Cost Utility of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease
Objective: The gene therapy voretigene neparvovec (VN) is the first Food and Drug Administration-approved treatment for vision loss owing to the ultra-rare RPE65-mediated inherited retinal disorders. We modeled the cost-utility of VN compared with standard of care (SoC). Study Design: A 2-state Markov model, alive and dead, with a lifetime horizon. Methods: Visual acuity (VA) […]