OBJECTIVES: Biomarker testing is indispensable for the implementation of precision medicine (PM) in oncology. The aim of this study was to demonstrate the value of biomarker testing from a holistic perspective based on the example of advanced non-small cell lung cancer (aNSCLC).

METHODS: A partitioned survival model was populated with data from pivotal clinical trials of 27 first-line treatments in aNSCLC. Three testing scenarios were considered; "no biomarker testing" encompassing chemotherapy treatment, "sequential testing" for EGFR and ALK encompassing treatment with targeted- or chemotherapy, and "multigene testing" covering EGFR, ALK, ROS1, BRAF, NTRK, MET, RET, KRAS, HER2, PIK3CA and encompassing treatment with targeted- or immunotherapy. Analyses were run for nine countries across all continents. A one-year and five-year time horizon was applied. Information on test accuracy was combined with country-specific information on epidemiology and unit costs.

RESULTS: Compared to the no-testing scenario, all health outcomes improved with increased testing. One- and five-year survival rates increased from 51% and 2% to 60–68%/70–80% and 5–7%/12–18% in scenarios two and three, respectively. The highest rates were observed in East Asia due to a higher prevalence of targetable mutations among patients. Costs for testing and medicines also increased in these scenarios, while other direct costs (including administration, adverse events, and terminal care costs) decreased throughout all years. Non-health care costs (sick leave and disability payments) decreased during the first year but increased over a five-year time horizon due to increased survival.

CONCLUSIONS: Use of PM in aNSCLC leads to increased costs for testing and medicines. Other direct costs are decreased, notably for treatment of serious adverse events. Non-health care costs decrease initially but increase over time. This is a result of more efficient treatment assignment leading to improved health outcomes, in particular prolonged progression-free disease phase and overall survival.