Abstract
BACKGROUND: Acute lymphoblastic leukemia (ALL) is the most common malignancy of childhood. With the introduction of novel cellular therapies, cost of care is a critical component and financial toxicity experienced by patients and society requires evaluation. This study aims to assess the total cost of CAR-T therapy for pediatric ALL patients with commercial insurance coverage in the United States.
STUDY DESIGN: Using de-identified commercial insurance data from the OptumLabs® Data Warehouse, a cohort of 37 patients, aged 1-25 years, with B-ALL treated with CAR-T therapy between Oct 2016 and Dec 2021 in the United States was identified. Cost was evaluated for a 90 day period encompassing CAR-T infusion and by administration and complication characteristics.
RESULTS: Among the 37 identified ALL patients that received a CAR-T product infusion, 14 patients were female, median age at administration was 13 years. The median 90-day total cost was $620,500 (Mean: $589,108). Inpatient cost accounted for approximately 71% of the total cost with an average of 28 inpatient days per patient. Although inpatient cost was slightly higher in the older age group (aged 10-25 years) and in patients with a code for CRS, these differences were not statistically significant.
CONCLUSION: This real-world cost analysis shows for the first time the encompassing cost of CAR-T therapy for pediatric ALL patients in the US with commercial insurance. This study provides a valuable benchmark that can be used to analyze the financial implications of CAR-T therapy for pediatric ALL therapy on health systems.
Introduction
Acute lymphoblastic leukemia (ALL) is the most common cancer of childhood and adolescence, with approximately 3000 new cases diagnosed each year in individuals under age 18 years of age in the United States (US).1 Approximately 85% of pediatric ALL expresses B-cell lineage cell-surface and cytoplasmic markers (B-ALL).2 Over the last five decades, survival of children with B-ALL has improved drastically from less than 10% in the 1960s to over 90% today.2,3 However, relapsed and refractory B-ALL remains a significant and challenging issue with 5-year survival for patients with relapsed disease ranging from 40-60%.4-7
Chimeric antigen receptor (CAR) T-cells targeting CD19 on B-ALL leukemic blasts have changed the landscape of treatment for relapsed and refractory disease. The landmark ELIANA trial that led to the FDA approval of Novartis’s tisagenlecleucel (Kymriah), a CD19 CAR-T product, showed an 82% rate of complete remission (CR) or CR with incomplete hematologic recovery (CRi) in 79 infused children, adolescents, and young adults.8,9 Among 65 responders, the median relapse-free survival (RFS) was 43 months and 5-year RFS rate was 44% (95% confidence interval (CI), 31%-56%).10 In addition to prompting FDA approval for tisagenlecleucel, this trial and subsequent studies of the real world efficacy of this therapy have shown that CAR-T therapy can be a curative treatment option for heavily pretreated pediatric and young adult patients with relapsed or refractory B-ALL.11-14
Upon FDA approval for tisagenlecleucel in 2017, the product was priced at $475,000 for commercial use in the United States.15 Notably however, this price tag does not capture the cost to payers for the full course of treatment for patients who receive this therapy. Total cost of the therapy includes that of leukapheresis, lymphodepleting chemotherapy, treatment of complications such as cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) and hospitalization for necessary supportive care.16 In the ELIANA trial, 77% of patients experienced some degree of CRS and 48% received tocilizumab,8 an IL-6 monoclonal antibody that also contributes to the total cost of care outside the listed price of tisagenlecleucel.17
There are limited data regarding the comprehensive cost of delivering commercial cellular therapy in the US. Previous studies have been limited to cost-effectiveness analyses based on listed drug prices or have used algorithms or proxies as estimates for cost.17-21 There are also multiple studies investigating the cost of CAR-T therapy delivery in other countries’ health systems,22-29 however studies of comprehensive real-life cost of cellular therapy for pediatric B-ALL are not available in the US.
Previously, our group utilized commercial insurance claims data to investigate the utilization and cost of pediatric ALL care in newly diagnosed patients in the US.30 With rising US healthcare costs and the profound financial burden of childhood cancer to patients and society,31 it is imperative that we understand the current cost of treatment so that the incremental cost and benefit of future therapeutic modifications can be considered. The aim of this study was to use administrative claims data to evaluate the real-world comprehensive cost of CAR-T therapy for B-ALL in the US, for a cohort of commercially insured pediatric patients.
Materials and Methods