Routine Use of Axicabtagene Ciloleucel Could Be Cost Effective in Relapsed/Refractory Diffuse Large B-Cell Lymphoma

Patients treated with bypassing therapy experienced significantly higher medical and outpatient pharmacy costs.
Patients treated with bypassing therapy experienced significantly higher medical and outpatient pharmacy costs.
Researchers sought to determine whether the use of second-line CAR-T therapy could be cost effective in DLBCL.

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).

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

This article originally appeared on Hematology Advisor