Posted by Michael Wonder on 15 Jun 2017
Institute for Clinical and Economic Review to evaluate voretigene neparvovec in upcoming review
15 June 2017 - First gene therapy to be submitted for approval to FDA; potential for life-time benefits with single treatment raises questions for value assessment, pricing, and payment options.
The Institute for Clinical and Economic Review will develop a report assessing the comparative clinical effectiveness and value of voretigene neparvovec (Spark Therapeutics), a potentially one-time gene therapy for vision loss associated with biallelic RPE65-mediated retinal disease.
Voretigene neparvovec is the first gene therapy to be submitted for approval by the US FDA.
