COST-EFFECTIVENESS OF BLINATUMOMAB FOR PEDIATRIC HIGH-RISK RELAPSED B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA IN BRAZIL: A REAL-WORLD ECONOMIC EVALUATION
Author(s)
GUTEMBERG GUEDES MONTE, PhD Candidate1, Mariana Michalowski, MD, PhD2, Ciliana Rechenmacher, PhD2, Liane Daudt, MD, PhD2.
1Postgraduate Program in Child and Adolescent Health, Federal University of Rio Grande do Sul, Porto Alegre, RS, Brazil, 2Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.
1Postgraduate Program in Child and Adolescent Health, Federal University of Rio Grande do Sul, Porto Alegre, RS, Brazil, 2Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.
OBJECTIVES: To evaluate the cost-effectiveness of blinatumomab compared with high-risk consolidation chemotherapy (HC3) for pediatric patients with high-risk first-relapse B-cell acute lymphoblastic leukemia (B-ALL) from the perspective of the Brazilian Unified Health System (SUS).
METHODS: A partitioned survival model with three health states (event-free survival, post-event survival, and death) was developed using a lifetime horizon. Clinical efficacy data were obtained from the randomized phase III trial by Locatelli et al. Survival outcomes were extrapolated using parametric models under the proportional hazards assumption. Real-world direct medical costs were estimated using data from pediatric patients treated at a Brazilian tertiary public hospital. Health-state utilities were derived from published literature. Costs and outcomes were discounted at 5% annually according to Brazilian guidelines. Deterministic and probabilistic sensitivity analyses (5,000 Monte Carlo simulations) were performed.
RESULTS: Blinatumomab increased total costs by R$340,490.07 versus chemotherapy but generated gains of 3.37 life-years and 3.22 quality-adjusted life-years (QALYs). The incremental cost-effectiveness ratio was R$105,742.26 per QALY gained, remaining below commonly adopted willingness-to-pay thresholds in Brazil. Sensitivity analyses identified blinatumomab acquisition cost and event-free survival hazard ratios as key drivers of uncertainty. Probabilistic analyses demonstrated a high probability of cost-effectiveness across conventional Brazilian thresholds.
CONCLUSIONS: From the SUS perspective, blinatumomab was cost-effective compared with consolidation chemotherapy for pediatric high-risk relapsed B-ALL in Brazil. These findings support the adoption of innovative immunotherapies in middle-income healthcare systems and highlight the importance of incorporating real-world evidence into health technology assessment in pediatric oncology.
METHODS: A partitioned survival model with three health states (event-free survival, post-event survival, and death) was developed using a lifetime horizon. Clinical efficacy data were obtained from the randomized phase III trial by Locatelli et al. Survival outcomes were extrapolated using parametric models under the proportional hazards assumption. Real-world direct medical costs were estimated using data from pediatric patients treated at a Brazilian tertiary public hospital. Health-state utilities were derived from published literature. Costs and outcomes were discounted at 5% annually according to Brazilian guidelines. Deterministic and probabilistic sensitivity analyses (5,000 Monte Carlo simulations) were performed.
RESULTS: Blinatumomab increased total costs by R$340,490.07 versus chemotherapy but generated gains of 3.37 life-years and 3.22 quality-adjusted life-years (QALYs). The incremental cost-effectiveness ratio was R$105,742.26 per QALY gained, remaining below commonly adopted willingness-to-pay thresholds in Brazil. Sensitivity analyses identified blinatumomab acquisition cost and event-free survival hazard ratios as key drivers of uncertainty. Probabilistic analyses demonstrated a high probability of cost-effectiveness across conventional Brazilian thresholds.
CONCLUSIONS: From the SUS perspective, blinatumomab was cost-effective compared with consolidation chemotherapy for pediatric high-risk relapsed B-ALL in Brazil. These findings support the adoption of innovative immunotherapies in middle-income healthcare systems and highlight the importance of incorporating real-world evidence into health technology assessment in pediatric oncology.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
EE57
Topic
Economic Evaluation
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology, SDC: Pediatrics, SDC: Rare & Orphan Diseases