CAR-T ‘highly cost-effective’ as second-line treatment for large B-cell lymphoma



Perales MA, et al. Abstract LBA2. Presented at: Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR; April 23-26, 2022; Salt Lake City.

Disclosures: Perales reports consultant roles with or research funding/honoraria from AbbVie, Astellas, Bellicum, Bristol Myers Squibb, Celgene, Incyte, Karyopharm, Kite Pharma/Gilead Sciences, Miltenyi Biotech, Merck, MorphoSys, Nektar Therapeutics, Novartis, Omeros, OrcaBio, Takeda , VictivBio AG and Vor Biopharma. Please see the abstract for all other researchers’ relevant financial disclosures.

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Despite a high upfront price, treatment with axicabtagene ciloleucel appeared more cost-effective as second-line therapy than standard treatment for relapsed or refractory large B-cell lymphoma, according to study results.

Treatment with axicabtagene ciloleucel (Yescarta, Kite Pharma/Gilead Sciences) — a CD19-directed chimeric antigen receptor T-cell therapy — improved survival outcomes at an incremental price difference well below the typical threshold third-party payers often use to determine insurance coverage, findings presented at Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR showed.

Increase in QALYs by treatment type.
Data derived from Perales MA, et al. Abstract LBA2. Presented at: Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR; April 23-26, 2022; Salt Lake City.


The FDA in April expanded the label for axicabtagene ciloleucel to include second-line treatment for adults with large B-cell lymphoma who are refractory to first-line chemoimmunotherapy or who experienced disease relapse within 12 months of initial treatment.

The FDA based the new indication on data from the randomized phase 3 ZUMA-7 trial, which showed axicabtagene ciloleucel — often referred to as axi-cel — significantly improved EFS compared with standard treatment, which consists of platinum-based salvage combination chemotherapy, followed by high-dose therapy and autologous hematopoietic stem cell transplant for those who responded to salvage chemotherapy.

Miguel-Angel Perales, MD

Miguel-Angel Perales

“In the context of the positive results from the ZUMA-7 trial, as well as the recent FDA approval of axi-cel as second-line [therapy]we set out to do a study to estimate the cost-effectiveness of axi-cel from a third-party US payer perspective in the second-line setting,” Miguel-Angel Perales, MD, chief of the adult bone marrow transplant service at Memorial Sloan Kettering Cancer Center, said during a presentation. “The findings from our cost-effectiveness study suggest a sizable improvement in quality and length of life after axi-cel compared with standard of care.”


Perales and colleagues conducted an estimated cost-effectiveness study of axi-cel as second-line therapy for adults with relapsed or refractory large B-cell lymphoma.

“We took the trial data and extrapolated it over a lifetime horizon using established statistical models,” Perales said.

Investigators used a mixture cure modeling approach that included survival curves from the ZUMA-7 trial to extrapolate estimates of OS, EFS and time to next treatment.

They reported results in 2021 US dollars, quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER) — a cost-effectiveness statistic often used to compare different health care interventions across disease states.

Key findings

Researchers projected median OS of 59 months among patients who received axi-cel compared with 25 months for those who received standard treatment. This translated to a gain of 1.34 life-years and 1.37 QALYs for patients treated with axi-cel compared with standard treatment. Seventy-four percent of the additional projected QALYs in the axi-cel group occurred during their EFS state.

Axi-cel treatment cost $103,808 more per patient, but the cost of subsequent lines of therapy among patients who received standard treatment offset this additional amount. Notably, use of CAR-T cell therapy as third-line treatment after disease progression on standard care proved to be the major contributor to the overall increase in cost of subsequent therapy among patients who initially received standard care, researchers concluded.

The analysis yielded an ICER of $75,890/QALYs for axi-cel compared with standard-of-care treatment when considering incremental costs and QALY difference. A subsequent sensitivity analysis showed that axi-cel has a 72% probability of being cost-effective vs. standard therapy at a willingness-to-pay threshold of $150,000/QALY.

Clinical implications

The use of third-line or later CAR T-cell therapy as a subsequent treatment in the model was based on observed use in the ZUMA-7 trial and may not reflect real-world treatment patterns, Perales said. However, similar patterns have been observed in trials of other CAR T-cell therapies in the second-line setting for large B-cell lymphoma, he added.

“The cost offsets in subsequent CAR T-cell therapy use and reductions in disease progression led to a limited incremental cost difference, resulting in a highly cost-effective ICER,” Perales said. “This study suggests that axi-cel is a cost- effective treatment option that can address a critical unmet clinical need for our patients while offering good value.”

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