Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with transfusion-dependent β -thalassemia in the United States

Key Points

• To evaluate the cost-effectiveness of exagamglogene autotemcel gene therapy for transfusion-dependent β-thalassemia in the United States.
• Cost-effectiveness analysis compared exagamglogene autotemcel with standard of care (SOC)
• Projected reduction in red blood cell transfusions (RBCTs) and complications
• Evaluation of survival rates and disease-related costs.
• Exagamglogene therapy projected to significantly reduce RBCTs
• Improvement in survival rates compared to SOC
• Lower overall disease-related costs, indicating cost-effectiveness.