The equitable challenges to quality use of modulators for cystic fibrosis in Australia

Fawcett, Laura K; Waters, Shafagh A; Jaffe, Adam

doi:10.5694/mja2.52527

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Cystic fibrosis, an autosomal recessive disease, causes premature mortality with a current life expectancy of 56 years.1 Variations in a single gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR), an anion channel, cause this multisystemic disease.2 Bronchiectasis remains the most significant contributor to mortality, with other affected systems including the gastrointestinal, pancreatic, hepatobiliary, sweat glands and reproductive systems.3 Clinical manifestations of cystic fibrosis vary widely, leading to diverse phenotypic expressions.

1 Sydney Children's Hospital Randwick, Sydney, NSW
2 University of New South Wales, Sydney, NSW

Correspondence: laura.fawcett@health.nsw.gov.au

Acknowledgements:

LF is supported by the Rotary Club of Sydney Cove/Sydney Children's Hospital Foundation and UNSW postgraduate award scholarships. SW is supported by the UNSW Scientia program and the Australian National Health and Medical Research Council. There was no role of the funding sources in the planning, writing or publication of the work. Colman Taylor provided feedback on a draft manuscript regarding health technology assessment pathways.

Competing interests:

AJ is chair of the scientific and medical advisory committee of Rare Voices Australia and has received speaker payments from Vertex Pharmaceuticals. LF has been a sub‐investigator on Vertex clinical trials and received sponsorship of travel costs to attend educational meetings. SW has received competitive funding sponsored by Vertex Pharmaceuticals. Vertex Pharmaceuticals had no involvement in the planning, writing or publication of this article.

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The equitable challenges to quality use of modulators for cystic fibrosis in Australia

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