Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade Glioma

Juan David Rios,Eric Bouffet,Liana Nobre,Petros Pechlivanoglou,Derek S Tsang,Uri Tabori,Cynthia Hawkins,Russanthy Velummailum,Julie Bennett,Avram Denburg

doi:10.1186/s12887-021-03069-1

Abstract

BackgroundTreatment personalization via tumor molecular testing holds promise for improving outcomes for patients with pediatric low-grade glioma (PLGG). We evaluate the health economic impact of employing tumor molecular testing to guide treatment for patients diagnosed with PLGG, particularly the avoidance of radiation therapy (RT) for patients with BRAF-fusion.MethodsWe performed a model-based cost-utility analysis comparing two strategies: molecular testing to determine BRAF fusion status at diagnosis against no molecular testing. We developed a microsimulation to model the lifetime health and cost outcomes (in quality-adjusted life years (QALYs) and 2018 CAD, respectively) for a simulated cohort of 100,000 patients newly diagnosed with PLGG after their initial surgery.ResultsThe life expectancy after diagnosis for individuals who did not receive molecular testing was 39.01 (95% Confidence Intervals (CI): 32.94;44.38) years and 40.08 (95% CI: 33.19;45.76) years for those who received testing. Our findings indicate that patients who received molecular testing at diagnosis experienced a 0.38 (95% CI: 0.08;0.77) gain in QALYs and $1384 (95% CI: $-3486; $1204) reduction in costs over their lifetime. Cost and QALY benefits were driven primarily by the avoidance of long-term adverse events (stroke, secondary neoplasms) associated with unnecessary use of radiation.ConclusionsWe demonstrate the clinical benefit and cost-effectiveness of molecular testing in guiding the decision to provide RT in PLGG. While our results do not consider the impact of targeted therapies, this work is an example of the value of simulation modeling in assessing the long-term costs and benefits of precision oncology interventions for childhood cancer, which can aid decision-making about health system reimbursement.

Highlights

Treatment personalization via tumor molecular testing holds promise for improving outcomes for patients with pediatric low-grade glioma (PLGG)
Kaplan-Meier estimates of progression-free survival (PFS) and overall survival (OS) for the SickKids institutional cohort are provided in Supplementary Fig. 1, transition probabilities are provided in Supplementary Fig. 2
We demonstrate the mortality difference between molecular testing and no molecular testing under ‘no radiation benefit’ and ‘radiation therapy (RT) benefit’ scenarios

Summary

Introduction

Treatment personalization via tumor molecular testing holds promise for improving outcomes for patients with pediatric low-grade glioma (PLGG). We evaluate the health economic impact of employing tumor molecular testing to guide treatment for patients diagnosed with PLGG, the avoidance of radiation therapy (RT) for patients with BRAF-fusion. Treatment for patients with PLGG may include surgery, chemotherapy, radiation therapy (RT), or targeted therapies, which may. Several studies have demonstrated the benefit of RT in improving progression-free survival (PFS) in patients with PLGG [6,7,8,9,10,11]. Patients with central nervous system tumors, of which PLGGs are the most prevalent, are among the most expensive to manage [15], especially when RT is included in the treatment regimen [16]

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