The CMS Sickle Cell Gene Therapy Access Model

In January 2024, the US Centers for Medicare & Medicaid Services (CMS) launched the Cell and Gene Therapy (CGT) Access Model, which uses novel outcome-based payment mechanisms to improve access to CGTs and reduce Medicaid spending.¹

The model initially focuses on CGTs for sickle cell disease (SCD), a genetic blood disorder affecting approximately 100 000 people in the US, 50% of whom are covered by Medicaid.² Current SCD treatment options include pharmacotherapies that reduce symptoms and bone marrow transplantation for a limited proportion of patients.² Two recently approved CGTs offer new hope for people living with SCD: lovotibeglogene autotemcel and exagamglogene autotemcel. These therapies have demonstrated significant clinical benefit in clinical trials. However, uncertainty about their long-term benefit and adverse effects and their estimated $2 million to $3 million price tag have raised concerns.² Given that SCD disproportionately affects Black and lower-income US residents,² there are also concerns about equitable access to these treatments. In this Viewpoint, we describe the model and its objectives, discuss critical decision points for its implementation, and provide policy recommendations.

Ballreich, J., Odouard, I. C., & Socal, M. P. (2025). The CMS Sickle Cell Gene Therapy Access Model. JAMA pediatrics, 179(2), 118-120.