The White House announced (fact sheet) the selection of sickle cell disease (SCD) as the initial focus of the Cell and Gene Therapy (CGT) Access Model, aimed at enhancing health outcomes, increasing access to therapies, and reducing healthcare costs for vulnerable populations. With over 100,000 Americans affected by SCD, the model, led by the Centers for Medicare and Medicaid Services (CMS), will negotiate outcomes-based agreements with manufacturers, which will tie pricing for SCD treatments to whether the therapy improves health outcomes for people with Medicaid, beginning in 2025. Participating states will then decide whether to enter into an agreement with manufacturers based on the negotiated terms and offer the agreed-upon standard access policy in exchange for rebates as negotiated by CMS. The initiative aligns with broader efforts to lower drug costs and responds to President Biden’s executive order. Medicaid-enrolled individuals with SCD stand to benefit from increased access to potentially transformative gene therapies. The model aims to address access challenges, lower long-term health spending, and improve patient outcomes for those with SCD.
