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