Author Reply:

We thank Buessing et al. for their comments and suggestions. We would like to address several points raised in response to our study “Cost Utility of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease.” We modeled the cost utility of the gene therapy voretigene neparvovec (VN) compared with standard of care (SoC) for vision loss caused by the ultra-rare RPE65-mediated inherited retinal disorders. We concluded that, at the current price, VN was unlikely to reach traditional cost-effectiveness standards compared with SoC. We agree that VN has the potential to provide substantial benefit to patients and has important implications for both the development and pricing of future gene therapies; therefore clinical and economic analyses must be carefully considered.