Evaluating the Cost-Effectiveness of Dabrafenib and Trametinib for the Treatment of Pediatric Patients With a BRAF V600E Mutation Low-Grade Glioma in England and Wales.
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OBJECTIVES: Pediatric low-grade gliomas are rare diseases, but they remain the most common pediatric brain tumor and pose a significant burden to children, adolescents, and their families. The combination (D+T) of dabrafenib and trametinib was assessed in the TADPOLE trial in pediatric patients with glioma with a BRAF V600E mutation. The aim of this analysis is to assess the cost-effectiveness of D+T compared with current clinical management in pediatric patients with BRAF V600E mutation-positive low-grade gliomas who require systemic treatment in England and Wales. METHODS: The economic evaluation uses an individual-based state-transition model, whereby simulated patients move through a series of progression health states. Efficacy data were taken from the TADPOLE trial, supplemented by external data. Utility values and healthcare resource use were obtained from the literature, supplemented by clinical opinion. The analysis was conducted from the perspective of the National Health Services and Personal Social Services over a lifetime horizon and in line with the National Institute of Clinical Excellence reference case. RESULTS: The probabilistic incremental cost per quality-adjusted life-year (QALY) gained was £26 606 after QALY weighting, with an 70.7% probability of D+T being a cost-effective treatment option at a £30 000/QALY gained willingness-to-pay threshold. Results were robust to changes in sensitivity analysis. CONCLUSIONS: Gliomas can be devastating for children and their families. In addition to providing a cost-effective option, treatment with D+T allows patients to be managed away from the hospital and so may help alleviate National Health Services capacity issues.