Approval of the drug tisagenlecleucel (Kymriah; Novartis) by the US Food and Drug Administration (FDA) for the treatment of pediatric and young adult acute lymphoblastic leukemia (ALL) in August 2017 was a landmark in oncology. The science underlying chimeric antigen receptor T cells (CAR-T) heralds a new era of treatment, and the list price of $475 000 for the new drug (delivered as a one-time infusion) definitively shattered oncology drug pricing norms.1
Bach PB, Giralt SA, Saltz LB. FDA Approval of Tisagenlecleucel: Promise and Complexities of a $475 000 Cancer Drug. JAMA. 2017;318(19):1861–1862. doi:10.1001/jama.2017.15218
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