LAUSR

The ZUMA-7 study demonstrated that Axicabtagene ciloleucel (axi-cel) improved event-free survival (EFS) compared to standard of care (SOC) salvage chemoimmunotherapy followed by autologous stem cell transplant in primary refractory/early relapsed diffuse large B-cell lymphoma (DLBCL) leading to its recent FDA approval in this setting. We modeled a hypothetical cohort of US adults (mean age, 65 years) with primary refractory/early relapsed DLBCL by developing a Markov model (lifetime horizon) to model the cost-effectiveness of second-line axi-cel compared to SOC using a range of plausible long-term outcomes. EFS and OS were estimated from ZUMA-7. Outcome measures were reported in incremental cost-effectiveness ratios, with a willingness-to-pay (WTP) threshold of $150,000/quality-adjusted life-year (QALY). Assuming a 5-year EFS of 35% with second-line axi-cel and 10% with SOC, axi-cel was cost-effective at a WTP of $150,000/QALY ($93,547/QALY). Axi-cel was no longer cost-effective if its 5-year EFS was ≤ 26.4% or if it cost more than $972,061 at a WTP of $150,000. Second-line axi-cel was the cost-effective strategy in 73% of the 10,000 Monte-Carlo iterations at a WTP of $150,000. If the absolute benefit in EFS is maintained over time, second-line axi-cel for aggressive RR-DLBCL is cost-effective when compared to SOC at WTPs of $150,000/QALY. However, its cost-effectiveness is highly dependent on long-term outcomes. Routine usage of second-line CAR-T would add significantly to healthcare expenditures in the USA (>$1 billion each year), even when used in a high-risk subpopulation. Further reductions in the cost of CAR-T are needed to be affordable in many regions of the world.