Economic Evaluation of Dinutuximab Beta in the Maintenance Therapy of High-Risk Neuroblastoma in Brazil
Author(s)
Antônio Gaspar, BSc1, HELDER Henrique PAIVA, Sr., MASc, MSc, PharmD, PhD2, Fabian Schmidt, MA, MBA3, Jaro Wex, PhD, MD4.
1Heads in Health, São Paulo, Brazil, 2Recordati Rare Diseases, São Paulo, Brazil, 3Recordati Rare Diseases Sarl, Puteaux, France, 4Recordati Rare Diseases, Hemel Hempstead, United Kingdom.
1Heads in Health, São Paulo, Brazil, 2Recordati Rare Diseases, São Paulo, Brazil, 3Recordati Rare Diseases Sarl, Puteaux, France, 4Recordati Rare Diseases, Hemel Hempstead, United Kingdom.
OBJECTIVES: Dinutuximab beta (DB), an anti-GD2 monoclonal antibody, is indicated for immunotherapy of newly diagnosed and relapsed/refractory patients with high-risk neuroblastoma. DB is the current standard of care recommended by international collaborative groups. We sought to evaluate cost-utility of DB from the perspective of the private healthcare system in Brazil.
METHODS: Prospective studies of DB were identified in a systematic literature review. Three-state partitioned survival economic model was built using parametric fits to PFS, EFS and OS data. DB was modelled as long-term infusion (LTI), reported to have superior survival benefit over short-term infusion, versus historical controls (HC). Drug use was based on trial data; state-specific utilities were obtained from literature. Utilities and costs, based on 2025 drug list prices, were discounted at 5% over lifetime horizon. Cure threshold of 10 years was used with accelerated mortality assumed for survivors. No further immunotherapy was modelled following subsequent relapse.
RESULTS: Two studies with matching control groups adjusted for predictors of survival (age, sex, stage, MYCN status) were identified: APN311-302 and (HR-NBL1/SIOPEN) APN311-202. In newly diagnosed patients DB was R$1,164,761 more costly than HC with 13.4 LY gain, 3.7 incremental QALYs and ICER=R$315,493 (€49,974)/QALY. In relapsed patients, the respective results were: R$1,292,440, 14.7 LYG, 4.0 QALYs with ICER R$326,652 (€51,709)/QALY. Probabilistic median ICER was R$324,027 (min. 257,563; max 408,702) in newly diagnosed and R$322,364 (279,300; 389,080) in relapsed patients. ICER was most sensitive to cost of DB (94% of total), efficacy of DB and discount rate for utilities. Using 1.5% discount rate, the respective ICERs were R$160,348 (€25,415)/QALY and R$172,918 (€27,408)/QALY.
CONCLUSIONS: DB as LTI offers good value for money to payers in private healthcare in Brazil both in newly diagnosed and relapsed/refractory patients. As almost all total cost is attributable to DB, the results are driven by price and are broadly applicable to other countries.
METHODS: Prospective studies of DB were identified in a systematic literature review. Three-state partitioned survival economic model was built using parametric fits to PFS, EFS and OS data. DB was modelled as long-term infusion (LTI), reported to have superior survival benefit over short-term infusion, versus historical controls (HC). Drug use was based on trial data; state-specific utilities were obtained from literature. Utilities and costs, based on 2025 drug list prices, were discounted at 5% over lifetime horizon. Cure threshold of 10 years was used with accelerated mortality assumed for survivors. No further immunotherapy was modelled following subsequent relapse.
RESULTS: Two studies with matching control groups adjusted for predictors of survival (age, sex, stage, MYCN status) were identified: APN311-302 and (HR-NBL1/SIOPEN) APN311-202. In newly diagnosed patients DB was R$1,164,761 more costly than HC with 13.4 LY gain, 3.7 incremental QALYs and ICER=R$315,493 (€49,974)/QALY. In relapsed patients, the respective results were: R$1,292,440, 14.7 LYG, 4.0 QALYs with ICER R$326,652 (€51,709)/QALY. Probabilistic median ICER was R$324,027 (min. 257,563; max 408,702) in newly diagnosed and R$322,364 (279,300; 389,080) in relapsed patients. ICER was most sensitive to cost of DB (94% of total), efficacy of DB and discount rate for utilities. Using 1.5% discount rate, the respective ICERs were R$160,348 (€25,415)/QALY and R$172,918 (€27,408)/QALY.
CONCLUSIONS: DB as LTI offers good value for money to payers in private healthcare in Brazil both in newly diagnosed and relapsed/refractory patients. As almost all total cost is attributable to DB, the results are driven by price and are broadly applicable to other countries.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE379
Topic
Clinical Outcomes, Economic Evaluation
Disease
Oncology, Rare & Orphan Diseases