• 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

  • Good News on Orkambi and Kalydeco

    CFI has confirmation from both Vertex and the HSE that the final contracts for Orkambi and Kalydeco extension and pipeline drugs have been signed and it is likely that some CF centres will be beginning to get the drugs as early as next week.

    Please note: It will still take a while for all eligible patients to receive the drugs as there are plus 600 patients involved and multiple CF centres. Individual assessments are important for health and safety reasons. In this context we continue to urge reasonable patience.

    CFI will continue to work to ensure access to proven pipeline therapies when they become available, including for those who are not presently eligible for a bespoke CFTR drug therapy.

    Click here to read the Vertex press statement in full.

  • 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 

  • 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.

  • 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 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.

  • 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.

  • The Pipeline Aspect of the Recent Vertex and HSE Agreement

    A couple of CFI members have asked us to help clarify the ‘pipeline aspect’ of the recent HSE/Vertex agreement. This is an innovative aspect of the deal and we understand it has never being implemented before in Ireland and may even be unique in Europe. CFI pushed for and supported the pipeline dimension during the negotiations and we acknowledge the positive role of Vertex, the HSE and Minister Harris in this development. 

    It is important to recognise that while this is a very positive and innovative development, it will not (of course) be a panacea for resolving all future CF drug therapy reimbursements. 

    CFI understands the HSE/Vertex agreement currently covers:

    • Orkambi for all CF patients 12 years and older who have two copies of the F508del alteration 
    • Expands access to Kalydeco for children aged 2 to 5 with any approved gating alteration (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D) 
    • Access to Kalydeco for people aged 18 and older who have an R117H mutation
    • Rapid access for people with all the above alterations (ie: two copies of the F508del mutation, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D, R117H) if the labels of the existing medicines are expanded to cover additional age groups and if new Vertex medicines are approved for these populations

    If, for example, a drug therapy is developed (either by Vertex, or indeed another pharma company) for the gene alterations not listed above, there would have to be further discussions between the company and the HSE as and when required.

    The bottom line is that we estimate that as a result of the Orkambi and the previous Kalydeco agreement of 2013 that around 65% of the CF population in Ireland (and probably more) will have now access to a bespoke CFTR drug therapy, with more pipeline drugs for those groups to follow. This is important as the impact of Orkambi can vary from one individual to another. 

    Needless to say, CFI will continue to adopt a robust approach to potential reimbursement of future drug therapies including in particular for those who do not have a bespoke drug at present, provided the CFI Board is convinced on the efficacy, safety and impact of such drugs. In short, the 35% of CF patients without a CFTR drug in Ireland will remain an absolute priority for CFI. It is worth noting the indirect impact of the reimbursement of Orkambi will be to encourage further research into ground breaking CF drugs. Further, the CFF in the US continues to invest millions of dollars into further drug therapies, including those without a bespoke therapy at present. In short this agreement is a major step forward but there remains so much more to do. 

    In consultation with expert clinical advice, CFI will also be also publishing important advice to our members about Orkambi and the need to continue other treatments and the need to keep up a good diet and exercise regime.