OBJECTIVES: The combination of biomarkers and drugs represents an opportunity to improve effectiveness and costs of treatment for solid tumours thus also attracting growing interest of regulators, physicians and companies. We aimed to describe literature evidence about the cost-effectiveness and cost-utility of biomarkers use in solid tumours as tools for customizing immunotherapy to identify what further research needs.

METHODS: A systematic review of the literature was carried out according to the PRISMA statement guidelines. PubMed and Embase were queried from 2010 to 2020. The PICO Model was applied: patients with solid tumours treated with immunotherapy were target population (P); the use of predictive biomarkers was the intervention (I); the comparators (C) were any other strategies; the outcomes (O) were expressed in terms of cost-effectiveness, cost-utility, net-monetary benefit, life years gained and quality of life. This review was prospectively registered in PROSPERO (CRD42020201549).

RESULTS: 524 records were identified from Embase and Pubmed. After the abstracts and full-text screenings, 35 articles met the inclusion criteria. 74% of them are cost-effectiveness analysis, and more than half of those analyses were based on a Markov model. Among patients diagnosed with non-small cell lung cancer (NSCLC), nivolumab was not cost-effective (CE) unless combined with previous biomarker test (PDL-1). Treatment with pembrolizumab with previous PDL-1 test is CE compared to chemotherapy. Nivolumab and pembrolizumab were not CE over chemotherapy for head/neck cancers (HNCs) also with previous biomarker test.

CONCLUSIONS: The use of predictive biomarkers increases cost-effectiveness of immunotherapy in solid tumours. The identification of new biomarkers can be an auxiliary tool for early detection of solid tumours, opening up promising new targets in therapeutic intervention strategies that are sustainable for the healthcare system.