In a report published by the US Institute for Clinical and Economic Review (ICER), healthcare experts stated that the recently approved gene therapy for Leber’s congenital amaurosis (LCA), “Luxturna voretigene neparvovec-rzyl”, from Spark Therapeutics Inc. (NASDAQ:ONCE), may require substantial discounts in order to be cost-effective. The new drug, developed by Spark Therapeutics, a gene therapy company based in Philadelphia, USA, is pitching a price tag of $850,000 per patient, $425,000 per treated eye. While the report was positive in respect of the clinical and scientific evidence presented in support of regulatory approval, uncertainty existed in respect of the drug’s long-term benefit and durability.
Recommendation of Luxturna, previously known as “SPK-RPE65” was secured on the company’s Phase III clinical trial data, which included data from an intent-to-treat population randomized over a 12-month time period. According to Spark, the clinical trial results showed “a statistically significant and clinically meaningful difference between intervention (n=21) and control participants (n=10) at one year, per the clinical trial’s primary endpoint, mean bilateral multi-luminance mobility testing (MLMT) score change (difference of 1.6; 95% CI, 0.72, 2.41; p =0.001).” The company additionally announced that treated patients had a “marked difference compared to control participants across the first two secondary endpoints: full-field light sensitivity threshold (FST) testing averaged over both eyes (p =0.001) and the mobility test score change for the first injected eye (p =0.001).”
Nevertheless, a price tag of $425,000 per eye was deemed by ICER not to be cost effective. According to the study, if one is to assume a 10- to 20- year benefit of treatment for 15-year-olds, and calculating only direct health care system costs, ICER research indicates that for Luxturna to meet the standard cost-effectiveness threshold range the drug would should be priced at between $152,612 and $216,738 per patient. This essentially translates into a 75-82% reduction in the price being requested by Spark. Obviously the calculus would change if one were to assume a 30+ year benefit, potentially accessible through the treatment of 3 year olds, in which case the current price of $850,000 over the lifetime use of the drug could, in particular scenarios, meet the relevant threshold range. Additional analyses that consider cost effectiveness for ultra rare disorders with no other treatment options available indicated that for patients aged 15 years of age the current price of the Luxturna drug would need to be discounted by between 22% and 90% to meet relevant cost effectiveness thresholds. Spark has done considerable work with payors and insurers in an effort to obtain re-imbursement using a variety of innovative payment methods, including spreading the cost of the drug over multiple years and pegging the cost to the drug’s performance in a broader real world population. The commercial success of the drug remains to be seen over the next few years and the experience of Spark will be watched closely by many gene therapy and pharma companies assessing the actual, as opposed to the forecasted value, of gene therapy.