Socio-economic burden of cystic fibrosis in Europe: a systematic review of cost of illness evidence

BACKGROUND: Cystic Fibrosis (CF) is one of the most prevalent severe genetic diseases, with a high economic impact on healthcare systems due to its complexity. The life expectancy of CF patients has substantially lengthened due to early diagnosis and improvements in therapeutic regimens with Cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Therefore, strategies to ensure equitable access to these treatments should be optimized. Our study aimed to summarize existing literature regarding the costs of CF in Europe. METHODS: A systematic literature review was performed querying three online database (PubMed, Scopus, Web of Science) from January 2013 to March 2023. Studies carried out in European countries, written in English language, and focused on the socio-economic burden of CF were selected. RESULTS: Overall, 11 studies were considered. Across countries, the average total annual cost for a CF patient amounted to €14.174,00-53.256,00, higher for children (€15.771-60.412,00) compared to adults (€15.714-€47.217,00). The largest expenditures were recorded for annual direct healthcare costs, which amounted, per patient, to €9861,00-28.827,00, followed by direct non-healthcare costs (€4512,00-18.329,00). For adults, drugs, tests, and hospitalizations represented the primary cost drivers of direct healthcare costs, while for children, direct non-healthcare costs, especially for caregivers, were predominant. Moreover, indirect costs, whose main expenses were related to productivity loss and early retirement, ranged between €1,094-12,443 per patient per year. CONCLUSIONS: The CF management is associated with relevant costs. CFTR modulators represent an important therapeutic opportunity of reducing the clinical and economic burden of CF. Therefore, new evidence and value-based economic evaluations, that also take into account a social perspective, are needed to support the decision-making process. KEY MESSAGES: • The use of new health economic models is needed to guide the policy-makers in a value-based evaluation of Cystic Fibrosis treatments that take into account their whole value. • The assessment of the socio-economic burden of Cystic Fibrosis could guide the identification of the main actions to be implemented to respond adequately to the real health needs of patients.