Cost Utility of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease.

•Voretigene neparvovec (VN) was first treatment for vision loss associated with RPE65–mediated inherited retinal disease, an ultra-rare condition, and the first Food and Drug Administration–approved gene therapy that targets a disease caused by mutations in a specific gene. VN improves visual ability related to night vision, but substantial heterogeneity has been observed in the treatment response. This clinical uncertainty, in conjunction with a price of $850 000 per patient to treat both eyes, indicates that the value of these treatments warrants evaluation.•We found that the incremental cost-effectiveness ratio for VN compared with supportive care was $643 800/quality-adjusted life-years (QALY) from a US healthcare system perspective and $480 100/QALY from a modified societal perspective. We did find VN to be more cost-effective for those with better visual ability at the time of treatment.•Although a single treatment for a rare disease may not produce a substantial budget impact, there are many rare diseases, thus as additional expensive gene therapies are approved, the aggregate cost may not be sustainable. Given the unique aspects of treatments for rare diseases and especially gene therapies, there is an opportunity for VN to develop new pricing or reimbursement structures to offset costs, such as value-based pricing or refunds based on efficacy.