CMS Expands Access to Lifesaving Gene Therapies Through Innovative State Agreements

Strategic Insights -

The U.S. Department of Health and Human Services' Centers for Medicare and Medicaid Services has announced a significant expansion of access to groundbreaking therapies for people on Medicaid living with sickle cell disease. The Center for Medicare and Medicaid Innovation is introducing the Cell and Gene Therapy (CGT) Access Model, a new approach to delivering cutting-edge treatments for patients with this debilitating condition. The model allows states to negotiate outcomes-based agreements with CGT manufacturers, providing a guaranteed discount and rebate if the therapies fail to deliver their promised therapeutic benefits.

Key Takeaways:

  • 33 states, plus the District of Columbia and Puerto Rico, will participate in the CGT Access Model, representing approximately 84% of Medicaid beneficiaries with sickle cell disease.
  • The model is the first time the federal government has negotiated outcomes-based agreements with CGT manufacturers on behalf of state Medicaid agencies.
  • Participating states will receive guaranteed discounts and rebates from participating CGT manufacturers if the therapies fail to deliver their promised therapeutic benefits.
  • Sickle cell disease can lead to higher rates of emergency department visits and hospitalizations, as well as higher long-term healthcare expenditures.
  • The model includes several key features: CMS-negotiated outcomes-based contracts with manufacturers, optional federal support of up to $9.55 million per state, flexible start dates between January 2025 and January 2026, and potential future expansion to cover other diseases with high-cost, high-impact therapies.
  • The model is voluntary for states and manufacturers, and participation is open to manufacturers that responded to CMS' Request for Applications and agreed to negotiate terms based on clinical outcomes.

Statistics:

  • 33 states and the District of Columbia and Puerto Rico will participate in the CGT Access Model (84% of Medicaid beneficiaries with sickle cell disease).
  • Up to $9.55 million per state optional federal support for implementation, outreach, and data tracking.
  • Participating states will have flexible start dates between January 2025 and January 2026.
  • Approximately 84% of Medicaid beneficiaries with sickle cell disease will have access to the CGT Access Model.
  • Sickle cell disease can lead to higher emergency department visits and hospitalizations, as well as higher long-term healthcare expenditures.

Sources:

  • "CMS Expands Access to Lifesaving Gene Therapies Through Innovative State Agreements", a press release from the U.S. Department of Health and Human Services' Centers for Medicare and Medicaid Services (July 15).
  • www.cms.gov/priorities/innovation/innovation-models/cgt