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) and visual field (VF) were tracked to model quality-adjusted life-years (QALYs). VN led to an improvement in VA and VF that we assumed was maintained for 10 years followed by a 10-year waning period. The cost of VN was $850,000, and other direct medical costs for depression and trauma were included for a US healthcare system perspective. A modified societal perspective also included direct nonmedical costs and indirect costs.

Results: VN provided an additional 1.3 QALYs over the remaining lifetime of an individual. The average total lifetime direct medical cost for individuals treated with VN was $1,039,000 compared with $213,400 for SoC, leading to an incremental cost-effectiveness ratio (ICER) of $643,800/QALY from the US healthcare system perspective. Direct nonmedical costs totalled $1,070,900 for VN and $1,203,300 for SoC, and indirect costs totalled $405,400 for VN and $482,900 for SoC, leading to an ICER of $480,100/QALY from the modified societal perspective.

Conclusions: At the current price, VN was unlikely to reach traditional cost-effectiveness standards compared with SoC. VN has important implications for both development and pricing of future gene therapies; therefore clinical and economic analyses must be carefully considered.