OBJECTIVES: Cystic fibrosis (CF) is a rare, progressive disorder affecting the lungs, pancreas and other organs, and the most prevalent genetic condition among the Caucasian population. We aimed to identify comprehensive evidence on the economic burden of CF in five key European countries.

METHODS: We systematically searched Embase^® and MEDLINE^® databases via Embase.com for English-language articles published from 2013–2023 reporting the economic burden (cost and resource use) of CF in children and adults in the UK, France, Germany, Italy and Spain.

RESULTS: Among 1,431 records screened, 11 studies were included reporting data for France (4), Italy (3), the UK (2), Germany (1) and all five countries (1). In France, total cost per patient increased sharply between 2006–2017 (mean €14,174 to €44,585). In Germany (2016), mean direct medical cost was €17,551 per patient-year, driven mainly by outpatient drug prescription (€12,869). In Italy, annual total cost per patient increased with age and lung disease severity from €4,164 in children aged ≤5 years to €30,123 in patients aged >5 years. In the UK, the key cost drivers were informal care (44.1 %), medications (14.5 %), acute hospitalizations (13.9 %), early retirement (9.1 %) and outpatient and primary healthcare visits (7.9 %). In Spain, mean total costs per patient was €32,911 in 2012; in children, total mean direct costs varied from €22,666–€60,412. In France, use of ivacaftor (CFTR modulator) reduced hospitalization rate from 0.47 hospitalizations per person-year to 0.22 after treatment. Children had higher direct costs than adults, and direct healthcare costs and resource utilization increased with disease duration.

CONCLUSIONS: Disease duration, hospitalizations and drug utilization are key drivers for cost and resource use in patients with CF. Total costs increased with patients’ level of dependence on others. Further measures are needed to alleviate this economic burden.