Cost-effectiveness for KTE-X19 CAR T therapy for adult patients with relapsed/refractory mantle cell lymphoma in the United States

Abstract

Aims: The objective of this study is to estimate the cost-effectiveness of KTE-X19 versus standard of care (SoC) in the treatment of relapsed/refractory (R/R) mantle cell lymphoma (MCL) patients from a US healthcare perspective. Materials & Methods: A three-state partitioned-survival model (pre-progression, post-progression and death) with a cycle length of one month was used to extrapolate progression-free and overall survival (OS) over a lifetime horizon. Due to the long tail of the OS curve, OS was modeled applying a mixture-cure methodology, using the assumption that patients whose disease had not progressed after five years experienced long-term remission. Population inputs were derived from the ZUMA-2 trial. This was also the source of KTE-X19 efficacy and safety data, while this data was obtained from the literature for SoC. Costs and resource use inputs were derived from the published literature and publicly available data sources. Health state utilities were derived from the ZUMA-2 trial (NCT02601313), applying the US tariff. Adverse event disutilities were derived from the published literature. Costs and health outcomes were discounted at 3% per year. The model estimated expected life years (LY), quality-adjusted life years (QALY) and total costs for KTE-X19 versus SoC. Deterministic and probabilistic sensitivity analyses were performed. Results: Median survival was 9.71 years for KTE-X19 and 2.13 for SoC.

Capturing the Impact of Constraints on the Cost-Effectiveness of Cell and Gene Therapies: A Systematic Review

“…Thirty-two CEAs of cell (n=20) [33][34][35][36][37][38][39][40][41][42][43][44][45][46][47][48][49][50][51][52] or gene therapies (n=12) [53][54][55][56][57][58][59][60][61][62][63][64] met the inclusion criteria. Table 1 summarises the design of each CEA and whether constraints that affect the cost-effectiveness of treatment were described qualitatively or included quantitatively by each study.…”

Cost-Effectiveness of KTE-X19 for Adults with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia in the United States

“…Based on data from the Cost-Effectiveness Analysis Registry of the Tufts Medical Center and the Institute for Clinical and Economic Review's analysis of CAR-T therapies, CAR-Ts provide significantly greater effectiveness than prior pharmaceutical innovations [38], providing incremental gains of 5.03 QALYs above the average pharmaceutical intervention, addressing a large unmet in patients. Despite their high up-front costs, the cost-effectiveness of CAR-T therapies has [40].…”

“…In pediatric R/R B-cell ALL, CAR-T therapy, compared to standard of care, was found to result in an ICER of $64,600/QALY and was cost-effective in 94.8% of iterations at a WTP of $100,000/QALY[ 39 ]. Simons et al found that KTE-X19 was cost-effective for adult patients with R/R mantle cell lymphoma in the US, with an ICER of $31,985/QALY versus standard of care [ 40 ].…”

Resource utilization for chimeric antigen receptor T cell therapy versus autologous hematopoietic cell transplantation in patients with B cell lymphoma

“…Thus, since policies, procedures, and also outcomes might be different in other institutions or in future scenarios, our results should be interpreted carefully and with appropriate adaptions wherever indicated. In contrast to other analyses on the cost-effectiveness of CAR-T cell therapy that derive value from incremental survival and health-related quality of life benefit [ 11 , 12 , 31 ], we did not perform economic evaluations or budget impact analyses to investigate the economic burden of new therapies to the society and health care system. We also did not consider patient outcomes into the analysis by measurement of so-called quality adjusted life years (QUALYs) per dollar spent [ 12 ].…”