Cost-Effectiveness of Proton Versus Photon Therapy in Pediatric Medulloblastoma Treatment: A Patient Volume-Based Analysis

Background: Proton radiation therapy offers advantages over photon therapy, assisting with severe side effect avoidance. Pediatric patients with medulloblastoma have demonstrated benefit from this technology in recently published cohort studies. Objectives: To compare the costs and benefits between proton and photon therapy in treating pediatric medulloblastoma. Methods: The model was built with a lifetime horizon from the Brazilian health system perspective using a 3% discount rate. A microsimulation model was developed after a literature search, comparing scenarios of equipment life span and number of patients treated per year (50, 100, and 150 patients with 10, 25, and 20 years of equipment life span). The baseline parameters were 50 patients treated annually and 20 years of equipment life span. Results: The quality-adjusted life-year gain was 2.71, and the average incremental cost-effectiveness ratio was $34 590.54 per quality-adjusted life-year. For the willingness-to-pay threshold of 1 gross domestic product per capita, it was observed that the incorporation of the technology would be cost-effective if more than 150 patients were treated per year. The weight of the equipment life span and other variables was limited when it varied in the sensitivity analysis, without significant changes to the model results. Conclusions: Proton therapy is not cost-effective for pediatric medulloblastoma treatment from the Brazilian health system perspective. The investment is not worth when considering the number of potential patients and the country dimensions.