Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with sickle cell disease with recurrent vaso-occlusive crises in the United States.

Key Points

• This analysis aims to evaluate the cost-effectiveness of exagamglogene autotemcel therapy compared to standard of care for patients with sickle cell disease.
• Projected outcomes were compared between exa-cel therapy and standard of care (SOC)
• Cost analysis included disease-related healthcare expenses
• Survival rates and frequency of vaso-occlusive crises (VOCs) were assessed
• Exa-cel therapy was projected to significantly reduce the number of VOCs.
• Survival rates improved with the use of exa-cel in patients.
• Overall disease-related costs decreased with exa-cel therapy compared to SOC.