Cost-Utility Analysis of the Treatment With Ataluren Plus Standard of Care Compared With Standard of Care Alone in Patients With Duchenne Muscular Dystrophy in Brazil

Key Points

• This analysis aims to evaluate the cost-effectiveness of ataluren in combination with standard care for patients with Duchenne muscular dystrophy in Brazil.
• Conducted a cost-utility analysis comparing two treatment approaches.
• Included patients with nonsense mutation Duchenne muscular dystrophy.
• Evaluated costs and effectiveness from the perspective of the Brazilian public health system.
• Ataluren plus standard care was found not to be cost-effective compared to standard care alone.
• The analysis suggests that standard care may be more economical in the long run.