Abstract

To the Editor:

In their recent article titled “Cost utility of voretigene neparvovec for biallelic RPE65-mediated inherited retinal disease,” Zimmermann et al¹ propose a cost-effectiveness model for the first gene therapy approved by the Food and Drug Administration (FDA) designed to treat an inherited genetic disorder. It is important to note that the publication consists of an abbreviated version of the analysis that the Institute for Clinical and Economic Review (ICER) released in February 2018, which culminated in a favorable review committee vote for voretigene neparvovec-rzyl (VN) of 9-3 of high/intermediate versus low long-term value for money. The committee’s vote was informed by ICER’s sensitivity analyses of their base-case model, which demonstrated that VN may be cost-effective when using appropriate health utilities, indirect costs that address the high societal impact of blindness, and/or a lifetime treatment effect. Nonetheless, Zimmermann et al conclude that “[a]t the current price, VN was unlikely to reach traditional cost-effectiveness standards compared with SoC [standard of care]."