NAXITAMAB FOR THE TREATMENT OF PEDIATRIC PATIENTS WITH RELAPSED OR REFRACTORY HIGH-RISK NEUROBLASTOMA - A COST-EFFECTIVENESS ANALYSIS FROM THE BRAZILIAN PRIVATE HEALTHCARE SYSTEM PERSPECTIVE
Author(s)
Adalton G. Ribeiro, Sr., Master1, Fernanda Gimenes, Master1, Deborah Rigo, Master2, Gabriel Leonel Marasco, Master3, Carolina Padula, Master4.
1Market Access, Adium Pharma, São Paulo, Brazil, 2Market Access, Adium Pharma, SÃO PAULO, Brazil, 3Health Economics, Origin Health, Santo André, Brazil, 4Health Economics, ORIGIN Health, Rio de Janeiro, Brazil.
1Market Access, Adium Pharma, São Paulo, Brazil, 2Market Access, Adium Pharma, SÃO PAULO, Brazil, 3Health Economics, Origin Health, Santo André, Brazil, 4Health Economics, ORIGIN Health, Rio de Janeiro, Brazil.
OBJECTIVES: To assess the cost-effectiveness of naxitamab versus dinutuximab beta in pediatric patients (≥1 year) with relapsed or refractory high-risk neuroblastoma in the Brazilian private healthcare system.
METHODS: A partitioned survival model with three health states (progression-free, progressive disease, and death) was developed over a 17-year horizon using 28-day cycles. Clinical data were obtained from Trial 201 (naxitamab) and APN311-303/304 (dinutuximab beta). Overall survival (OS) and progression-free survival (PFS) were extrapolated using parametric models. Direct medical costs (BRL, March 2026) included drug acquisition, monitoring, disease management, and end-of-life care. Outcomes included life-years (LYs), quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). A 5% annual discount was applied. Scenario analyses assessed parameter uncertainty.
RESULTS: Naxitamab increased survival and QALYs versus dinutuximab beta, with higher costs. Compared with APN311-303, incremental gains were 2.97 LYs and 1.34 QALYs, with an ICER of BRL 38,819/QALY. Compared with APN311-304, gains were 2.97 LYs and 1.01 QALYs, with an ICER of BRL 176,391/QALY. Drug acquisition was the main cost driver, while survival improvement drove incremental benefits. Results were sensitive to drug price and long-term survival assumptions.
CONCLUSIONS: Naxitamab provides meaningful clinical benefits in a population with high unmet need. Its cost-effectiveness depends on pricing and willingness-to-pay thresholds, supporting consideration of managed entry agreements in the Brazilian private healthcare setting.
METHODS: A partitioned survival model with three health states (progression-free, progressive disease, and death) was developed over a 17-year horizon using 28-day cycles. Clinical data were obtained from Trial 201 (naxitamab) and APN311-303/304 (dinutuximab beta). Overall survival (OS) and progression-free survival (PFS) were extrapolated using parametric models. Direct medical costs (BRL, March 2026) included drug acquisition, monitoring, disease management, and end-of-life care. Outcomes included life-years (LYs), quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). A 5% annual discount was applied. Scenario analyses assessed parameter uncertainty.
RESULTS: Naxitamab increased survival and QALYs versus dinutuximab beta, with higher costs. Compared with APN311-303, incremental gains were 2.97 LYs and 1.34 QALYs, with an ICER of BRL 38,819/QALY. Compared with APN311-304, gains were 2.97 LYs and 1.01 QALYs, with an ICER of BRL 176,391/QALY. Drug acquisition was the main cost driver, while survival improvement drove incremental benefits. Results were sensitive to drug price and long-term survival assumptions.
CONCLUSIONS: Naxitamab provides meaningful clinical benefits in a population with high unmet need. Its cost-effectiveness depends on pricing and willingness-to-pay thresholds, supporting consideration of managed entry agreements in the Brazilian private healthcare setting.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE101
Topic
Economic Evaluation, Epidemiology & Public Health, Health Technology Assessment
Disease
Oncology, Pediatrics, Rare & Orphan Diseases