New CMS Model Aims to Unlock Access to Sickle Cell Gene Therapies

Nearly three dozen U.S. states are partnering with a new federal initiative to boost access to high-cost gene therapies for sickle cell disease. The Centers for Medicare and Medicaid Services (CMS) announced Wednesday that 33 states, D.C., and Puerto Rico will participate in the “Cell and Gene Therapy Access Model” to centrally coordinate insurance coverage. These states cover about 84% of Medicaid beneficiaries with the condition.

The program targets Vertex Pharmaceuticals’ Casgevy ($2.2 million) and Bluebird bio’s Lyfgenia ($3.1 million), both FDA-approved in December 2023. Despite their efficacy in eliminating severe pain, uptake has been slow due to their price, complex administration, and associated risks.

CMS aims to increase access through “outcomes-based agreements,” where payments are tied to treatment success, with rebates issued if benefits aren’t realized. This model will standardize negotiations across states and include federal coverage for ancillary costs like fertility preservation and travel. CMS may also provide up to $9.55 million in state support for outreach and data tracking, with potential expansion to other high-cost therapies.

CMS Deputy Director Abe Sutton stated, “This model has the potential to improve health outcomes for patients with sickle cell disease while also ensuring state and taxpayer dollars are being used more effectively.”