Sickle Cell Disease (SCD) is one of the most common genetic disorders in the world. The disease has significant impacts on quality of life and life expectancy for racial and ethnic minorities in the United States. Breakthrough, potentially curative gene therapy treatments have recently been developed and approved but remain costly and difficult to provide in terms of the long patient journey and access. In an effort to improve outcomes for those suffering from severe manifestations of SCD, the Center for Medicare and Medicaid Innovation (CMMI) is standing up a new state-based alternative payment model, the Cell and Gene Therapy Access Model, for these SCD gene therapies.
In this paper, after covering background on SCD, the new gene therapies, and the CMMI model, we leverage Milliman’s data assets to provide a high-level overview of the landscape of individuals with SCD and the providers who treat them.
