The Biden-Harris administration has announced an initiative to improve access to gene therapies for sickle cell disease under the Cell and Gene Therapy Access Model.

This voluntary model, developed by the Center for Medicare and Medicaid Innovation, seeks to enhance access to these therapies, improve patient outcomes, and reduce healthcare costs by tying payments to treatment efficacy for Medicaid recipients.

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Two drug manufacturers, Bluebird Bio Inc. (NASDAQ:BLUE) and Vertex Pharmaceuticals Inc. (NASDAQ:VRTX), have agreed to participate in the model with their FDA-approved therapies, Lyfgenia and Casgevy.

CMS is working to engage states and U.S. territories enrolled in the Medicaid Drug Rebate Program, providing technical support and data infrastructure for implementation.

States may apply to participate starting this month, with applications accepted until Feb. 28, 2025.

Over 100,000 individuals in the U.S. live with sickle cell disease, more than half of whom rely on Medicaid. The disease significantly reduces life expectancy and imposes an annual economic burden of nearly $3 billion in healthcare costs.

The Food and Drug Administration (FDA) approved Lyfgenia and Casgevy in December 2023 as gene therapies targeting sickle cell disease.

Launching in January 2025, the model allows states to join until January 2026. States can also apply for optional funding to support implementation.

This initiative aligns with President Biden's 2022 Executive Order to expand access to novel therapies and reduce prescription drug costs. Future expansions of the model may include other cell and gene therapies.

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