The Biden-Harris Administration has declared sickle cell disease (SCD) as the initial focus of the Cell and Gene Therapy (CGT) Access Model, first introduced in February 2023. This initiative aims to enhance health outcomes, widen access to cell and gene therapies, and diminish healthcare expenses for some of the nation’s most vulnerable demographics.

Addressing Sickle Cell Disease Challenges

Sickle cell disease is notably painful, disproportionately affecting Black Americans and offering limited treatment options. In the United States, over 100,000 individuals grapple with SCD, experiencing a reduced life expectancy by more than two decades compared to those without the condition. Furthermore, SCD often leads to severe complications such as stroke, acute chest syndrome, and chronic end-organ damage, resulting in increased emergency department visits and hospitalizations. Access to quality and affordable care remains a challenge for SCD patients.

Government Response and Objectives

The Health and Human Services (HHS) Secretary Xavier Becerra highlighted the administration’s commitment to utilizing all available resources to enhance healthcare access and reduce costs. The CGT Access Model aligns with the administration’s broader efforts to lower prescription drug expenses, responding to President Biden’s executive order from October 2022. Led by the Centers for Medicare & Medicaid Services’ (CMS’) Innovation Center, the model will trial outcomes-based agreements (OBAs) for revolutionary CGTs, aiming to increase access to potentially life-changing treatments.

Expanding Access and Affordability

CMS Administrator Chiquita Brooks-LaSure emphasized the transformative potential of gene therapies for SCD, not only improving patient health but also potentially generating savings for states and taxpayers. The initiative targets Medicaid enrollees, as approximately 50% to 60% of SCD patients are covered by Medicaid, with related hospitalizations and health episodes costing nearly $3 billion annually.

The Framework and Negotiations

CMS will collaborate with states and manufacturers to develop a framework for expanding access to gene therapies for SCD treatment. Negotiations between CMS and manufacturers will involve OBAs tying pricing to therapy outcomes, along with additional pricing rebates and a standardized access policy. States will then decide whether to engage based on the negotiated terms.

Future Perspectives*

The CGT Access Model, commencing in January 2025, offers states flexibility in choosing participation timelines. CMS aims to address care delivery gaps during negotiations, including the provision of fertility preservation services alongside gene therapy for SCD treatment. Optional funding will incentivize activities promoting equitable access to cell and gene therapies, as well as comprehensive care for Medicaid recipients with SCD.

Highlights

• The Biden-Harris Administration prioritizes sickle cell disease for the Cell and Gene Therapy Access Model, aiming to improve health outcomes and reduce healthcare costs.
• Sickle cell disease disproportionately affects Black Americans, with limited treatment options and significant health complications.
• The initiative responds to President Biden’s executive order, focusing on outcomes-based agreements to enhance access to groundbreaking therapies.
• Medicaid enrollees, comprising 50-60% of SCD patients, are targeted for expanded access to gene therapies, potentially saving billions in healthcare expenses.
• CMS will negotiate agreements with manufacturers, offering states flexibility in participation and addressing care delivery gaps, including fertility preservation services.
• The CGT Access Model, starting in January 2025, emphasizes equitable access and comprehensive care for SCD patients under Medicaid.