OBJECTIVES: Cystic fibrosis (CF) is a rare, progressive disorder affecting the lungs, pancreas, and other organs. Approximately 40,000 people in the US live with CF. We aimed to identify comprehensive evidence on the economic burden of CF in the US.

METHODS: We searched Embase^® and MEDLINE^® databases via Embase.com to identify English-language articles published from 2013 to 2022 reporting the economic burden (cost and resource use) of CF in children and adults.

RESULTS: Among 3,645 records screened, 22 studies were included. The mean annual healthcare cost for commercially and Medicaid-insured children with CF (<17 years; N=4,727) was considerably higher ($82,096) than those without CF (N=14,181; $3,165). Key cost drivers for patients with CF were hospitalizations ($35,617), outpatient visits ($10,634), and outpatient prescriptions ($35,845). The mean (standard deviation) hospital costs for children (N=3,412) identified from Kid’s Inpatient Database (KID) and adults (≥21 years, N=10,258) identified from the National Inpatient Sample (NIS) were $26,249 ($40,593) and $21,601 ($31,997), respectively. Median hospitalization costs for patients with CF with and without liver cirrhosis were $66,543 and $53,365, respectively (p < 0.001). Hospitalization costs were higher for patients with CF with infectious complications than without ($72,292 vs $54,069) and with mental disorders than without ($42,797 vs $34,197). Annual treatment costs for severe, moderate, and mild CF-related pulmonary exacerbations were $119,900, $40,800, and $30,100, respectively (p < 0.001 across all groups). Children with CF experienced significantly more mean annual hospitalizations (range: 0.19–1.25) than those without CF (range: 0.02–0.06; p<0.05). NIS adults had a lower mean length of hospital stay (8.54) compared with KID children (10.51).

CONCLUSIONS: Hospitalizations, drug utilization, and complications were key cost and resource use drivers in patients with CF. Treatment costs increase with disease severity. Further measures are needed to enhance health security for patients with CF and relieve the economic burden.