FDA Approval of Tisagenlecleucel: Promise and Complexities of a $475 000 Cancer Drug

Approval of the drug tisagenledeucel (Kymriah; Novartis) by the US Food and Drug Administration (FDA) for the treatment of pediatric and young adult acute lymphoblastic leukemia, definitively shattered oncology drug pricing norms for its list of price $475 000. The price of this therapy should serve as a reminder that future revenue outlooks for a pharmaceutical company are in part related to the ability to generate many millions in revenue even when few patients benefit from the new therapies. The price of $475 000 for a single treatment with tisagenledeucel is difficult to put into context because expensive cancer treatments typically cost $10 000 to $20 000 per month and reflect prices that have already increased more rapidly than the benefits they have provided patients over the past several decades.