ICER Report Shows Cost of CAR-T Therapies Aligned With Clinical Benefit


US Food and Drug Administration (FDA)-approved chimeric antigen receptor (CAR) T-cell therapies may be associated with high costs, but the therapies are priced in alignment with their clinical value, according to an Institute for Clinical and Economic Review (ICER) evidence report (February 15, 2018).

In August 2017, tisagenlecleucel (Kymriah, Novartis) received FDA approval for the treatment of B-cell acute lymphoblastic leukemia (B-ALL) in pediatric and young adult patients. In October of the same year, axicabtagene ciloleucel (Yescarta, Kite Pharma/Gilead) received approval for the treatment of adult patients with diffuse large B-cell lymphoma (DLBCL) who have not responded to or have relapsed after two previous treatments.

The ICER report included a cohort of patients aged 25 years or less with relapsed or refractory B-ALL and a cohort of patients aged at least 18 years with relapsed or refractory B-cell lymphoma who were eligible for autologous stem cell transplant. Both CAR-T therapies were compared with chemotherapy.

The average sales price for chemotherapy treatments were used and the wholesale acquisition cost was used for tisagenlecleucel ($475,000) and axicabtagene ciloleucel ($373,000).

Results of the comparisons showed a $462,000 price difference with total costs for axicabtagene ciloleucel almost four times greater than the total costs for chemotherapy. Additionally, gains in life-years and quality-adjusted life years (QALYs) were more than double, resulting in an incremental cost-effectiveness ratio of approximately $136,000 per QALY gained and approximately $112,000 per life-year gained.

The report further detailed that the cost-effectiveness of both CAR-T therapies fell below or within commonly cited thresholds of $50,000 to $150,000 per QALY.


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“CAR-T therapies represent a critical advancement in treating B-cell malignancies,” said Dan Ollendorf, PhD, chief scientific officer, ICER, in a statement (February 21, 2018). “Based on the evidence currently available, these therapies provide important clinical benefits and appear to be priced in alignment with these benefits.”

Dr Ollendorf acknowledged that the evidence to support this claim is limited and short-term. Affordability questions may still arise that will require innovative solutions, he explained.—Zachary Bessette