Posted by Michael Wonder on 13 Jan 2018
Institute for Clinical and Economic Review Report on voretigene neparvovec suggests substantial price discount needed to meet traditional cost-effectiveness standards
12 January 2018 - Special considerations include patient age, benefit durability, and rarity of condition.
The Institute for Clinical and Economic Review (ICER) today released its revised Evidence Report assessing the comparative clinical effectiveness and value of voretigene neparvovec (Luxturna, Spark Therapeutics) for treatment of vision loss associated with biallelic RPE65-mediated retinal disease.
“A scientific milestone, voretigene neparvovec is the first therapy approved in the US that inserts a new working copy of a gene into the cells of patients who have a genetic disorder,” noted David Rind, MD, MSc, Chief Medical Officer at ICER. “While the evidence is clear the therapy improves vision for patients over several years, the long-term duration of this benefit remains unknown. Assuming a 10- to 20-year period of benefit, at list price the treatment does not meet standard cost-effectiveness thresholds, even after accounting for the broader societal benefits improved vision has on productivity and education cost. The cost-effectiveness findings move into standard ranges only in analyses that consider these non-medical benefits while also assuming that only younger patients with the best baseline vision receive treatment.”
