• Minister's Statement on Orkambi and Kalydeco

    Cystic Fibrosis Ireland welcomes the statement last night, by Minister for Health Simon Harris TD that progress is being made in relation to the approval of ground breaking CF drugs Orkambi and Kalydeco. CEO of CFI Philip Watt stated 'while this is a positive statement of intent there will remain high anxiety among the CF community in Ireland until these drug therapies are finally approved by the HSE and the Minister.'

    CFI is supporting a dignified protest to highlight the urgency of approving these drug therapies outside the Dail on Wednesday 1 March at 1.15pm.

    Click here to read the Minister's statement.

  • Open letter to Minister for Health, Simon Harris TD

    29th November 2016

    Dear Minister,

    On behalf of Cystic Fibrosis Ireland we would ask to meet with you as soon as possible.

    We know from previous contact and your recent letter to us, that you are a compassionate and caring person, and we know your task as Minster for Health is a difficult one, including in respect of approving high cost innovative therapies.

    However, there is no disguising the reality that there has been considerable upset within the CF community in Ireland over the last couple of days which has not been helped by how the Orkambi 'decision' has been conveyed to patients. We note that you share some of our concern in this regard. This heartless form of communication to ill patients has since been compounded by the dearth of information that has been provided by the HSE to our patients since the 'decision' was leaked late on Saturday evening to a well-respected journalist.

    We note in your letter to CFI that you do not see this as the end of the process. We hope this means there is light at the end of this tunnel. However we remain very unclear to what you mean in practice. We would wish to discuss this further with you.

    In the absence of any communication from the HSE to us, we remain concerned that the impact of Orkambi appears to be 'talked down'. We would point out the following:

    Why would the HSE negotiate for 25 weeks/6 rounds of negotiations, if the HSE believed that Orkambi was not effective in the first place?

    In this context, we are further concerned that there appears to be no public acknowledgement by the HSE of the long term research data that was published on the 28th of October 2016 that confirmed:

    • A 40% decrease in hospitalisations
    • The retardation of the progression of CF for those on Orkambi
    • The reduction in other costs resulting from less hospitalisations and less dependency on other CF drugs

    We note that the highly respected Professor Stuart Elborn CBE formerly of Belfast City Hospital and now lead clinician in the lead CF hospital in the UK (Brompton Hospital in London) and Principal Investigator for the clinical trials for Orkambi stated on the 28th October 2016:

    "Long-term follow-up data has indicated that this treatment can prevent disease progression.

    "Initially, we were able to show that you can make people a bit better. Now we're seeing exciting and reassuring long-term improvement."

    "We hope this will lead to a further rethink about the long-term benefits," said Prof Elborn. "There are likely to be reductions in the cost of hospital visits and other treatments."

    "I'm really excited by the therapy and also the pipeline of other powerful drugs that could get us closer to a cure."

    (Source: 28.10.16 Press Association)

    To conclude, we would urge you as Minister to intervene and make Orkambi available for the 550 people who stand to benefit as soon as possible, even if this involves some further negotiations. We would also urge that negotiations for the extension of Kalydeco for 2-5 year olds commences as soon as possible. We will continue to lobby Vertex to significantly lower the cost of this important drug.

    Yours sincerely

    Philip Watt

    CEO Cystic Fibrosis Ireland 

  • Open Letter to Vertex Pharmaceuticals from CFI

    29th November 2016

    President, and Vice President
    Regional General Manager
    Europe North
    Vertex Pharmaceuticals (Europe) Ltd

    Dear Sirs,

    On behalf of Cystic Fibrosis Ireland (CFI) we would strongly urge, even at this late hour, that further negotiations are undertaken between Vertex Pharmaceuticals and the HSE to find a fair price for the reimbursement of Orkambi for people with CF in Ireland.

    We would urge you to reduce/further reduce the price of Orkambi and if possible enter into a ‘shared risk’ agreement to enable this important drug to be made available in Ireland – if these are the sticking points in the negotiations.

    Through a failure to communicate from the HSE, we have not been privy to the reasons why negotiations have been seemingly concluded/suspended except from snippets gleaned from the media in relation to concerns about the price and efficacy of Orkambi. We would hope the negotiations will continue.

    We have written to the Minister for Health, Simon Harris TD to seek a meeting to discuss these issues and to be reassured that as he has stated this is not the end of the process. We would equally urge Vertex to seek further compromises from which a fair decision on price can be reached and we would also urge a meeting with you on the same to convey these concerns.

    Yours Sincerely

    Philip Watt

  • 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 and Kalydeco extension update

    CFI understands from reliable sources that the HSE and Vertex met again this week in the latest round of negotiations on the price of Orkambi. We understand that progress is being made, but whether this progress will be sufficient to secure a deal remains unclear. The positive news is that negotiations are continuing on an active basis. CFI has called for these negotiations to be concluded as soon as possible and a fair deal for both parties is reached as this decision is of immense importance to people with CF and their families in Ireland. Cystic Fibrosis Ireland has also made representations to the HSE on the importance of commencing (as soon as possible) negotiations on the extension of Kalydeco to 2-5 year olds which impacts on 18 children with CF in Ireland. We will continue to keep our members updated on both these issues.

  • Orkambi and Kalydeco Update

    The Irish Independent reported on Saturday 14th of January that Vertex has made a revised offer to the HSE for the reimbursement of Orkambi and Kalydeco and this offer is currently under consideration by the HSE (download press report here).

  • Orkambi and Kalydeco: Advocacy and Protest Strategy of Cystic Fibrosis Ireland

    Cystic Fibrosis Ireland (CFI) understands that a decision on Orkambi and the extension of Kalydeco to 2-5 year olds will be decided within the next two weeks by the HSE Directorate. We understand that a revised offer has been made by the manufacturer (Vertex) and this is what is presently being discussed. 

    In other words, negotiations are at an advanced stage and we must be careful on the one hand to keep up lobbying behind the scenes for these vital drugs but at the same time provide a space for the HSE and Vertex to reach an agreement and for the government to have a reasonable time to make a decision.  

    It’s vitally important that the government gets the best deal on price possible, not least because there are other very promising CF ground-breaking drugs in the pipeline in the near future that will need to be resourced. 

    It is therefore the view of the Board of Cystic Fibrosis Ireland which met on 28th of January that it would be better to give space for these negotiations but to support a further protest on 1 March if necessary. 

    CFI understands the high level of anxiety among our members. This was heightened by an incorrect headline in the Irish Times of 1 February which wrongly stated that talks had broken down. Both the HSE and Vertex contacted CFI on the 1 February to say that this was incorrect and that negotiations are continuing and are at an advanced stage.

    We understand that there will be a candlelight vigil held in some parts of the country on the 17th of February or on other dates. CFI wishes the organisers good will and the best of luck with this initiative and hope that it is successful in raising awareness. If you wish to support this initiative, please do so. 

    However on balance, CFI believes it is better to give our official support to the protest that brings a lot of people together at a time when the national media and politicians are more likely to sit up and take notice. In this context we would propose that CFI members get fully behind a protest outside the Dail on the 1 March 2017 at 1.15pm. We very much hope that a positive decision in the meantime will mean that this protest can be cancelled. 

    A small coordination group of all those active in the various Orkambi and Kalydeco groups will be held on Monday 13th of February to ensure a coordinated response. We have no idea as to whether these important therapies will be approved or not, but we do know that progress has been made and we ask for your continuing support for all of us to work together. Thanks for everyone’s contribution to date, it is much appreciated!

    Philip Watt

    CEO on behalf of Cystic Fibrosis Ireland 

    7th February 2017


  • Orkambi and Kalydeco Update

    May 22nd 

    CFI has written to both Vertex and the HSE to urge them to agree the final details that will make Orkambi (and the extension of Kalydeco) available to our patients as soon as possible. CFI understands from both parties they do not envisage any problems at this stage which is good news. 

  • Orkambi negotiations: latest update

    The Sunday Business Post reported at the weekend that negotiations between the state and the manufacturer of cystic fibrosis drug Orkambi have finally begun.

    You can download the article here. (pdf)


  • Orkambi protest information organised by Jillian McNulty

    In response to information requests from our members about next week's protest:

    Jillian McNulty, an independent advocate for CF, is organising a protest outside Dail Eireann in Kildare Street on Wednesday 7th of December. She is asking for everyone who wishes to participate to assemble at 1.15pm outside the main gate to the Dail in Kildare Street. Please note the protest will conclude at 2.30pm.

    Dail Eireann

    Link to Jillian McNulty's event page on Facebook: https://www.facebook.com/events/772854432852488/

    CFI Note: Please observeCommunity Cross Infection precautions

  • 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

  • Orkambi Update - 30th November 2016

    The following are points made by Cystic Fibrosis Ireland in response to the public debate on Orkambi over the past few days.


    1. The Urgent need For Ministerial Intervention and Positive Response by Vertex

    2. The Efficacy of Orkambi

    Cystic Fibrosis Ireland urges the HSE to provide a more comprehensive and fairer public statement on the efficacy of Orkambi, particularly in relation to recent research that has shown that CF progression is slowed down by Orkambi and the impact of the 40% reduction in exacerbations.

    Orkambi (also known as ‘lumacaftor/ivacaftor’) is for people aged over 12 with two copies of F508-del gene alteration, which about 550 people in Ireland (compared with around 3,200 people across the whole of the UK).

    Declining lung function and exacerbations of Cystic Fibrosis is ultimately the main cause of death among people with cystic fibrosis. By reducing this decline, precision medicines like Orkambi can help people with cystic fibrosis stay healthier for longer. The gap in lung function widens for every year that someone eligible is not on Orkambi. This is particularly poignant where one sibling is on Orkambi and one is not.

    Orkambi reduces exacerbations by up to 40%- that is worsening of a condition that results in hospitalisations. Every time a person with CF experiences an exacerbation research has shown it can reduce their survival expectancy from 3-6 months.

    Cystic Fibrosis Ireland urges the Department of Health/HSE to take into account a recent study (28th October 2016) carried out over 96 weeks.

    Analysis of the 96-week PROGRESS extension study of the pivotal phase 3 TRAFFIC and TRANSPORT studies of Orkambi confirmed the safety profile, and mean lung function was maintained above baseline for up to 120 weeks and the 40% reduction in exacerbations.

    Professor Stuart Elborn formerly of Belfast City Hospital and now lead Cf adult consultant in the Brompton hospital commented:
    "Long-term follow-up data has indicated that this treatment can prevent disease progression.

    "Initially, we were able to show that you can make people a bit better. Now we're seeing exciting and reassuring long-term improvement."

    "We hope this will lead to a further rethink about the long-term benefits," said Professor Elborn. "There are likely to be reductions in the cost of hospital visits and other treatments."

    "I'm really excited by the therapy and also the pipeline of other powerful drugs that could get us closer to a cure."

    3. Meeting the Cost of Orkambi from Savings in the Drugs Budget

    CFI has acted responsibly at all times. We did not engage in advocacy over the 25 weeks that the HSE negotiated with Vertex, the pharmaceutical company. This was a deliberate strategy to support the HSE to obtain the best price possible for this important drug. We have called on Vertex to make significant reductions in price and to enter into shared risk agreements with the HSE, if these are the sticking points on which the negotiations have foundered.

    We would not expect the HSE to pay for a drug that does not work on some patients. We would question recent assertions that Orkambi only works on about one quarter of those taking part in clinical trials and we have sought expert opinion on this.

    IPHA agreement and related savings

    CFI notes from the Minister’s statement to the Joint Oireachtas Committee meeting in September 2016 re the IPHA agreement
    ‘The States team objective was to improve the assessment and reimbursement process for new drugs and to secure significant price reduction so as to enable continued access to new and existing drugs for Irish patients while reducing growth in the HSE’s overall drugs bill…..The new Agreement, which runs to the middle of 2020, is projected to result in savings – that is, expenditure foregone – of some €600 million from IPHA companies, with a further €150 million in savings anticipated from non-IPHA companies over the lifetime of the deal’.

    The Fight for Orkambi will continue

    CFI will continue to lobby the HSE/Department of Health and Vertex to reach a fair price on Orkambi.

    Philip Watt

    CEO, Cystic Fibrosis Ireland

  • Orkambi Update - Ongoing Negotiations

    Cystic Fibrosis Ireland have been advised that Vertex met with the HSE yesterday (05.01.17) and negotiations on the re-imbursement of Orkambi are ongoing.

  • Orkambi update

    CFI knows for certain that the Orkambi negotiations are still very active and have entered into a crucial third round of talks focussed around price. We understand these talks are at a delicate stage. As with all important negotiations the two parties should be given the space to get a successful outcome, but they should also be concluded as soon as possible to ease the anxiety of people with CF and their families. This negotiating space is important as a fair price for these drugs also increases the chances of future funding for other promising CF drugs that are currently in the pipeline.

    We will inform our members as soon as we hear the outcome of these negotiations.

    Philip Watt
    CEO CFI 6 October 2016

  • Pat Kenny Tonight features Orkambi


    Linda and Ronan Whitmarsh, parents of Finn (PWCF) along with Philip Watt CEO of Cystic Fibrosis Ireland were featured on the ‘Pat Kenny Tonight’ TV3 programme on Wednesday 23 November.

    The programme also featured one of the awareness videos from the ‘Yes to Orkambi’ campaign led by Bernie Martin. The programme also featured parents of children with CF in the audience.

    The focus of the programme was on the need for the government to approve Orkambi as soon as possible.

    The podcast is available at http://www.tv3.ie/3player/show/1032/ (after about one minute of adverts).  

  • Photos from the Orkambi Protest

    Orkambi protest outside the Oireachtas, 7 December 2016 organised by Jillian McNulty 

















    Progress in US on new CF Drug and on gene therapy trials in the UK

    ORKAMBI approved in the United States

    The first drug to treat the underlying cause with people with Cystic Fibrosis with the most common CF gene alteration in the world f508del has been approved in the United States by the Food and Drug Administration (FDA). The drug is called ORKAMBI. Key points in relation to this drug and Ireland are:

    It is aimed at People with two copies of f508del which is 56.6% of Irish CF population (654 people) although to date it is aimed at those who are aged 12 or more which is just under 50% of the CF population in Ireland (we estimate)

    It has to be approved by the European Medicines Agency (EMA) and the NCPE/ HSE CPU unit in Ireland before it is made available for people with CF. It will likely undergo a Health Technology Assessment (HTA) in Ireland before they make a decision to a) approve the drug b) reject the drug or c) reject the drug on cost grounds but leaving the door open for price negotiation.

    The Results of Orkambi show an increase in lung function of up to 4% (statistically significant)

    Reduction in exacerbations (worsening of condition often leading to hospitalisation) of between 30 – 39%

    Philip Watt, CEO of CFI stated ‘We have evidence that Orkambi has had Life-changing effects for people on clinical trials who have been in contact with us and who have reported a significant increase in quality of life. We would urge the EMA and Irish government to assess this drug as soon as possible.’

    Katie Murphy, Research Officer of CFI stated ‘The promising results from the US and UK are both the outcomes of patient groups investing heavily over many years into research for innovative therapies. We wish to acknowledge the work of the Cystic Fibrosis Foundation in the US and the Cystic Fibrosis Trust in the UK in this context.’

    UK Gene therapy trial results published

    The results of an on-going trial into the potential use of gene therapy to improve clinical outcomes for people with CF have been published. The trial, which was primarily funded by the Cystic Fibrosis Trust in the UK, was conducted by the Gene Therapy Consortium (GTC) and shows ‘promising yet modest’ results. Scientist say there is more work to be done but believe they are on track to deliver a therapy that can make a meaningful difference to patients' lives.

    Professor Eric Alton, from Imperial College London, who co-ordinated the trial, said: "there has been a significant benefit compared with placebo in cystic fibrosis patients. If a bigger study shows big benefits then it's feasible that we'll be able to offer this treatment to patients by the end of the decade."

    The trial, conducted in London and Edinburgh, compared the effects of inhaled gene therapy and a placebo treatment on 136 patients with CF aged 12 and over. Over the course of a year, patients were given 12 treatments at monthly intervals.

    The results, published in The Lancet Respiratory Medicine journal, showed that at the end of the trial lung function was 3.7% better in patients who received the "active" treatment. Participants with the worst lung function at the start of the study experienced a much greater 6.4% gain compared with those in the placebo group. It is important to note that these improvements and effects were inconsistent across the 62 patients in receipt of ‘active’ treatment.

    The scientists are now seeking funding for the bigger "improved liposome" trial that could cost tens of millions of pounds, and would hopefully pave the way for rolling out the treatment. Professor Alton has said;
    “We hope to carry out follow-up studies looking at higher, more frequent doses, combined with other treatments, and better methods for delivering the DNA into cells.”

    Speaking to the BBC, Professor Stuart Elborn of Queen’s University Belfast has said “it is too soon to proceed with larger Phase-three trials” And he called for more small-scale tests to see if a larger dose would be more effective.

    Philip Watt CEO of CFI stated ‘These are the results of a Phase 2 Trial which seek to prove a concept. It will take a great deal of funding and further trials at phase 2 and phase 3 level to bring this research through to a drug that can impact on people with CF. Nevertheless it is a promising development as gene therapy might impact on everyone with CF and not just those with a specific gene alteration. They will have to do further work on drug dosage and combination trails with proven CF drugs.’

    3 July 2015

    For further information please contact Katie Murphy, Research Officer at This email address is being protected from spambots. You need JavaScript enabled to view it.

  • Protest and March

    Wednesday 12th April Kildare Street, Dublin at 1pm

    To support access to Orkambi and Kalydeco Cystic Fibrosis drug therapies


    Assemble 1pm sharp outside the Dail

    Wear something purple and bring a whistle

    In support of our member’s wishes and in partnership with CF advocates, Cystic Fibrosis Ireland is urging all our members and supporters to take part in a protest and march commencing outside the Dail in Kildare Street, Dublin on Wednesday the 12 April 2017 at 1pm sharp.

    It has now been 10 months since the assessments of the CF ground-breaking Orkambi and Kalydeco have concluded. There is heightened anxiety within the CF community of continued delays in approving these drug therapies, despite some recent positive statements.

    CFI calls on both the HSE and Vertex to reach an agreement to provide these ground-breaking drugs without further delay.

    The protest will also include a short march.

    Note: Negotiations are likely to be concluded very shortly. If an agreement is reached before Wednesday 12th of April we will be happy to call off the protest. 

  • Protest at the Dáil today at 1.15pm

    Cystic Fibrosis Ireland will join the CF Community at a protest today, Wednesday 1 March at 1.15pm outside the Dáil to highlight the urgency of approving Orkambi and Kalydeco. We invite anybody who can, to join the CF Community in the continued effort for access to these vital drug therapies.

    CFI welcomed the recent statement by Minister for Health Simon Harris, TD that progress is being made in relation to the approval of Orkambi and Kalydeco. However, for people living with CF time is precious and they have already been waiting nine months for a decision and almost two months since the revised offer came in from Vertex. We understand this revised offer provides a very significant discount on the original list price.

    Jillian McNulty and Philip Watt (CFI) were invited to a private meeting with the Minister for Health Simon Harris at short notice last Monday. The Taoiseach referred to this meeting in the Dáil, so we can now publicly acknowledge that meeting without breaking confidences. The meeting was courteous and constructive, but fell far short of a ‘yes’ decision on Orkambi and Kalydeco, as can be seen from the subsequent Minister’s statement. The Minister made it clear that while he was against the protest today, it would not jeopardise a possible final positive decision. Once again we welcome the support and efforts by Minister Harris and we will continue to engage with him and the Government to ensure we get a positive decision.CFI will also continue to provide support with the CF community at this difficult and anxious time. 

  • Protest outside the Dail Wednesday 1 March 1.15pm

    Cystic Fibrosis Ireland will be supporting a further protest outside the Dail in Kildare Street on Wednesday 1 March at 1.15pm. The aim of the protest is to highlight the urgent need for the government to approve the ground breaking Cystic Fibrosis drugs Orkambi (550 patients) and the extension of Kalydeco to 2-5 year olds and the R117H group (about 25 patients in total). 

    Background: The protest is being led by Jillian McNulty who has CF and who has seen at first hand the benefits of being on Orkambi as she was part of the clinical trials for this ground breaking drug. We urge a further good turn out for this protest. More details to follow.