Background Cystic fibrosis (CF) is the most serious and frequent hereditary autosomal disease that causes respiratory, hepatic and pancreatic dysfunction.
Purpose To assess the pharmaceutical cost associated with CF outpatients from the Adult Cystic Fibrosis Unit at a third-level hospital.
Material and methods Retrospective observational study of CF medication in adult patients throughout the year 2017, patients without complete annual monitoring were excluded. Collected data: age, sex, mutation of cystic fibrosis transmembrane conductance regulator (CFTR) gene, forced expiratory volume in 1 s (FEV1), colonisation by Pseudomonas aeruginosa (PA) and drug therapy. Considered costs were laboratory selling price notified in Nomenclator. CFTR modulator drugs and hypertonic 7% sodium chloride solution as master preparation were not considered for overall costs (purchase price was zero). The SPSS program (15.0 version) was used for data analysis.
Results Fifty-nine adult patients were included, 54.2% were female and average age was 32.2 years (±9.2): 35.6% patients were homozygous for F508 deletion, 42.4% were heterozygous and 22.0% had another mutation. The average FEV1 was 72.6%: 55.9% patients were FEV1 ≥70%, 39.0% FEV1=40%–69% and 5.1% FEV1 <40%. In addition, 32.2% were colonised by sensitive PA and 8.5% by PA multidrug-resistant.
The annual cost was €547,085.70 and the median cost was €7,147.58 (IQR=€14,397.72). The average cost in homozygous patients was €12,129.56, in heterozygous it was €8479.80 and for other mutations it was €6,182.32. The average cost distributed by FEV1 groups was: €7,565.09 in patients with FEV1 ≥70%, €117,04.65 for FEV1=40%–69% and €9,410.16 for FEV1 <40%: all differences were statistically significant. The cost difference between patients without infection and with sensitive PA was €77,183.31 and between multidrug-resistant PA patients it was €10,272.82: both differences were statistically significant. High-cost medicines were dornase alfa (Pulmozyme), aztreonam (Cayston) and inhaled tobramycine (Bramitob).
Conclusion Cystic fibrosis is a relatively costly disease, although new CFTR modulator drugs will increase costs considerably. Treatment costs per patient are similar to those reported in the literature. The pulmonary function is related to treatment cost: severe dysfunction means lower expenditure than intermediate function, on account of excluding CFTR modulators. The relationship between treatment adherence and cost should be analyse in further studies.
References and/or acknowledgements https://www.ncbi.nlm.nih.gov/pubmed/28404533
No conflict of interest.
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