Breaking ground

The first wave of cell and gene therapies to hit the U.S. market offer the possibility of cures but also are beginning to push the limits of existing drug pricing and payment paradigms. Through new models now in the planning phases, Spark Therapeutics Inc. and Express Scripts Holding Co. could lay the groundwork for approaches that make it easier for payers to allow access to one-time treatments that provide long-term benefits.

In patients with a rare genetic disease that can result in total or near-total vision loss, a single dose of Spark’s Luxturna voretigene neparvovec-rzyl restored vision to a level that allowed patients who were previously dependent on caregivers and devices to function independently...