• Candlelight Vigil in Support of Orkambi

    A group of parents are organising a candlelight vigil in support of Orkambi. While this is not a CFI event we are happy to let our members know in case they wish to support.

    Families and friends of Cystic Fibrosis sufferers in Ireland will be holding candle light vigils at various points throughout the country and in their homes between 7pm and 9pm on Fri 17th Feb in a bid to raise awareness of the battle their loved ones go through daily.

    Check this event on Facebook for further details and to take part -

  • Cystic Fibrosis Ireland Vows to Fight Decision of HSE to Turn Down Ground-breaking CF Drug Orkambi: Calls for Minister Harris to Intervene

    27th November 2016

    Cystic Fibrosis Ireland Vows to Fight Decision of HSE to Turn Down Ground-breaking CF Drug Orkambi: Calls for Minister Harris to Intervene

    Philip Watt, CEO, Cystic Fibrosis Ireland, comments:

    'Cystic Fibrosis Ireland, Ireland's national charity supporting people with cystic fibrosis (CF), has today expressed its dismay and its opposition to the news - leaked to a Sunday newspaper - that the Health Service Executive will not fund Orkambi, the ground-breaking drug for CF.'

    'We will fight this decision and we call on the Minister for Health, Simon Harris TD, to use his Ministerial powers to intervene to ensure that Orkambi is made available in Ireland for the 550 patients who stand to benefit from this life-lengthening drug.'

    'With around 1,200 people with cystic fibrosis here in Ireland, we have the highest rate of the disease per head of population in the world. We also have some of the most severe forms of cystic fibrosis.'

    'It is rare that a therapy comes along that has been shown to reduce hospitalisations by up to 40 per cent and further it significantly retards the progression of cystic fibrosis for those who stand to benefit from this drug. This drug would have resulted in other major savings to the HSE from both reduced hospitalisation and less reliance on other expensive CF drugs and reduced dependency on transplants.'

    'The long-term benefits of Orkambi have been confirmed by Professor Stuart Elborn, formerly of the City Hospital Belfast and now the lead CF clinician in the Brompton Hospital London. The data confirming the impact of Orkambi was presented at the North American CF Conference in October 2016.'

    'Cystic Fibrosis Ireland is angry at the cynical way the HSE has conveyed this information to our patients, some of whom are very ill. The decision was leaked to a Sunday newspaper even before the Minister was informed, never mind patients. It’s a disgraceful and heartless form of communication.'


    For further information: Philip Watt, CEO, Cystic Fibrosis Ireland, is available on 087 637 0557


    Letter Received from Minister for Health Simon Harris on November 27th 2016

    Dear Philip,

    I wanted to write to you today on foot of the article in the Sunday Business Post regarding the drug, Orkambi.

    I am extremely eager that your members, patients with CF and their families are kept fully informed of the process which the HSE has been engaged in with the drugs company, Vertex. I am both annoyed and upset that details seemed to leak regarding these discussions today and I understand the HSE has issued a statement to clarify the position.

    Firstly, let me state that I have not yet received a decision from the HSE Directorate regarding Orkambi. The HSE is responsible for the assessment of new medicines, as you know, which is a statutory process.

    Secondly, following the recommendation of the NCPE, the HSE has been engaged in negotiations with Vertex to try and find a way forward. I am frustrated that this seems to have been extraordinarily difficult. I understand that the HSE has engaged extensively with the company and has sought a significant price reduction.  I fully support the HSE in its attempts to lower the exorbitant price the company is seeking to charge for Orkambi for Irish patients.  It takes two sides to negotiate and Vertex clearly has a critical role in this regard.

    Thirdly, as you are aware, the assessment process is a statutory process. The Oireachtas, in legislating for this process in 2013, gave full responsibility to the HSE.  My Department can only consider requests for funding requirement for a new drug if the HSE decides to reimburse the drug but does not have the resources to fund the medicine. 

    Fourthly, whilst, again, the HSE has not concluded their assessment process, I want to assure CF patients and their families that I do not see this as the end of the process. I want everyone to work to try and find a way forward but let me be clear, this will require Vertex to further engage on a meaningful and innovative basis and to significantly reduce their price.

    Orkambi has not yet been approved for reimbursement by any public health service in Europe. Whilst the drug is available in Germany and France, it is through a very different process. Ireland, the UK, Canada and Australia all find themselves trying to negotiate without success so far. Clearly the time has come for a different approach from Vertex. I now intend to write to fellow Health Ministers in these countries engaged with Vertex on this matter to seek an understanding of their experience and perspective in addressing this.

    I can only imagine how stressful  a time this must be for many families and that is why I want to assure them that we will not leave any stone unturned on this matter – but equally Vertex need to do likewise. In the meantime it is important to remember and to remind CF patients that Vertex have confirmed (as stated on the NCPE website) that patients currently receiving Orkambi under the Managed Access Scheme will continue to receive the drug until reimbursement is finalised in Ireland.

    Philip I will keep you updated and please do keep in touch,

    Best wishes.

    Yours sincerely,


    Simon Harris TD

    Minister for Health

    Letter written by CFI CEO, Philip Watt to Minister for Health Simon Harris on November 27th 2016

    Thank you Minister 

    On behalf of CFI I acknowledge your letter and phone call and we will be responding to the points you raise more comprehensively when I have had a chance to discuss them with our association. From the responses I have heard this morning I know that many families are devastated by the news to refuse funding of the ground breaking drug Orkambi. 

    We take this opportunity to ask for a meeting with you as soon as possible to appeal this decision and to explore if there are grounds in your letter  that this is not the the end of the process. The HSE have been very disrespectful in the way that this has been conveyed to our patients through an apparently cynical leak to a Sunday newspaper at a very late hour and we have had scores of phone calls, texts and messages this morning from very upset parents and people with CF who share our sense of anger. I would request that immediate action is taken on this heartless method of communicating potentially life altering information to patients in this country, some of whom are extremely ill. We ask again that you use your powers as Minister to expedite the reimbursement of Orkambi as soon as possible as we know you have the powers to do so. 

    Yours Sincerely,

    Philip Watt


    Cystic Fibrosis Ireland

  • Decisions on Orkambi and Kalydeco to be made at high level HSE meeting before end of February

    Update 1 February 2017

    Cystic Fibrosis Ireland has received many calls this morning about a headline in the Irish Times (1 February 2017) that states ‘Talks break down over cost of cystic fibrosis drug’.

    We understand after seeking clarification with the HSE and the pharma company (Vertex) that this headline is premature and indeed on closer reading the headline is at variance with the article itself.

    We understand (as stated in the article itself) that discussions within the HSE on Orkambi and the extension of Kalydeco are continuing and that a high level committee at directorate/HSE leader’s level will meet within the next couple of weeks to make a decision.

    Cystic Fibrosis Ireland notes in the Irish Times report the ‘HSE is unlikely to give the go ahead for the drug without a firm indication from Government that extra funding will be available to pay for it’.

    CEO of Cystic Fibrosis Ireland, Philip Watt stated ‘Judging from the amount of calls we received this morning there remains huge anxiety about the forthcoming decision to fund Orkambi and the extension of Kalydeco. This is little wonder as these are the first ever drugs to treat the underlying cause of Cystic Fibrosis (in respect of Orkambi - about 50% of the CF population in Ireland). The increase in lung function of up to 10% and the decrease in hospitalisations of 40% shows how effective Orkambi can be.’

    Philip Watt further stated ‘Cystic Fibrosis Ireland urges the Department of Health and the Department of Finance to avoid an impending row and together agree a mechanism to fund these ground-breaking drugs. We acknowledge the challenges faced by government, but would point out that as well as proven significant health benefits these ground breaking drugs will bring other savings, especially through reduced hospitalisations and less dependency on other expensive medications and treatments.’ 

  • EU Approval for Orkambi in Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation 

    Cystic Fibrosis Ireland welcomes the decision of the European Medicines Agency (EMA) to extend the Cystic Fibrosis drug ‘Orkambi’ to children aged 6 to 11 years old. Previously the drug had been available to young people and adults aged 12 years and over in Ireland and the rest of the European Union, where approved for reimbursement.

    Orkambi was approved for reimbursement by the Irish Government in April 2017. As part of that agreement, in a unique 'pipeline deal’, it was agreed that future extensions of Orkambi or Kalydeco (or future drugs that improve on Orkambi or Kalydeco from Vertex pharmaceuticals), would be made available to people with CF in Ireland.

    Cystic Fibrosis Ireland calls on the Minister for Health to ensure that children with CF who have the potential to benefit from this extension of Orkambi are given the choice of accessing this groundbreaking and innovative drug as soon as possible (subject to the advice of their consultant).

    Orkambi treats the most common CF gene alteration/mutation in Ireland (and the world) - that is people who have 2 copies of the F508del alteration. It is difficult to make a precise forecast of the number of additional children that will benefit from the extension of Orkambi, but we would estimate that up to 50 children in Ireland will  ultimately benefit from this new decision. 

    A statement from Vertex on the Approval of Orkambi in children with CF, ages 6 - 11, with two copies of the F508del Mutation can be viewed by clicking here.

    Philip Watt
    Chief Executive
    Cystic Fibrosis Ireland

  • Italy has followed Ireland and other EU countries such as Germany, Austria, Denmark and Luxembourg in approving Orkambi for patients with CF who have the particular genotype that stand to benefit from this important drug therapy.

    CF Ireland hopes the efforts of the CF Trust in the U.K. will soon result in Orkambi being reassessed positively by regulatory authorities in London, Edinburgh and Belfast. There are many patients in Northern Ireland who could benefit from this drug and we offer our mutual support to them in particular at this time of uncertainty. 


    Statement from Vertex Pharmaceuticals

    Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Italian Medicines Agency (Agenzia Italiana del Farmaco, or AIFA) has agreed to reimburse ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The agreement is published online in the Italian Official Gazette. Regional authorities will now begin implementation to provide the hundreds of eligible patients in Italy access to this important medicine. Recent European pricing and reimbursement agreements have enabled broad access to ORKAMBI for thousands of eligible patients in Austria, Denmark, Germany, Ireland, Italy and Luxembourg. Negotiations continue in a number of other countries where CF is prevalent, including France and the United Kingdom.

    "We are pleased to have reached this agreement on behalf of CF patients in Italy who have been waiting for this important medicine," said Simon Bedson, Senior Vice President and International General Manager at Vertex. "We continue negotiations with other countries including France and the United Kingdom, and we encourage these national health authorities and governments to work quickly with us to achieve reimbursement for all patients who may benefit."

    About ORKAMBI® (lumacaftor/ivacaftor) and the F508del mutation

    In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

    ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. ORKAMBI is available as tablets and is typically taken twice per day.


    For complete product information, please see the Summary of Product Characteristics that can be found on

  • Statement from Cystic Fibrosis Ireland
    Continuing lack of deal for Orkambi in England for people with cystic fibrosis will also impact on Northern Ireland

    Date of issue: July 5, 2018

    Cystic Fibrosis Ireland (CFI) is very disappointed to learn today (5 July 2018) that there continues to be no agreement for the provision of Orkambi and future cystic fibrosis (CF) drugs in England as negotiations have once again broken down without agreement between NHS England and Vertex Pharmaceuticals, despite a new deal being put on the table.

    The failure to find an agreement also impacts on people with CF in Northern Ireland as it has been made clear by the Northern Ireland Secretary of State that they will be guided by decisions on new medications by NHS England. 

    Philip Watt, CEO of CFI, stated:

    “The absence of an Assembly in Northern Ireland means that there is very limited opportunity for regional pressure to be brought to bear on behalf of patients in Northern Ireland, except by individual politicians and the fantastic work of our colleagues in the UK-based patient group Cystic Fibrosis Trust, including its CEO David Ramsden.

    “We know from our experience in Ireland, that CF patients in the UK and their families will feel devastated today by this cruel and harsh decision.

    “The decision not to fund Orkambi appears to be final and is all the more inexplicable given the fact that the drug was recently approved in the Netherlands and Sweden. Both of these countries adopted the innovative ‘Irish solution’ of providing Orkambi and providing rapid access to future drugs that are already showing excellent results in advanced clinical trials, as part of the one deal. Sweden, in particular, has a very similar drug approval system to the UK.

    “We would call on the Taoiseach, Minister for Health and politicians in the Republic of Ireland and Northern Ireland to speak out and show solidarity on this issue. While this decision will be devastating news throughout the UK for those with CF and their families, it will be particularly devastating for those in Northern Ireland who know that these innovative and ground-breaking drugs are available only a few miles south of the border and our thoughts and support are with them today.”


    For more information:

    Philip Watt, CEO, Cystic Fibrosis Ireland, email This email address is being protected from spambots. You need JavaScript enabled to view it.  / telephone 087 637 0557

    Issued on behalf of Cystic Fibrosis Ireland by Don Delaney, d2 communications, email This email address is being protected from spambots. You need JavaScript enabled to view it. / telephone 087 793 3249

    Click here to read the statement issued by CF Trust.


  • Letter to Tony O Brien, Chief Executive HSE, Re: Orkambi negotiations

    Yesterday Philip Watt, CEO of Cystic Fibrosis Ireland sent this letter to Tony O Brien in the HSE regarding the negotiations between the HSE and Vertex. A copy of this letter was also sent to the Minister for Health Simon Harris, John Hennessy, National Director, Primary Care in the HSE and Michael Barry, Director of the National Centre for Pharmacoeconomics.


    15 December 2017  

    Dear Mr O Brien,

    In relation to Orkambi, we greatly welcome the fact that negotiations have recommenced between the HSE and Vertex and we urge both parties to take this important opportunity to negotiate with a view to reaching a compromise that is fair to both parties and most important of all is fair to patients with CF in Ireland.

    We would further urge both parties ensure that these negotiations are held in camera and not in public to give the negotiations a real chance of success. We have written to Vertex along similar lines with the hope that a sustainable compromise can be reached.

    As the national patient body for patients with CF we are not party to the negotiations but we would offer the following observations arising from recent public discourse

    1. All of the positive benefits of Orkambi need to be considered when assessing the impact of Orkambi. These include the improvements to lung function; the reduction in the rate of exacerbations; improvements in weight and improvements in overall quality of life. CFI recognizes that these impacts will vary from one patient to another because of the nature of CF.

    2. Preventing the inevitable rate of lung function decline needs to be full taken into account. Recent long-term data published at the North American CF conference demonstrates that Orkambi slows this decline by 42% over a 2 year period.

    3. CFI has and will continue to speak out against inequities in the international pharmaceutical industry including the level of CEO salaries, however our patients cannot afford to wait for such major systemic reforms, as their health needs are in more critical and immediate. We note that these inequities are not confined to Vertex.

    4. We note that the Government’s National Plan on Rare Disease recommended the consideration of a new system of assessing orphan drugs (drugs for rare diseases) we would urge that this recommendation 30 is implemented as soon as possible.

    Yours Sincerely,

    Philip Watt

    CEO CFI 

  • 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 Political Party Leaders from CFI and Jillian McNulty (advocate)

    An open letter to party leaders will be handed in during the protest outside the Oireachtas on Wednesday 7th December 1.30-2.30pm in Kildare Street.

    The letter is from CFI (CEO Philip Watt) and Jillian McNulty, person with CF an independent advocate who has benefitted from the ground breaking drug Orkambi.

    You can download the letter here or read it below.



    Dear Party Leader,

    We are writing to you to ask your support to ensure that meaningful and substantive discussions on the reimbursement of the ground-breaking drug Orkambi are continued by the Health Service Executive (HSE) and the pharmaceutical company Vertex.

    We would further urge that negotiations commence and are concluded positively and swiftly on the reimbursement of Kalydeco for 2-5 year olds, which impacts on about 18 children in Ireland.

    As representatives of patients we feel very much caught in the middle of the current stand-off between the HSE and Vertex Pharmaceuticals on a price for Orkambi.

    There continues to be a high level of anxiety among the families of the 500 plus CF patients who stand to benefit from Orkambi – the first to treat the underlying cause of CF in this group of patients. Families have expressed dismay to us that they fear they may never receive this drug or its availability may be significantly delayed.

    We continue to urge Vertex to reduce the price of Orkambi and enter into an affordable shared-risk agreement with the HSE. We make clear again we do not support the excessive payments to senior staff in any international pharmaceutical companies, including but not confined to Vertex

    We continue to urge the HSE to give a more complete and less selective analysis of the impact of Orkambi. In particular the HSE should explicitly recognise the importance of a 40% reduction in hospitalisations and the outcomes of long studies published in October 2016 that demonstrate how Orkambi slows the progression of CF, including the decline in lung function. It is a matter of concern that the HSE have not done so to date.

    The role of exacerbations (worsening of CF leading to hospitalisations) in progressing CF from which 25% of patients do not recover their baseline lung function, has been widely recognised in CF research. 

    We fully support the Minister for Health in his quest for international support to improve Ireland’s bargaining position on expensive drugs but we are concerned this may not result in short/medium term breakthroughs in relation to access to Orkambi for Irish patients. Orkambi is too important to be delayed.

    We re-state our belief that the Minister and this government and opposition parties are compassionate and sincere about CF but we would also call for efforts to be redoubled to find a way past this present impasse.

    We would point out that since 2012, 32 new drugs for cancer have been approved for use in Ireland. This is to be strongly welcomed. However there have only been two new types of Cystic Fibrosis drugs made available in Ireland over the past 20 years and Orkambi is one of these drugs and this should also be taken into consideration. In short, new drugs for rarer diseases are rare in themselves.

    Finally, we would also seek you support the extension of Kalydeco to 2-5 year olds in Ireland. We understand negotiations on this are about to commence. We would urge that a decision is taken swiftly as Kalydeco is already available to 6 year olds and over in Ireland.

    Note: This extension is already available to patients in England, Scotland, Germany, Wales, Netherlands, Belgium and the US. There are 18 children who would stand to benefit from this extension in Ireland.

    Yours sincerely,

    Philip Watt, CEO CFI and Jillian McNulty, Independent CF Advocate




  • 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 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 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.

  • 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 (after about one minute of adverts).  

  • Photos from the Orkambi Protest

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
















  • Response from Vertex

    VertexOn Tuesday 29th November, CEO Philip Watt wrote an open letter to Vertex Pharmaceuticals regarding the negotiations around Orkambi. You can download Vertex's response below.


  • Talks to Re-open on Orkambi: Important statements by Minister and Vertex, 7 December 2016


    Cystic Fibrosis Ireland (CFI) welcomes the statements by Minister for Health Simon Harris TD and from Vertex Pharmaceuticals that talks on Orkambi will reopen. CFI sincerely hopes that a settlement will be reached that will be fair to both the State and the pharma company and most important of all, which makes Orkambi available in Ireland to people with CF who stand to benefit from this important drug.

    Statement from Minister for Health Simon Harris TD, 7 December 2016 (see below)
    Statement from Vertex Pharmaceuticals, 7 December 2016
    Press report from Paul Cullen, Irish Times, 7 December 2016 (for information)


    Statement by Mr Simon Harris TD, Minister for Health on Orkambi
    7 December 2016

    Minister for Health Simon Harris has said he is pleased to learn that Vertex has responded to the HSE with a view to re-engage in negotiations with them on Orkambi. Speaking from a meeting with EU Health Ministers in Lisbon today (Wednesday) on drug price negotiations, the Minister said:

    "I have always said that Vertex need to re-engage in a meaningful way with the HSE that addresses the core issue of price and affordability. In that context the company must return to the table with a significantly better offer. This has not happened to date and I again call on the company to re-engage in a meaningful way. However, I was pleased to learn that Vertex has responded to the HSE today with a view to re-engage in negotiations with them on Orkambi.

    "In a meeting with some of my EU counterparts, I raised this issue of the unacceptably high price Vertex has put on this drug, making it inaccessible not just for Ireland's CF patients but for other patients around Europe. I intend to continue to intensify my engagement with colleagues in Europe and indeed other countries on this issue.

    "I want to see CF patients receive access to the best treatments possible. That remains my priority."



  • The battle for Orkambi and Kalydeco: CFI Update 15 December 2016

    Where are we at now?

    Orkambi: Following sustained pressure from the CF community, including CFI, the HSE and Vertex agreed to enter into new negotiations on the 7th of December. The first of what is likely to be a series of meetings took place on the 14th of December and a further meeting is likely on the week beginning the 19th of December. As of 15th December, no decisions have yet been reached. It is estimated around 550 CF patients in Ireland stand to benefit from Orkambi. These are people with CF 12 years and over that have the relevant genotype which is 2 copies of the F508del gene alteration (the most common CF gene alteration in Ireland and worldwide).

    Extension of Kalydeco 2-5 year olds: It has been further agreed that negotiations will begin on the reimbursement price of the extension of Kalydeco to 2-5 year olds, which impacts on 18 children with CF in Ireland. This is part of the ongoing negotiations with Vertex and as of the 15th of December, no decisions have yet been reached.

    How long will the negotiations last?

    We don’t know yet. CFI are urging both the HSE and Vertex to conclude the negotiations as soon as possible. It is important to find a balance between urgency and getting a workable and sustainable agreement. As with all negotiations, there needs to be give and take and CFI have urged that negotiations should not be undertaken over the airwaves.

    Is the HSE right in saying Orkambi only impacts on 25% of the eligible group of patients?

    No: The HSE has persisted in stating over the past 2 weeks that only those who have experienced a 10% increase in lung function are those really benefitting from Orkambi (which they estimate at about 25% of the eligible CF population). CF is a multi-organ and multi-dimensional disease and as well as lung function, other issues such as exacerbations, slowing the progression of the disease, weight; general health gain and improvement in life quality should also be taken into account. Further, a 3-4% increase in lung function can make a significant difference in terms of health gain, especially over time and compared with someone not on Orkambi.

    Is CFI right to emphasise the importance of exacerbations in measuring the impact of Orkambi?

    Yes: Exacerbations are the worsening of CF resulting in hospitalisation. Orkambi has shown a 40% decrease in exacerbations. Our view is backed up by hard scientific evidence. For example Dr DB Sanders et al have demonstrated in a widely respected research article that 25% of CF patients do not recover their baseline lung function once they have had an exacerbation. (Source: Failure to recover to baseline pulmonary function after cystic fibrosis pulmonary exacerbation.) CFI has urged the HSE to take these issues into account.

    Why are drugs for rarer diseases so expensive and why do they get turned down?

    Innovative and effective Drugs for rare disease (usually referred to as orphan drugs) are often expensive to produce and, by definition, will benefit only small numbers of patients. After assessment, few orphan drugs get close to meeting the cost effectiveness criteria for funding that healthcare providers (such as the HSE/NCPE) use for general drugs. This is a problem that is not confined to Ireland.

    What about the expensive salaries to CEO’s of pharma companies?

    Our members will not be surprised when we tell them CFI can’t sort out world pharma-economics, including the outrageous salaries of the CEO’s of most major pharma companies. We are at the end of the day a patient group trying to get important new therapies for our patients and their families. CFI have been and will remain very critical of such salaries as highlighted again by CFI ambassador Joe Brolly on the recent Claire Byrne Show and in a Sunday Independent article. Note for information: The following bio-pharma companies pay their CEO’s more than $20m per annum: Regeneron: Vertex: Allergan; Bristol Myers Squibb; Merck; Celgene; Pfizer and Abvie. In short the issue of high CEO salaries is a systemic problem in the bio-pharma industry.

    Surely there has to be a better way of dealing with CF and other rarer disease drugs?

    The Rare Disease Taskforce that brings together many key non-government stakeholders, in particular the members of MRCG; IPPOSI and GRDO. We work together with many other patient groups to seek to implement the Government’s National Rare Disease Plan. Recommendation 30 of the Plan acknowledges the problem of accessing rare disease (orphan) drugs and proposes the review of the existing and the consideration of a new process. We will continue to press for this recommendation to be implemented as soon as possible.

    CFI will continue to keep our members informed about further developments including further concerted action if necessary.


    Failure to recover to baseline pulmonary function after cystic fibrosis pulmonary exacerbation.

  • Update on Orkambi and Extension of Kalydeco 

    May 30th 2017 

    CFI has been reliably informed that patients will not have to wait much longer to gain access to Orkambi and the extension of Kalydeco. We understand the final arrangements on the roll out of these important drug therapies is nearing completion and has progressed significantly since our last update. Please note that clinicians will also have to make preparations in relation to assessment and monitoring of these drug therapies on a regular basis. With around 600 patients and many specialised centres involved, there is an inevitable lead-in time before all preparations are in place. This is important to ensure the health and safety of CF patients. We will update you further when we get further news but would urge patience and understanding in the mean-time.