The clinical, psychosocial, and economic burden of cystic fibrosis lung disease in the era of CFTR modulator therapy.

Cystic fibrosis (CF) lung disease imposes a significant clinical, psychosocial, and economic burden on people with CF (pwCF), their caregivers, and healthcare systems. Although the introduction of CF transmembrane conductance regulator (CFTR) modulator therapies has led to significant improvements in symptoms, lung function, exacerbations, and quality of life, substantial burden remains. A subset of pwCF taking CFTR modulator therapy experience residual infection, neutrophilic inflammation (to levels seen in non-CF bronchiectasis), exacerbations, and pulmonary complications. Furthermore, 10%-15% of the global CF population is either ineligible for or intolerant to current CFTR modulator therapies, with some pwCF (although in the minority) experiencing adverse events that necessitate treatment discontinuation. For these people, the burden of disease remains. The worsening of mental health experienced by some pwCF on CFTR modulator therapy adds to the psychosocial burden. Although some evidence suggests a decrease in treatment burden, in general, CFTR modulators have been added to therapeutic regimens rather than replacing symptomatic treatments. While reductions in healthcare resource use have been reported, hospitalizations and emergency department visits, and their associated costs, have not been eliminated. Given the expected improvements in life expectancy following the introduction of these therapies, the burden is likely to continue into old age. A better understanding of the residual clinical, psychosocial, and economic burden that lung disease imposes on pwCF in the era of CFTR modulator therapy highlights the remaining unmet needs and could assist healthcare systems in better planning resource allocation.