Routine use of second-line chimeric antigen receptor T-cell (CAR-T) therapy in patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL) could be cost effective in the United States, but its cost effectiveness would be highly dependent upon long-term outcomes, according to research published in Blood.

The ZUMA-7 (Efficacy of Axicabtagene Ciloleucel Compared to Standard of Care [SOC] Therapy in Subjects With R/R DLBCL) study showed that second-line axicabtagene ciloleucel (axi-cel) had a benefit in event-free survival (EFS; 24-month EFS, 41% with axi-cel vs 16% with SOC; P <.001) in patients with R/R DLBCL, leading to its recent approval by the US Food and Drug Administration.

In this study, researchers modeled cost effectiveness of second-line axi-cel compared with SOC in a hypothetical cohort of US adults (mean age, 65 years) with primary refractory or early relapsed DLBCL. They developed a Markov model using a range of plausible long-term outcomes, estimating EFS and OS from ZUMA-7, and reported outcome measures in incremental cost-effectiveness ratios, with a willingness-to-pay threshold of $150,000 per quality-adjusted life-year (QALY).


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When assuming a 5-year EFS of 35% with second-line axi-cel and 10% with SOC, the team found axi-cel was cost effective at the willingness-to-pay threshold of $150,000 per QALY ($93,547 per QALY). When assuming axi-cel produced a 5-year EFS of ≤26.4% or if it would cost more than $972,061 at the willingness-to-pay threshold of $150,000 per QALY, they found axi-cel was no longer cost effective. The team reported that second-line axi-cel was the cost-effective strategy in 73% of 10,000 model iterations at the willingness-to-pay threshold of $150,000 per QALY.

“These analyses can help provide support for payor coverage and clinicians’ utilization of CAR-T in the second-line setting,” the researchers wrote in their report. “Widespread adoption of CAR-T in the second-line setting will lead to substantially increased costs even in a high-risk subgroup, highlighting the importance of alternative reimbursement models and other efforts to reduce its cost.”

Limitations of the study included lack of long-term follow-up data from ZUMA-7, the ability to model a single CAR-T product only, modeling using primarily the ZUMA-7 study (which had strict inclusion criteria), potential inability to reflect real-world patients population or patient management with model inputs informed by ZUMA-7, probable CAR-T cost variability in different settings, and lack of data on the feasibility of stem cell collection and the efficacy of therapies.

Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures. 

Reference

Kambhampati S, Saumoy M, Schneider Y, et al. Cost-effectiveness of second-line axicabtagene ciloleucel in relapsed refractory diffuse large B-cell lymphoma. Blood. 2022;140(19):2024-2036. doi:10.1182/blood.2022016747