OBJECTIVES: The cost of new medicines is constantly debated, although attempts to introduce innovative pricing schemes have faced challenges. In 2017, a ‘pipeline deal’ was agreed for a new range of cystic fibrosis (CF) treatments in Ireland. We explore the merits of this deal.

METHODS: Targeted search of health technology assessment (HTA) reports and associated bodies in the UK and Ireland for CF treatments.

RESULTS: In the UK, ivacaftor was reviewed by both the Scottish Medical Consortium (SMC) and All Wales Medicines Strategy Group (AWMSG) reporting annual costs of €207,271 per patient (pp) and incremental cost effectiveness ratios (ICERs) ranging from €237,097–€2,699,192/QALY (SMC). Ivacaftor is now available throughout the UK following government input. Ivacaftor-lumacaftor is still not available despite considerable public pressure; annual costs of €118,440pp and ICERs from €154,287–€522,852/QALY (NICE).

In Ireland a ‘pipeline deal’ was agreed in 2017 where full access would be granted to current options and future treatments (phase 3: tezacaftor, VX-659, and VX-445) for a confidential price, expanding treatment to approximately 90% of CF patients. Early clinical data for tezacaftor-ivacaftor looks promising; significant (p<0.001) improvement in absolute (4 percentage points) and relative (6.8 percentage points) change in percentage of the predicted FEV1. A similar deal is being explored in the UK, but a deal has yet to be agreed.

CONCLUSIONS: HTAs to date have reported high ICERs for the CF treatment options appraised. Whilst the ‘pipeline deal’ is confidential, it would seem that a significant price decrease would be required to meet conventional thresholds. Adding the uncertainty of future treatment options further complicates the case. Innovative pricing strategies may aid reimbursement in the future, but it is likely that a ‘portfolio’ approach will only be suitable for indications with a monopoly manufacturer rather than indications where multiple companies are competing.