The NCPE have recommended:

• Orkambi - PWCF with two copies of F508del aged 12 and over
• Kalydeco - expanded use for children with a 'gating mutation', such as G551D aged 2 - 5 years
• Kalydeco - expanded use for adults, aged 18 and over, with genetic mutation R117H

to undergo a full Health Technology Assessment - more information visit www.ncpe.ie 

CFI have prepared and completed the 'Patient Interest Group Submission of Evidence' template, developed by the NCPE. The aim of our submission, and indeed continued communications & representation, is to call for fair access to innovative therapies for people with CF (PWCF). 

This submission is a key opportunity to highlight the importance of YOUR views and present information that is not already available to assessors. 

In order to ensure our submission to the NCPE, accurately reflects the views and opinions of people with CF, carers and families we have been actively engaging with the CF community in a number of ways to gather this information – we sincerely thank everyone who took the time to engage with CFI on this matter. Click on the image below to read a summary document of our submission.

This first submission focusses primarily on Orkambi but CF Ireland are currently finalising a more specific submission to be incorporated during the assessment of Kalydeco for both; 

• expanded use for children with a 'gating mutation', such as G551D aged 2 - 5 years
• expanded use for adults, aged 18 and over, with genetic mutation R117H

We have also collected a number of patient testimonials from people who have been on the clinical trials for the above therapies - the families of those who have been involved and indeed, patients who could potentially benefit from the CFTR corrector therapies currently under-going assessment. Given the personal nature of these testimonials, and direct requests from some of those who shared their stories, we have not shared these within the document - but have made all available to the NCPE.