Cost-Benefit Considerations for CAR T Cell Therapy

CAR T-cell therapies tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta) have been scrutinized for their high costs.

Both FDA-approved chimeric antigen receptor (CAR) T-cell therapies, tisagenlecleucel (Kymriah, Novartis) and axicabtagene ciloleucel (Yescarta, Kite Pharma/Gilead), may come with hefty price tags, but the therapies are priced in alignment with their clinical value, according to an Institute for Clinical and Economic Review (ICER) report.

In August, tisagenlecleucel gained approval for the treatment of B-cell acute lymphoblastic leukemia (B-ALL) in certain pediatric and young adult patients. In October, axicabtagene ciloleucel was green lit by the FDA for the treatment of adult patients with diffuse large B-cell lymphoma who have not responded to or have relapsed after 2 other kinds of treatment; tisagenlecleucel is currently under FDA evaluation for this indication.

Using a cohort of patients aged 0 to 25 with relapsed/refractory B-ALL and a cohort of patients aged 18 and older with relapsed/refractory B-cell lymphoma who were ineligible for auto-stem cell transplant, the report compared the CAR T therapies with chemotherapy. The average sales price for chemotherapy treatments were used, while the wholesale acquisition cost was used for CAR T therapies, as it was the only available estimate. For this indication, the cost of tisagenlecleucel is $475,000, and the cost of axicabtagene ciloleucel is $373,000, not including other costs related to the administration of CAR T therapy. A hospital mark-up was added for each hospital-administered treatment. It was assumed that the survival benefits demonstrated over the short duration of clinical trials would persist over a patient's lifetime.

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