Dinutuximab Beta Versus Naxitamab in the Treatment of Relapsed/ Refractory Neuroblastoma – Matching-Adjusted Indirect Treatment Comparison and Economic Evaluation in the Private Health Insurance System in Brazil
Author(s)
Przemyslaw Holko, PhD1, Katarzyna Sladowska, PhD1, Pawel Kawalec, PhD, MD2, Antônio Gaspar, BSc3, Helder Paiva, PhD4.
1Jagiellonian University Medical College, Kraków, Poland, 2Professor, Jagiellonian University, Kraków, Poland, 32Heads in Health, São Paulo, Brazil, 4Recordati Rare Diseases, São Paulo, Brazil.
1Jagiellonian University Medical College, Kraków, Poland, 2Professor, Jagiellonian University, Kraków, Poland, 32Heads in Health, São Paulo, Brazil, 4Recordati Rare Diseases, São Paulo, Brazil.
OBJECTIVES: There are no head-to-head trials comparing anti-GD2 immunotherapies dinutuximab beta (DB) and naxitamab (NAX) in the treatment of relapsed/refractory neuroblastoma (NB). We aimed to indirectly compare efficacy and cost-effectiveness of the two therapies.
METHODS: Systematic literature review was conducted to identify evidence for matching-adjusted indirect comparison (MAIC). Individual patient data from DB trials matching NAX licensed population were included and adjusted for predictors of outcomes: MYCN-A, relapsed vs refractory disease, and sex. Semi-Markov cost-effectiveness model was built based on parametric fits to progression free survival (PFS) data with overall survival estimated from time from progression to death. Frequency of grade 3+ adverse events was assumed to be comparable. Dosing of DB and NAX was based on clinical trial regimens. Half of the patients were assumed to be treated with one of the anti-GD2 immunotherapies following subsequent relapse. Brazilian private health insurance costs and 2025 drug list prices were used. Utilities obtained from literature and costs were discounted at 5% over lifetime horizon.
RESULTS: Aggregated NAX data from Study 201 (N=52) and individual patient data from DB Studies APN311-304 and APN311-202 (N=77) met the inclusion criteria. Compared to NAX, DB significantly improved PFS (HR=0.48, 95% CI: 0.28; 0.83, p=0.009) with 2.87 additional years (95% CI: 1.39; 4.33) and 2.68 additional QALYs. NAX was 2,832,867 BRL more costly and was dominated by DB. Results were most sensitive to price of anti-GD2 therapy and the number of cycles of NAX. With NAX use restricted to 6 cycles, cost saving was 1,004,305 BRL. In a sensitivity analysis with subsequent relapse not treated with anti-GD2 immunotherapy, the cost saving with DB was 2,619,040 BRL.
CONCLUSIONS: Compared to naxitamab, dinutuximab beta improves survival in patients with relapsed/refractory neuroblastoma and is cost-saving in private health insurance system in Brazil.
METHODS: Systematic literature review was conducted to identify evidence for matching-adjusted indirect comparison (MAIC). Individual patient data from DB trials matching NAX licensed population were included and adjusted for predictors of outcomes: MYCN-A, relapsed vs refractory disease, and sex. Semi-Markov cost-effectiveness model was built based on parametric fits to progression free survival (PFS) data with overall survival estimated from time from progression to death. Frequency of grade 3+ adverse events was assumed to be comparable. Dosing of DB and NAX was based on clinical trial regimens. Half of the patients were assumed to be treated with one of the anti-GD2 immunotherapies following subsequent relapse. Brazilian private health insurance costs and 2025 drug list prices were used. Utilities obtained from literature and costs were discounted at 5% over lifetime horizon.
RESULTS: Aggregated NAX data from Study 201 (N=52) and individual patient data from DB Studies APN311-304 and APN311-202 (N=77) met the inclusion criteria. Compared to NAX, DB significantly improved PFS (HR=0.48, 95% CI: 0.28; 0.83, p=0.009) with 2.87 additional years (95% CI: 1.39; 4.33) and 2.68 additional QALYs. NAX was 2,832,867 BRL more costly and was dominated by DB. Results were most sensitive to price of anti-GD2 therapy and the number of cycles of NAX. With NAX use restricted to 6 cycles, cost saving was 1,004,305 BRL. In a sensitivity analysis with subsequent relapse not treated with anti-GD2 immunotherapy, the cost saving with DB was 2,619,040 BRL.
CONCLUSIONS: Compared to naxitamab, dinutuximab beta improves survival in patients with relapsed/refractory neuroblastoma and is cost-saving in private health insurance system in Brazil.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE39
Topic
Economic Evaluation
Topic Subcategory
Trial-Based Economic Evaluation
Disease
SDC: Oncology, SDC: Pediatrics, SDC: Rare & Orphan Diseases