OBJECTIVES: Vaborem is a first-in-class, fixed dose combination of vaborbactam (a novel beta-lactamase inhibitor) and meropenem (a beta-lactam antibiotic) belonging to the carbapenem class with potent activity against target CRE pathogens, optimally developed for Klebsiella pneumoniae carbapenemase (KPC). The study aims to evaluate the cost-effectiveness of Vaborem versus Best Available Therapy (BAT) for the treatment of patients with CRE-KPC associated infections in the Italian setting.

METHODS: The analysis is based on a decision-tree model that simulates the clinical pathway followed by patients with a confirmed CRE-KPC infections in a 5-year time horizon. The National Health System perspective is adopted, and 3% discount rate applied. Vaborem efficacy in terms of cure rate, reduction of nephrotoxicity risk, 28-day mortality is mostly sourced from the Phase 3, randomized, clinical trial (TANGO II). Unit costs are retrieved from the Italian official drug pricing list and legislation, while patient resource use was validated by a national expert. Model outcomes include life years (LYs) and quality-adjusted life years (QALYs) gained, incremental costs, incremental cost-effectiveness ratio (ICER) and incremental cost-utility ratio (ICUR). Deterministic and probabilistic sensitivity analyses have been performed.

RESULTS: Vaborem is expected to decrease the burden associated to treatment failure, septic shock and nephrotoxicity reducing the need for chronic renal replacement therapy, while increasing costs related to drug acquisition and long-term care (due to higher survival). Treatment with Vaborem versus BAT leads to a gain of 0.475 LYs, 0.384 QALYs, and incremental costs of €3,549, resulting in an ICER and ICUR of €7,473/LY and €9,246/QALY, respectively. Results proved robust in sensitivity analyses, probability of being cost-effective reaches 90% when willingness to pay is €15,850/QALY.

CONCLUSIONS: The analysis suggests that Vaborem may be a cost-effective strategy in the Italian setting targeting a highly unmet medical need for the treatment of CRE-KPC infections.