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Original research
Utilisation, expenditure and cost-effectiveness of cystic fibrosis drugs in Ireland: a retrospective analysis of a national pharmacy claims database
  1. Amelia Smith1,2,
  2. Michael Barry1,2,3
  1. 1Department of Pharmacology and Therapeutics, Trinity Centre for Health Sciences, St James’s Hospital, University of Dublin Trinity College, Dublin, Ireland
  2. 2Medicines Management Programme, Health Service Executive, Dublin, Ireland
  3. 3National Centre for Pharmacoeconomics, St. James’s Hospital, Dublin, Ireland
  1. Correspondence to Dr Amelia Smith; smitha25{at}


Objectives To determine the use and expenditure associated with cystic fibrosis (CF) modulator therapies in Ireland since their reimbursement in 2013.

Design A retrospective analysis of a national drug claims database.

Setting The data included in this study are nationally representative (Ireland).

Participants Data on all persons receiving CF modulator therapies were included.

Methods We obtained national claims data for CF therapies from the Health Service Executive’s Primary Care Reimbursement Service. We determined the use and expenditure associated with CF therapies from January 2012 to March 2020.

Results The increased prescribing of CF modulator therapies was associated with an approximate fivefold increase in expenditure from €23 million in 2013 to €113 million in 2019. Many patients who initiated lumacaftor/ivacaftor in 2017 went on to receive symptomatic therapies, and subsequently initiated tezacaftor/ivacaftor in 2019.

Conclusion Despite none of these modulator therapies demonstrating value for money when subjected to health technology assessment, the associated Irish expenditure reached €113 million in 2019 alone.

  • cystic fibrosis
  • health economics
  • rationing

This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See:

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  • Contributors MB contributed to conception and design, acquisition of data, interpretation of data, drafting the article and final approval of the version to be published. AS contributed to design, analysis and interpretation of data, drafting the article and final approval of the version to be published. MB is the guarantor.

  • 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.

  • Patient consent for publication Not required.

  • Provenance and peer review Not commissioned; externally peer-reviewed.

  • Data availability statement Data may be obtained from a third party and are not publicly available. No additional data available.

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