Objectives: The results of a recent single-arm trial (ZUMA-5) of axicabtagene ciloleucel (axi-cel) for relapsed/refractory (r/r) FL demonstrated high rates of durable response and tolerable toxicity among treated patients. To quantify the value of axi-cel compared to standard of care (SOC) to manage r/r FL patients who have had at least two prior lines of systemic therapy (3L+), a cost-effectiveness model was developed from a US third-party payer perspective.
Methods: A three-state partitioned survival cost-effectiveness model was developed with a lifetime horizon. Patient-level analyses of the 36-month ZUMA-5 (axi-cel) and SCHOLAR-5 (SOC) studies were used to extrapolate progression-free and overall survivals. After 5 years of survival, an estimated 40% of the modeled population was assumed to experience long-term remission based on literature. Results include the incremental cost-effectiveness ratio (ICER) measured as incremental cost per quality-adjusted life year (QALY) gained. One-way sensitivity analysis (OWSA), probabilistic sensitivity analysis (PSA), and scenario analyses were performed. All outcomes were discounted 3% per year.
Results: Axi-cel led to an increase of 4.28 life-years, 3.64 QALYs and a total cost increase of $321,192 relative to SOC, resulting in an ICER of $88,300 per QALY. Across all parameters varied in the OWSA, the ICER varied between $133,030 and $67,277. In the PSA, axi-cel had a 99% probability of being cost-effective across 5,000 iterations using a $150,000 willingness-to-pay threshold.
Conclusions: Given the robustness of the model results and sensitivity analyses, axi-cel is expected to be a cost-effective treatment in 3L+ r/r FL.
Keywords: CAR-T; cost-effectiveness; economic analysis; immunotherapy.
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