At $850,000, price for new childhood blindness gene therapy four times too high, analysis says

The nonprofit Institute for Clinical and Economic Review (ICER) says the $850,000 list price for
voretigene neparvovec-rzyl (Luxturna—Spark Therapeutics), the first-of-its-kind gene therapy for a form of childhood blindness caused by a mutation in the RPE65 gene, is approximately four times too high for the value the drug provides. In a new report, ICER said a cost-effective price for Luxturna should be $153,000–$217,000, reflecting a discount of 75% or more. ICER cited a lack of data that Luxturna causes permanent improvements in vision as a key reason that its developer, Spark Therapeutics, should not be charging so much. ICER reached its suggested list price for Luxturna by assuming that a 15-year-old person (the average age of the patients enrolled in the clinical trials) would experience improvements for 1–2 decades and taking into account the benefits to the health care system. However, ICER determined that the drug's list price met its standards for "cost-effectiveness thresholds" when analyzing the treatment for patients aged 3 years old. In that case, it took into account both medical and societal benefits, and assumed the vision improvements would last for the patients' whole lives.