OBJECTIVES: Paediatric Low-Grade Gliomas (LGG) are rare diseases and pose a significant burden to children and young adults and their families. The combination (D+T) of dabrafenib (Finlee®) and trametinib (Spexotras®) was assessed in the TADPOLE trial in paediatric patients with glioma with a BRAF V600E mutation. The presence of the mutation is associated with poor outcomes and an increased risk of transformation to high-grade glioma. The aim of this analysis is to assess the cost-effectiveness of D+T compared with current clinical management in paediatric patients with BRAF V600E mutation positive LGG 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 was 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 NHS and Personal Social Services over a lifetime horizon.

RESULTS: Owing to the severity of the disease, paediatric patients with LGG experience a substantial quality-adjusted life-year (QALY) shortfall compared with the general population, and therefore this indication met the criteria for decision modifiers for severity of disease set out by NICE. The probabilistic incremental cost per QALY gained was £26,630, with an 87.6% 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 family. 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 NHS capacity issues, reduce the burden of adverse events compared with chemotherapy, and offer patients and their families a better quality of life.