The Institute for Clinical and Economic Review on Wednesday released a Final Evidence Report and Report-at-a-Glance on voretigene neparvovec (VN; Luxturna, Spark Therapeutics) for treatment of vision loss associated with RPE65-mediated retinal disease. Approved in 2017, VN is the first treatment for this condition.
ICER’s report was reviewed at a public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), where the Council unanimously voted that the therapy provides a net health benefit to those affected by RPE65-mediated retinal disease.
Results of votes on value were mixed, with the majority of the members voting that VN provided intermediate long-term value for money.
Uncertainty remains around the long-term effectiveness of the therapy, and ICER’s analyses found cost-effectiveness to far exceed commonly accepted thresholds; however, the therapy’s role as the only treatment option available for an ultra-rare condition, and as the first therapy approved in the U.S. targeting a disease caused by mutations in a specific gene, created special considerations that weighed heavily in many Council members’ votes.
The Final Evidence Report can be found here.