The Institute for Clinical and Economic Review (ICER) on Friday 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.”
“When considering treatments intended for ultra-rare diseases such as this form of blindness, decision-makers often give special weighting to other contextual factors that may lead to coverage at prices that exceed traditional cost-effectiveness thresholds. That being said, policymakers also will need to consider how to manage pricing and payment arrangements for voretigene neparvovec to ensure long-term affordability, as well as patient access to the coming wave of genetic therapies for other conditions.”