OBJECTIVES: High-risk Neuroblastoma is a rare malignant tumor in pediatric patients, 70% of which have a metastatic stage at the time of diagnosis with high rates of refractory/relapse disease after available therapies. Anti-GD2 immunotherapy is emerging as new options for this population with poor prognosis. To demonstrate the economic impact of incorporating the immunotherapy options Naxitamab plus GM-CSF and Beta-dinutuximab for the treatment of refractory/relapsed HR-NB in the private health system of Brazil.

METHODS: A BIM was carried out over a 5-year time horizon and a fixed market rate. For the CEA, a 3-state Markov model, based on published OS and EFS data. Deterministic and Montecarlo-type probabilistic sensitivity analyzes were added, based on the LYs obtained among the immunotherapy options.

RESULTS: According to the incidence model, a cohort of 17 patients was estimated within the horizon defined for the BIM, showing an accumulated cost of R$ 98,299,546.6 and R$ 102,150,478.4 in Naxitamab plus GM-CSF and Beta-dinutuximab arms, respectively. CEA base case showed 3.2 and 1.6 LYs gain, average cost per LYs of R$352,912.5 and R$740,255.8, average treatment costs of R$1,14 and R$1,18 million for Naxitamab plus GM-CSF and Beta-dinutuximab arms, respectively. The variable with the greatest impact in the deterministic analysis was the “probability of death of the Betadinutuximab”. Naxitamab ICER was -R$24,674/LYs with a probability of 0.85 and 0.55 of being cost effective and dominant compared to the competitor with a WTP of 3 GDP per capita after the probabilistic sensitivity analysis.

CONCLUSIONS: Naxitamab plus GM-CSF demonstrated to be a cost-saving and cost-effective option compared to Betadinutuximab for the treatment of refractory/relapsed HR-NB within the private health system in Brazil.