EE591 Economic Modeling Considerations for Rare Neurodegenerative Diseases of Infancy and Early Childhood

Innovations in regenerative therapies in the past decade have provided much-needed treatment options for rare neurodegenerative diseases of infancy and early childhood that were once considered untreatable. Although challenges in evaluating rare disease treatments using cost-effectiveness analyses (CEAs) have been reported widely, strategies for overcoming specific challenges in rare developmental disorders have not been well established.