For patients with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) and diffuse large B-cell lymphoma (DLBCL), the availability of chimeric antigen receptor T-cell (CAR-T) therapies represents an unprecedented advancement in treatment. The US Food and Drug Administration (FDA) approved tisagenlecleucel for the treatment of B-ALL in patients aged 0 to 25 years in 2017 and for use in adult patients with DLBCL in 2018. In addition, axicabtagene ciloleucel received FDA approval in 2018 for use in adults with DLBCL.1

Although these innovative therapies have shown favorable response rates in clinical trials, a single infusion can cost nearly $500,000. Thus, despite the demonstrated benefit of these drugs, it is unclear whether they are “priced in alignment with their benefits and if innovation in payment models will address short-term affordability concerns of these promising and potentially curative treatments,” according to a review article published in the Journal of Clinical Oncology.1

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Melanie D Whittington, PhD, of the University of Colorado Anschutz Medical Campus in Aurora, and colleagues reviewed evidence pertaining to the clinical and economic value of CAR-T therapies, as well as payment considerations associated with these agents.

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Clinical Findings

In 75 pediatric and young adult B-ALL patients who received a tisagenlecleucel infusion in the phase 2 single-cohort ELIANA study ( Identifier: NCT02435849), the overall 3-month remission rate (the primary end point) was 81%. At 6 months, event-free and overall survival rates were 73% (95% CI, 60%-82%) and 90% (95% CI, 81%-95%), respectively. At 12 months, these rates were 50% (95% CI, 35%-64%) and 76% (95% CI, 63%-86%), respectively. Transient grade 3 or 4 adverse events occurred in 73% of participants, cytokine release syndrome occurred in 77% of patients, and neurologic events occurred in 40% of patients.2

In the phase 1/2 ZUMA-1 study ( Identifier: NCT02348216), axicabtagene ciloleucel was successfully administered to 101 patients with DLBCL. The primary end point, complete or partial response, was achieved by 83% of patients. Median duration of response and progression-free survival were 11.1 months (95% CI, 4.2-not estimable) and 5.9 months (95% CI, 3.3-15.0), respectively. Grade 3 or higher adverse events occurred in 48% of patients, including cytokine release syndrome in 11% and neurological events in 32%.3

Among 93 patients with DLBCL who received tisagenlecleucel in the phase 2 JULIET study ( Identifier: NCT02445248), the primary end point of complete or partial response was achieved in 52% of cases, and 1-year rates of progression-free and overall survival were 65% and 49%, respectively. Adverse events affected 22% of patients, and cytokine release syndrome of any grade occurred in 58% of patients.4