OBJECTIVES: Paediatric High-Grade Gliomas (HGG) are rare diseases and associated with poor prognosis. Options are limited and often palliative. 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. D+T demonstrated to be a well-tolerated and effective therapy to delay progression, improve survival and quality of life compared with historical trials/studies. 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 relapsed/refractory HGG 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 HGG 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 in patients previously exposed or not to temozolomide was £28,575 and £28,186, with a 76% and 70% probability of D+T being a cost-effective treatment option at a £30,000/QALY gained willingness to pay threshold, respectively. 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, and offer patients and their families a better quality of life