The US Food and Drug Administration (FDA)’s approval of tisagenlecleucel (CTL019) for young patients with relapsed or refractory acute B cell lymphoblastic leukemia (ALL) was the agency’s first for a chimeric antigen receptor T cell (CAR-T) immunotherapy.1,2

CAR-T therapies like CTL019 are “living drugs” produced by removing patients’ T cells through leukapheresis, a type of apheresis specific to white blood cells, for genetic modification at an offsite lab, amplification, and reinfusion into the patient. The engineered T cells harbor engineered antigen receptors on their surface to recognize leukemic B cells’ antigen CD19.

In the phase 2 trial of 63 patients upon which FDA approval was based, CTL019 boasted a remarkable 83% overall remission rate, raising hopes that this immunotherapy could represent a much-needed option for patients with relapsed/refractory ALL.

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But its anticipated $475,000 price tag, announced by Novartis, the drug’s developer, during a time of uncertainty about consumer protections under US health insurance law, has also sparked concern among patient advocates.3

“[CDL019] will worsen the problem of financial toxicity in America,” David Mitchell, founder and president of Patients for Affordable Drugs, a patient advocacy group based in Washington, DC, told Cancer Therapy Advisor. “Novartis should lower the price and pledge not to charge US patients more than people in other wealthy nations.”

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American taxpayers paid more than $200 million in funding for the research that led to CAR-T therapies, Mr Mitchell noted.

“Novartis and all the corporations commercializing CAR-T drugs are standing on the shoulders of taxpayers and we will continue to call for them to price the drug fairly,” he added. “Novartis received additional significant government-conferred benefits in the form of a 50% tax credit on clinical research and a priority review voucher valued by the FDA at $225 million. These taxpayer contributions must be figured into a fair price. Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more. We think the drug is priced at least $125,000 to $150,000 too high.

“The drug pricing system in America is completely broken,” Mr Mitchell added. “Until policy in this country changes, the vicious cycle of patients struggling under high drug prices will continue.”