Novartis’ just-approved chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel (Kymriah) is going to be introduced on the market at a price of 5,000 for a single infusion, an amount that is within the range anticipated by oncologists and that Novartis characterized as well below a price level that could be justified on cost.
Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy. Each dose is a customized treatment created with a patient’s own T-cells, which are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein—a chimeric antigen receptor or CAR—that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.
Novartis estimates that the turnaround time for processing the cells is 22 days, and initially the FDA has approved one manufacturing site for conducting this work: Novartis’ Morris Plains, New Jersey, laboratory. Whereas the time involved in processing is considered critical because of the need for rapid treatment of patients, Novartis has a cryogenic process that enables it to freeze and store samples of patients’ blood cells for processing at more convenient times and earlier during the treatment cycle.
“[Tisagenlecleucel] is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Not only does [tisagenlecleucel] provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”
The price may be high for CAR T-cell therapy, but it is important to weigh the costs against the potential benefits for patients, Gwen Nichols, MD, chief medical officer for the Leukemia & Lymphoma Society, said in an interview with OncLive
. When children and young adults have their whole lives at stake, it’s easier to justify the expense of this new treatment.
Without CAR T-cell therapy, the costs of care, including a potential additional bone marrow transplant, are already “outrageously expensive,” Nichols said. “The real question is going to come when this therapy is poised to be expanded into other patients who are older, and providing the chance for 60 or 70 years of [additional] life is not what you’re talking about, and then we’re really going to have some tough value questions that we should be prepared to ask.”