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Centers for Medicare & Medicaid Services | CMS | 2024


CMS's Focuses on Sickle Cell Disease within the Cell and Gene Therapy Access Model

CMS's Focuses on Sickle Cell Disease within the Cell and Gene Therapy Access Model

The CMS declared that sickle cell disease (SCD) will take precedence in the Cell and Gene Therapy (CGT) Access Model, as initially disclosed in February 2023. The model aims to enhance health results, broaden access to cell and gene therapies, and decrease health care expenses for some of the nation’s most vulnerable communities.

Sickle cell disease is a highly agonizing condition, disproportionately affecting Black Americans and offering limited treatment alternatives. In the United States, over 100,000 individuals contend with SCD. Those with the disease face a life expectancy reduction of more than 20 years compared to individuals without SCD.

Additionally, enduring health complications from SCD, such as stroke, acute chest syndrome, and chronic end-organ damage, lead to heightened rates of emergency department visits and hospitalizations. Patients with SCD confront difficulties accessing quality and affordable care.

This model has the potential to enhance health outcomes for SCD patients and families, ensuring more effective utilization of taxpayer dollars. The CGT Access Model is part of the Administration's broader initiative to further reduce prescription drug costs, responding to President Biden's October 2022 executive order directing the Department of Health and Human Services to explore models enhancing access to innovative therapies and diminishing drug costs.

Led by the Centers for Medicare & Medicaid Services’ (CMS’) Innovation Center, the model will examine outcomes-based agreements (OBAs) for pioneering CGTs. Successful OBAs will boost affordable access to potentially life-saving and transformative treatments. Commencing in 2025, this model may expand to other CGT types in the future.

Approximately 50% to 60% of individuals with SCD are Medicaid enrollees. Health system costs for hospitalizations and other SCD-related episodes total nearly $3 billion annually. Gene therapies for SCD and other intricate conditions hold substantial potential to enhance patient outcomes, potentially reducing long-term health expenditures. However, their high cost poses challenges to state budgets. Over the next year, CMS will collaborate with participating states and manufacturers to establish a framework broadening access to gene therapies for SCD treatment. Under the model, CMS will negotiate an OBA with manufacturers, linking SCD treatment pricing to therapy's impact on Medicaid individuals' health outcomes. Negotiations will also involve additional pricing rebates and a standardized access policy. Participating states will decide whether to engage with manufacturers based on the negotiated terms, providing the agreed-upon access policy for rebates as determined by CMS.

As part of the CGT Access Model, CMS will negotiate financial and clinical outcome measures, overseeing results, and evaluating outcomes. The CGT Access Model kicks off in January 2025, with states having the option to join between January 2025 and January 2026.



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