Spark Therapeutics announced three new payer programs: an outcomes-based rebate arrangement with a long-term durability measure, an innovative contracting model and a proposal to CMS under which payments for LUXTURNA™ (voretigene neparvovec-rzyl) would be made over time. Together, these initiatives aim to help ensure eligible U.S. patients have access to LUXTURNA, a one-time gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. LUXTURNA should only be administered to patients who have viable retinal cells as determined by their treating physicians. Spark Therapeutics has reached agreement in principle with Harvard Pilgrim to make LUXTURNA available under the outcomes-based rebate arrangement and the innovative contracting model that aims to reduce risk and financial burden for payers and treatment centers. Spark Therapeutics also has reached an agreement in principle with affiliates of Express Scripts to enable the innovative contracting model. LUXTURNA and other potential one-time therapies face unique health insurance challenges given current practices and regulations in the U.S. health care system. Barriers to offering alternate models include the system’s focus on short-term value, largely because most patients switch health insurance companies on average every three years; government price reporting requirements that are not designed to reflect certain outcomes-based arrangements, therefore limiting a manufacturer’s ability to offer significant performance-based rebates, particularly for diseases with small patient populations; and complicated distribution models, which add costs and financial risk to parties involved in the delivery and reimbursement of specialty drugs and specialized medical care. Spark Therapeutics will share risk with certain health insurers by paying rebates if patient outcomes fail to meet a specified threshold, thereby linking the payment for LUXTURNA to both short-term efficacy (30-90 days) and longer-term durability (30 months) measures that are unique to this one-time gene therapy. The short-term and long-term measures will be based on full-field light sensitivity threshold (FST) testing scores, with a baseline to be established for each eligible patient before administration of LUXTURNA. In addition to its agreement in principle with Harvard Pilgrim, Spark Therapeutics is in active discussions with other commercial insurers regarding this offering.