OBJECTIVES : Precision diagnostic testing (PDT) employing appropriate biomarkers identifies patients who will best respond to precision medicines (PM) including targeted agents and immuno-oncology (IO) drugs. To date, there has been no published systematic appraisal of economic evaluations of PDT/PM in non-small cell lung cancer (NSCLC).

METHODS : We conducted a systematic review using Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA)-directed searches of key databases (2000-2019). Four reviewers screened, quality-assessed, and extracted data, using Consolidated Health Economic Evaluation Reporting Standards (CHEERS) methodology.

RESULTS : Of a total of 68 studies assessed for eligibility, 27 PM studies (40%) were included for quantitative assessment/qualitative narrative synthesis, while 41 (60%) were excluded for failing population, intervention, comparator, outcomes, and study design (PICOS) criteria. These included 6 PDT studies, which failed PICOS outcomes criteria. Twenty studies compared biomarker-guided PM to chemotherapy [epidermal growth factor receptor (EGFR), n=13; anaplastic lymphoma kinase (ALK), n=2; programmed death-ligand 1 (PD-L1), n=5)]. Seven studies compared biomarker-guided PM to non-guided therapy (EGFR, n=6; PD-L1, n=1).

Employing a US$100,000 willingness-to-pay cost-effectiveness threshold, biomarker-guided IO drugs were cost-effective compared to non-guided approaches, for six scenarios from one study (incremental cost-effective ratios (ICER) mean: $40,705). IO drugs were cost-effective in 40% of studies (overall ICER mean: $130,519, range: $78,050-$181,815) compared to chemotherapy alone. EGFR-directed therapy was cost-effective in 67% of studies (ICER mean: $57,051) compared to non-guided therapy and cost-effective in 75% of studies (ICER mean: $58,685) compared to chemotherapy alone. ALK-guided therapy was cost-effective in 50% of studies (ICER mean: $89,219), compared to chemotherapy alone.

CONCLUSIONS : Generally, cost-effectiveness was superior for biomarker-guided PM compared to non-guided therapy or chemotherapy. However, while PDT is critical in selecting appropriate PM interventions, ~30% of economic evaluations, including 100% of PDT evaluations, were of poor quality or missing vital data, compromising robust inter-study economic analysis.