Gene Therapy Access Model for Sickle Cell Disease

This document is a report detailing the Centers for Medicare & Medicaid Services' (CMS) announcement regarding the Cell and Gene Therapy (CGT) Access Model, which will focus on gene therapies for sickle cell disease (SCD). The model aims to enhance access to CGTs while linking therapy costs to clinical outcomes for Medicaid patients. The initial phase will involve outcomes-based agreements (OBAs) starting in 2025, with potential future expansions to other CGTs. The report outlines CMS's negotiation strategy with manufacturers, which includes rebates and a standardized access policy for participating states. It emphasizes the administrative support CMS will provide to state Medicaid programs, allowing them to focus on improving health outcomes. The document also discusses the approval of two gene therapies for SCD, their pricing, and the importance of considering health equity in treatment access. Strategies for state Medicaid programs are presented, highlighting the need for alignment with CMS requirements and the management of therapy administration.