Kaftrio Update 

CFI urges fast-tracking of the forthcoming HTA for the Kaftrio 35 and immediate access in the interim

It remains CFI’s clear understanding as per our previous update (find Mondays statement here) that we are heading towards a Health Technology Assessment (HTA) for the Kaftrio 35. Further, it remains CFI’s understanding that both the HSE and Vertex agree that this process is a necessary prerequisite before a decision on access for the 35 children aged 6-11 can be made. 

While this decision may appear very disappointing news at face value, the agreement to proceed with the HTA by both parties is new and also provides a potential path for resolution to this cruel dispute. CFI says this with caution - the HTA needs to be fast tracked with completion by the end of January 2023. Further delays are unacceptable. 

The pain and anguish of the families has been very clearly expressed over the past few days on RTE’s Joe Duffy Show and other media programmes. The efficacy of Kaftrio should no longer be questioned given the testimonies of those already on this ground-breaking drug therapy. It is very unfair that parents have to speak in public and to take part in demonstrations to access an essential drug. There needs to be a better approach, including the establishment of the promised National Medicines Agency and more Government funding for new and innovative drug therapies for all rare diseases.

CFI continues to urge that kaftrio is provided to the 35 in the interim and that the 2017 pipeline deal is extended so similar disputes do not reoccur in future. CFI will also send a submission and will seek to meet the NCPE when the HTA is shortly confirmed as we expect

What is a Health Technology Assessment?  

All expensive drugs and medical technology are generally required to go through an HTA

This is an assessment undertaken by the National Centre for pharmaco-economics (NCPE) that considers a range of issues including:

·      Clinical effectiveness and Safety 

·      Cost effectiveness 

·      Budget impact 

·      Social and ethical aspects 

Normally this process can take 90 days or more,  but given the unique features of this dispute,  there is no reason why the HTA cannot be fast tracked. CFI understands that fast- tracking is being considered at this time.