ICER Report: Costs of Approved CAR T-Cell Therapies Aligned With Clinical Benefit

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.

In the B-ALL cohort, the tisagenlecleucel arm had a total discounted cost of approximately $667,000 with 10.34 discounted life years (LYs) gained and 9.28 discounted quality-adjusted life years (QALYs) gained. The clofarabine comparator arm had a total discounted cost of approximately $337,000 with discounted LYs and QALYs gained of 2.43 and 2.48, respectively.

With a $330,000 cost difference, incremental results showed that the total cost for tisagenlecleucel was nearly twice that of clofarabine; however, gains in LYs and QALYs were more than 4 times greater, which resulted in an incremental cost-effectiveness ratio of approximately $46,000 per QALY gained an approximately $42,000 per LY gained.

In the B-cell lymphoma cohort, the axicabtagene ciloleucel arm had a total discounted cost of approximately $617,000 with 7.35 discounted LYs gained and 5.87 discounted QALYs gained. In the chemotherapy comparator arm, there was a total discounted cost of approximately $155,000 with discounted LYs and QALYs gained of 3.23 and 2.48, respectively.

The results represent a $462,000 price difference with total costs for axicabtagene ciloleucel approximately 4 times greater than the total costs for chemotherapy. Meanwhile, gains in LYs and QALYs were more than double. This resulted in an incremental cost-effectiveness ratio of approximately $136,000 per QALY gained and approximately $112,000 per LY gained.

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

“CAR T therapies represent a critical advancement in treating B-cell malignancies. Based on the evidence currently available, these therapies provide important clinical benefits and appear to be priced in alignment with these benefits,” said Dan Ollendorf, PhD, chief scientific officer, ICER, in a statement. “However, the evidence is limited and very short-term, and we may still face short-term affordability questions that will require innovative solutions.”

CAR T-cell therapy has emerged as a novel way to treat cancer, genetically reprogramming a patient's own white blood cells to attack tumor cells, and was recently named the American Society of Clinical Oncology's Advance of the Year. Earlier this month, long-term follow-up analyzing the toxic effects and results from a phase 1 clinical trial of adult patients with relapsed B-ALL who were treated with CAR T cells found patients with low disease burden had a longer median overall survival and a lower incidence of toxicity. Final updated results of the pivotal phase 2 study that led to the approval of tisagenlecleucel were published last month, showing an overall remission rate of 81%; 60% of patients had complete remission, and 21% had complete remission with incomplete hematologic recovery.