This update is provided from emerging information and will be amended/added to as more information becomes available.  

The positive news is that Cystic Fibrosis Ireland (CFI) understands that the extension of Kaftrio to children with CF aged 6-11 in Ireland will commence in May 2022, slightly earlier than we had previously anticipated. We estimate around 140 children with CF in this age cohort will gain first time access to this crucial CFTR modulator drug therapy, following on from the EMA decision in January 2022, previously reported by CFI. 

However it is very disappointing from available information that this good news may not be shared by all families of children with CF who are eligible for this EMA extension. This appears to us as a departure in both practice and spirit from the ground-breaking reimbursement Portfolio Agreement agreed in 2017 between the HSE and VERTEX.

CFI understands that as things stand at present, around 35 children with certain genotypes will not gain access to Kaftrio when it is added to the HSE High Tech Hub in coming weeks. CFI understands that the main stumbling block in on-going negotiations for reimbursement is an additional premium that is being sought by the manufacturers (VERTEX) for those with certain genotypes. It is unclear to CFI why this premium is being sought at this time and we are not privy to the extent of this additional premium. However CFI is concerned that this stumbling block has emerged so late in the day and as a first step we will be seeking further information from both VERTEX and the HSE, including confirmation of which cohorts of children will be impacted.   

CFI Next Steps 

CFI has always campaigned on the principle that ‘no child is left behind’ - that all those who stand to benefit from CFTR modulators, such as Kaftrio, should gain access. The responsibility for making sure that all children will benefit lies with both VERTEX and the HSE and we would implore both of them to redouble their efforts to find a solution as soon as possible. Your CF team will be able to advise if your child is one of the 35 young people with CF who are likely to be impacted. 

As a first step CFI will object to this unacceptable development (if confirmed as expected). We will seek meetings with both VERTEX and the HSE to voice our concerns. We will also establish a 'concerned parents group', whose children are affected, to keep you updated on a regular basis and to seek participation in our future advocacy.  Please contact Samantha Byrne, Senior Coordinator for CFI Services, if your think your child will be impacted and if you wish to be part of this group sbyrne@cfireland.ie