gene therapies are approved, the issue of wider access is coming to the fore. Bluebird’s thalassemia therapy Zynteglo costs $2.8 million for a one-time dose, uniQure’s hemophilia B treatment Hemgenix costs $3.5 million, and Novartis’ Zolgensma, for spinal muscular atrophy, is listed at $2 million plus. 

Countries around the world are struggling to make these available to patients. Brazil, for example, has agreed to begin covering Zolgensma for some patients, but will only pay about $1 million for each treatment.

In December last year, two gene therapies were approved for SCD by the FDA. As is typical for drugs for rare diseases, particularly gene therapies, they came with steep price tags. Bluebird Bio priced its Lyfgenia at $3.1 million, while Vertex Pharmaceutical set the cost of its Casgevy at $2.2 million. But approximately 50% to 60% of people living with SCD are enrolled in Medicaid. Hospitalizations and other health episodes related to SCD cost the health system almost $3 billion per year. 

Sickle cell disease is an extremely painful condition, which disproportionately impacts Black Americans and has had limited treatment options. In the U.S., more than 100,000 people live with SCD. Individuals with the disease have a shorter life expectancy, by more than 20 years, compared to someone without SCD. Additionally, many long-term health complications from SCD—including stroke, acute chest syndrome, and chronic end-organ damage—can lead to higher rates of emergency department visits and hospitalizations. 

“Many of the more than 100,000 Americans with sickle cell disease face difficulty accessing effective health care and groundbreaking treatments,” said U.S. health and human Services secretary Xavier Becerra. “While medical advancements bring us closer to cures, too many individuals with sickle cell disease and their loved ones still face challenges obtaining the care they need.”

The CGT Access Model is part of the Administration’s broader effort to further drive down prescription drug costs and was developed in response to an executive order that President Biden issued in October 2022 directing the Department of Health and Human Services to consider developing models that increase access to novel therapies and lower the high cost of drugs. The model, led by the CMS’ Innovation Center, will test outcomes-based agreements (OBAs) for groundbreaking CGTs. 

Under the model, CMS will negotiate an OBA with participating manufacturers, which will tie pricing for SCD treatments to whether the therapy improves health outcomes for people with Medicaid. Negotiations will also include additional pricing rebates and a standardized access policy. 

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