OBJECTIVES: Cystic fibrosis (CF) is an autosomal recessive condition caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein, leading to reduced CFTR function. CFTR modulator therapies target defects caused by mutations in CFTR thereby treating the underlying cause of CF. The aim of the study is to provide an update on the utilization and expenditure associated with CFTR modulator therapies in Ireland since their reimbursement in 2013.

METHODS: This study was performed using the Health Service Executive Primary Care Reimbursement Services (HSE-PCRS) national pharmacy claims database. Data on all claims for CFTR modulator therapies between January 2013 and December 2023 were included; ivacaftor (Kalydeco®), lumacaftor/ivacaftor (Orkambi®), tezacaftor/ivacaftor (Symkevi®) and ivacaftor/tezacaftor/elexacaftor (Kaftrio®). Reported expenditure excludes confidential commercial arrangements.

RESULTS: In 2013, ivacaftor was the only CFTR modulator therapy reimbursed in Ireland, with just under 150 patients and an associated expenditure of approximately €21 million. Lumacaftor/ivacaftor was reimbursed in 2017, tezacaftor/ivacaftor was reimbursed in 2019, and finally the triple therapy regimen elexacaftor/tezacaftor/ivacaftor was reimbursed in 2020. By 2023, there were over 1,100 patients in receipt of elexacaftor/tezacaftor/ivacaftor, with an associated expenditure of over €39 million. In 2023, the total expenditure associated with CFTR modulator therapies was almost €78 million.

CONCLUSIONS: Neither ivacaftor nor lumacaftor/ivacaftor were considered cost-effective when subject to Health Technology Assessment (HTA). Tezacaftor/ivacaftor and ivacaftor/tezacaftor/elexacaftor were not subjected to HTA prior to their reimbursement, due to the introduction of a ‘portfolio deal’ with the manufacturers (Vertex) in 2017. The portfolio deal introduced a budget cap for the suite of CFTR modulator therapies with some advantages, including; expedited patient access, and budget certainty for the payer. However, the HTA process plays an important role in obtaining value from pharmaceutical expenditure in Ireland and bypassing this process could have serious long-term financial implications for the healthcare system.