The survival of people with cystic fibrosis has improved dramatically over the past 60 years, such that adult patients now outnumber paediatric patients in some countries.
This improvement can be attributed largely to advances in the treatment of complications of cystic fibrosis, but new therapies that target the underlying cause of cystic fibrosis—the absence or impaired functioning of the cystic fibrosis transmembrane conductance regulator (CFTR) protein—promise to transform health outcomes for patients with the disease. Studies of ivacaftor, the first CFTR modulator therapy, have convincingly shown short-term benefits
and provided evidence that the drug can slow the progression of lung disease.
Implementation during early childhood of such CFTR modulator therapy could offer patients with cystic fibrosis the opportunity of a healthy lifespan. As the outlook for patients with cystic fibrosis changes, the time seems right for all stakeholders to consider what the future might hold. In a Lancet Respiratory Medicine Commission paper,