Since August 2017, we have seen the approvals of the first chimeric antigen receptor (CAR) T-cell therapies—Kymriah® (tisagenlecleucel) and Yescarta® (axicabtagene ciloleucel)—and the first directly administered gene therapy,
LuxturnaTM (voretigene neparvovec-rzyl). Though these novel treatments are offering new hope to patients with unmet medical needs, they come at a significant cost, raising important questions about how novel therapies should be evaluated and paid for.
In this white paper, we explore the current landscape of gene therapy to highlight the challenges payers, health systems, and manufacturers face in bringing these innovative medicines to the patients who need them most.