• As expected, both Orkambi and Kalydeco (for the treatment of children with CF aged 2 years and older) will be subject to a full review by the National Centre for Pharma Economics (NCPE) in Ireland. This is normal for such drugs. This process includes a Health Technology Assessment (HTA) that may take up to 90 working days. We will let you know the outcome of these two reviews as soon as we hear.

    We thank  everyone who responded to our recent survey – we received almost 500 responses which is an indication in itself how important this issue is  to our members. CFI is currently finalising our report which will include information drawn from this survey.

    In the meantime, Minister for Health, Leo Varadkar TD has agreed to meet with Cystic Fibrosis Ireland following our request for a meeting before Christmas. This will take place before the end of January. CFI welcomes this opportunity to discuss with the Minister issues such as access to new and innovative therapies as well as other key CF related issues including staffing levels in hospitals and investment in key CF centres.

    Many thanks to all our members for their support to date.

    Philip Watt

    CEO Cystic Fibrosis Ireland

  • Philip Watt CEO, of Cystic Fibrosis Ireland says "CF patients in Ireland are dismayed by today’s ruling from the NCPE which recommends that ground breaking drug ‘Orkambi’ is not funded".

    However CFI notes that the NCPE ruling states that Orkambi is not funded ‘at submitted cost’ and this opens the door to further price negotiations.

    Cystic Fibrosis Ireland calls on the Government to clarify if it will enter into further negotiations and calls on the pharma company Vertex to significantly reduce the price of Orkambi as part of these negotiations.

    Cystic Fibrosis Ireland welcomes the fact that the positive health impact of Orkambi is recognised by the NCPE in today’s judgement.

    Philip Watt CEO of Cystic Fibrosis Ireland states "we support a fairer deal for this drug but it will be a travesty if this drug is not provided to our patients or if there is a significant delay in providing this drug".

    Note: Around 500 people with CF have the potential to benefit from Orkambi in Ireland.


    For press enquiries please contact Philip Watt on 087-6370557. 


  • Extension of Kalydeco (Ivacaftor) Update

    CFI have been informed that some news about the extension of Kalydeco (Ivacaftor), which is currently undergoing a Health Technology Assessment (HTA), is due to be posted on the website of the NCPE (National Centre for Pharmacoeconomics) around the 12 October or shortly after. The extension application includes commencing treatment of those already on Kalydeco to 2 years and over (children with CF who have the G551D gene alteration are currently 6 years old when treatment commences). The extension also covers other class 111 gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R, as listed on the NCPE website.

    Please note that this news item should not be confused with the Orkambi negotiations. These negotiations are still on going and we are at an important stage.

  • Extension of Kalydeco (Ivacaftor): Update from NCPE

    Today (12 October), the National Centre for Pharmacoeconomics (NCPE) have issued their recommendation following the Health Technology Assessment (HTA) of Kalydeco for children aged 2- 5years. Kalydeco for children aged 2–5 year olds* has not been recommended for reimbursement at the submitted price. (Note: this is not related to the forthcoming Orkambi decision)

    CFI notes that the NCPE ruling leaves a door open to further price negotiations, but the decision will be very disappointing and frustrating for the 18 young children with CF and their families, who could benefit greatly from access to this ground-breaking therapy.

    Philip Watt, CEO of Cystic Fibrosis Ireland says “Accessing this life-changing treatment is essential as eligible patients stand to benefit so much at such a crucial time in their development, fighting a progressive, life-limiting disease. We will be urging the government to enter into price negotiations as soon as possible. We would also urge the pharma company, Vertex, to reduce the price of this important drug during these negotiations”.

    The treatment, which is currently only available for those over six years old, has been shown to significantly increase lung function, slow the progression of lung disease and significantly reduce hospital admissions.

    Research published earlier this year in ‘The Lancet Respiratory Medicine’ journal suggested that pre-school children with one of the nine mutations (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R) could also benefit from improved pancreatic function, weight gain and movement of salt between cells.


    *This assessment refers to children aged 2-5years who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P,S549N or S549R.

  • YesOrkambi

    A YesOrkambi group involving parents and people with CF in Ireland has posted a video on facebook in support of the reimbursement of Orkambi in Ireland. This is the first in a series of videos that includes people with CF who have benefitted from Orkambi as part of the clinical trials.


    News from CFI 

    The head of the NCPE Professor Michael Barry has indicated that both the HSE (Corporate Pharma Unit) and the drug manufacturer ‘Vertex’ are willing to negotiate on Orkambi in relation to price. CFI has urged that this process is completed as soon as possible and the drugs company should significantly reduce the price of Orkambi as part of these negotiations.


    Selection of Media coverage

    June 1
    NCPE issue statement that Orkambi ‘reimbursement not recommended at the submitted price’

    June 2
    Jillian McNulty (PWCF); Philip Watt (CFI) and later Professor Michael Barry NCPE on RTE ‘Morning Ireland’

    Orla Tinsley (PWCF) and Brian McCarroll (PWCF) on Sean O’Rourke’s RTE Programme

    Terri and Aisling Golden (Sligo branch) on Ray D’Arcy programme

    Irish Times website has regular updates

    There is also coverage in today’s Daily Mail, Irish Independent and Examiner and on local radio stations including Galway Bay FM; Clare FM and on Cavan radio involving CFI board and branch members including Kieran McCarthy and Lorraine O’Neill. Jillian McNulty will be on RTE and UTV television news this evening (Thursday 2 June).

  • Orkambi update

    CFI has recently written to the Minister for Health to urge once again that the negotiations on Orkambi and the extension of Kalydeco are concluded in a positive way for our members as soon as possible and to remind him and the government of the importance of these drugs to our members.

    Background note:

    Cystic Fibrosis Ireland has undertaken significant lobbying on this issue including:

    • Two meetings with the Minister for Health and Senior Officials
    • Meeting with 56 TD’s from all political parties
    • A comprehensive survey and submission to the National Centre for Pharma-Economics (NCPE)
    • Meetings with the NCPE
    • Media statements and coverage on both national TV and local and national radio and other advocacy at a local and national level.


    We acknowledge and thank patient advocates and parents who have been active on this crucial issue and we will bring you news as soon as we hear the outcome from the negotiations.

    Philip Watt

  • Calling all people with CF, parents, carers & family members - we want to hear your views!!

    Please complete our survey by clicking HERE


    Why is your participation so important?

    As many of you are aware, two new possible treatment options for some people with CF (PWCF) are currently being assessed for both clinical and cost-effectiveness by the National Centre for Pharmacoeconomics (NCPE). 

    1. Orkambi, which seeks to correct the basic underlying genetic defect in 57% of people with CF in Ireland, people with two copies of F508del mutation. 
    2. The expanded use of Kalydeco for mutations:
          • G551D & other non-G551D gating mutations (expanded use to PWCF aged 2-5 years)
          • R117H (for PWCF over 18 years)

    As with all new therapies, they must undergo Health Technology Assessments (HTA) which is

    "A form of research that generates information about the clinical and cost-effectiveness of health technologies. These technologies can include drugs, medical devices, diagnostic techniques, surgical procedures and public health programmes such as cancer screening programmes. A Health Technology Assessment (HTA) may also look at the social, ethical, medicolegal and organisational aspects associated with use of a technology including its resource implications and budget impact.

    The information provided by the HTA is used to inform health policy decisions regarding the investment in (or disinvestment from) these health technologies."

    Cystic Fibrosis Ireland (CFI) are putting together a submission which will be made to the National Centre for Pharmacoeconomics (NCPE). This primary aim of this submission is to ensure the patient & family perspectives are formally taken into consideration during the Health Technology Assessment of the new therapies: Orkambi & Kalydeco (for expanded use).

    The purpose of this survey is to gather information from both people with CF and their families about experiences & views of treatment options for cystic fibrosis & ultimately, 'what matters to you'.

    It is so important that the voice of the CF community is heard loud and clear throughout, so please fill in the survey to have your say today.

    This process and the arrival of similar innovative therapies in the future will challenge the HSE & Irish Government to look at what 'value for money' really means in the context of CF care. The whole community must speak up and make sure these decisions are guided by the unique knowledge and experience of people living with CF.

    This survey has been shared by the Cystic Fibrosis Trust, UK, who have kindly allowed CFI to use & edit to questionnaire to meet the needs of the Irish CF population.

    The survey will take approximately 15-20 minutes to complete & we ask you to provide as much detail as possible.

    The results of this survey will be used to represent the views of the CF community & will primarily be used for advocacy purposes, and to inform policy decisions.

    Your responses will be completely confidential and any published results will be entirely anonymous.

    If you have any questions about the research, please contact Katie, Research & Development Officer, CFI at This email address is being protected from spambots. You need JavaScript enabled to view it. or locall 1890 311 211