Utilisation, expenditure and cost-effectiveness of cystic fibrosis drugs in Ireland: a retrospective analysis of a national pharmacy claims database

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