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Cystic fibrosis (CF) is a progressive, life-limiting genetic disease caused by a dysfunctional CF transmembrane conductance regulator (CFTR) protein that leads to mucus clearance abnormalities, the development of chronic endobronchial infections and progressive, irreversible lung damage. Historically, treatments for CF have largely been supportive in nature and have focused on symptomatic improvement from the many CF-related disease manifestations, including pulmonary, gastrointestinal and sinus-related morbidities. The use of acute and chronic medications to address these disease-related complications has undoubtedly improved the lives of people with CF (PwCF) and extended life expectancy.
The approval of ivacaftor by the US Food and Drug Administration on 31 January 2012 for PwCF 6 years and older with a G551D mutation has heralded in a new era of CF care. For the first time, a CF therapy (termed CFTR modulator) was developed to target the underlying CFTR protein defect itself. Since its approval, several other CFTR modulators have been deployed in the clinical arena; the most recent (and arguably the most effective) of which is elexacaftor/tezacaftor/ivacaftor, a CFTR modulator currently approved for all PwCF 12 years and older with at least once copy of the Phe508del mutation.
While the approval of CFTR modulators has revolutionised the care of PwCF and might even improve life expectancy, there remains a number of potential barriers to CFTR modulator adherence, including cost, perceived medication benefit, delays in prior authorisations, decreased access to care and individual factors …
Contributors CSD and JDC: substantial contributions to the conception or design of the work; the acquisition, analysis, or interpretation of data for the work; drafting the work or revising it critically for important intellectual content; final approval of the version to be published; and agreement to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Provenance and peer review Commissioned; externally peer reviewed.