Recap on the Kaftrio Dispute- Access for 35 children with CF aged 6-11 and the continuing urgency for both the HSE and Vertex to reach an agreement  

The following is a recap of the reasons why CFI is organising a protest at lunchtime on Wednesday 14th September outside the Oireachtas in Kildare St, Dublin. 

• 35 young children with Cystic Fibrosis continue to be caught in the middle of a pricing dispute that deprives them of a ground breaking-drug (Kaftrio) that is already available in Ireland to all other eligible adults and children with CF 

• It remains the responsibility of both the HSE and VERTEX to resolve their differences and reach an agreement as urgently as possible 

• Delayed access to Kaftrio is delayed access to vital healthcare for the 35 children and raises major ethical and human rights issues   

• To date the HSE/Department of Health/Irish government has refused repeated requests for a meeting to hear the direct concerns of parents/CFI 

Since May 2022, 35 children with Cystic Fibrosis remain without access to the ground-breaking drug therapy Kaftrio, despite the approval of the European Medicine Agency (EMA) in January 2022. By contrast, there are presently 11 other European Countries that provide Kaftrio to 6-11 year olds including our nearest neighbour, Great Britain and Northern Ireland (UK). The 35 children in Ireland were excluded from this therapy in May 2022 at the same time as 140 other children with CF aged 6-11 successfully gained access to Kaftrio.

The exclusion of the 35 children stems from a pricing dispute between the HSE and VERTEX and the fact that their inclusion was not foreseen in a 2017 pipeline agreement. All other eligible children and adults with CF in Ireland have already gained access to Kaftrio, except for the 35 children at the centre of this dispute. Lack of agreement could delay access to Kaftrio for up to 5 years for some children. More children will be affected in future if the issue remains unresolved.   

Referring the issue to the NCPE will not help and will only cause further delays 

There has been some speculation about referring Kaftrio for the 35 to the NCPE for a Health Technology Assessment (HTA). This takes a minimum of 90 days, but can often take much longer. This is contradictory as Kaftrio is already being reimbursed for 6-11 year olds in Ireland (except for the 35 excluded children). As previously stated, it is also been approved by the EMA and is available in 11 other European countries. 

CFI lobbying of VERTEX and the Irish Government   

Cystic Fibrosis Ireland (CFI) continues to make representations to both VERTEX, the pharmaceutical company that manufactures Kaftrio and the Irish Government to ensure that an agreement is reached. We are also seeking to ensure that the agreement is future proofed to ensure it does not impact on other children with CF.  CFI put our concerns directly to senior VERTEX Officials in Rotterdam in June 2022 and we continue to urge the company to make every effort to reach an agreement.  We have sought meetings with the Irish Government since early May 2022 but to date, we have not been able to secure a meeting that would enable parents/CFI to provide our views directly and to seek/contribute to solutions. We have also been given mixed messages by government bodies through the media.  

Join us at the protest at lunchtime on Wednesday 14th September outside the Oireachtas commencing 12.45pm