Bessonova L, et al. - Given that understanding long-term effects of ivacaftor in cystic fibrosis (CF) is important for patients and healthcare providers, researchers used data from the US and the UK CF registries to assess clinical outcomes and disease progression in ivacaftor-treated patients in this ongoing, observational, postapproval safety study. Significantly lower risks of death, transplantation, hospitalisation and pulmonary exacerbation were reported for ivacaftor-treated US patients, compared with comparators; similar trends were reported in the UK. Findings demonstrated that in terms of clinically important outcomes, ivacaftor provided favourable results. This finding adds to the growing body of literature supporting disease modification by cystic fibrosis transmembrane conductance regulator modulation with ivacaftor.