Orkambi

Orkambi

  • ‘Government to sign off on CF drug deal next month’ (Sunday Business Post)

    Please see below an article from the Sunday Business Post which predicts the approval of Orkambi ‘next month’. This follows on from recent positive comments by the Minister for Health Simon Harris TD on both Orkambi and the extension of Kalydeco on RTE radio and his comments about the likelihood of a deal on CF pipeline drugs to be included in the forthcoming agreement with Vertex.

    While CFI will remain cautious until final approval is given, it is clear that all the signs are indicating a yes decision in coming weeks, which will be fantastic news for CF patients in Ireland who are waiting for both Orkambi and the extension of Kalydeco. CFI wishes to acknowledge the continuing commitment of Minister Harris and the Taoiseach in respect of new and innovative therapies and to the party leaders and health spokespersons of opposition political parties who have highlighted this issue in the Dáil in recent months with very significant effect.

     

    CFI in particular wishes to acknowledge the parents and people with CF who have spoken so courageously to the national media in support of these new therapies and the ongoing support of the Board of CFI led by Patricia Duffy Barber. While many have been involved and we thank you all, we would like to single out Jillian McNulty in particular for her work in this regard. Thanks also to the Whitmarsh family (Ronan and Linda) and to Hazel Robinson too and all on the coordination group. ‘Your voice and support has made all the difference’. 

  • 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 - https://www.facebook.com/events/1866532033630400/?ti=icl

  • Cystic Fibrosis Ireland met with Minister for Health Leo Varadkar and senior officials for the Department of Health on Monday 25th January 2016.

     A delegation from Cystic Fibrosis Ireland and a member of our Medical and Scientific Council (clinician) and a patient with CF met with Minister Leo Varadkar on Monday 25th January 2016.

    The meeting was wide ranging but with most focus on:

    1. Access to new and innovative therapies for people with CF (in particular Orkambi);
    2. Concerns about chronic staffing shortages in CF centres; 
    3. The need for progress in relation to Beaumont Hospital (adult in patient rooms- 20 needed only 6 available) and Cork University Hospital (paediatric inpatient and outpatient facilities);
    4. Transplant issues (including pre and post- transplant rooms and the introduction of soft opt out organ donation).

    CFI is also seeking return of €800,000 we have paid to the government in VAT arising from huge fundraising efforts to improve 7 CF Hospital centres around the country. This would be reinvested in vital CF services, including for example Beaumont Hospital and Cork University Hospital, if returned to CFI.

    A detailed briefing paper was drawn up by Cystic Fibrosis Ireland for the meeting.

     

    The main outcomes of the meeting were as follows:

    • The Minister and his officials received first-hand information about the impact of Orkambi, including the dramatic reduction in exacerbations/hospitalisations resulting from the drug (40% decrease).
    • A petition signed by over 40,000 people supporting access to Orkambi was handed to the Minister demonstrating how strongly patients and the public feel about ensuring CF patients have access to Orkambi.
    • There was powerful testimony at the meeting from a patient with CF (Jillian McNulty) who has benefited from being on Orkambi as part of the clinical trials and who has remained on the drug since the trials. She has experienced a dramatic reduction in hospitalisations and a general improvement in well-being. This is also reflected in other patient testimonies CFI has received.
    • CFI pointed out that Orkambi is expensive because of the huge costs of developing this drug and the relatively few patients (worldwide) from whom the pharma company can seek to recoup their costs and to invest in further research. CFI supports the government in getting the fairest price for Orkambi.
    • There will be very significant savings to the government arising from the dramatic decrease in hospitalisations arising from those on this drug.
    • The Minister told CFI that if Orkambi was positively assessed by the National Centre for Pharma Economics/HSE, additional funding from government, on top of the present health budget, would be sought. (Thus this drug will not impact on other health services- CFI).
    • The Minister and his officials promised that Orkambi would be assessed fairly and as efficiently as possible and they are awaiting further documentation from the drugs company who have produced Orkambi.

    Other outcomes.

    • The Minister promised that an audit of staffing levels in CF centres would be undertaken as part of the National Clinical Programme and would be completed shortly. The outcomes would then be considered by government.
    • The Minister said that ‘soft opt out organ’ donation would in included in the Fine Gael Manifesto and (if elected) in the next programme for government
    • The Minister said he was concerned about the high level of VAT tax paid by CF for hospital buildings and this issue would be further investigated. He knew from experience it also affected other charities. 
  • CFI says thanks to the Government and all Political Parties for support for Orkambi and Kalydeco

    Cystic Fibrosis Ireland wishes to acknowledge Government, and all political parties for their support in ensuring Orkambi and Kalydeco was approved

    Among the many we would like to thank are:

      • An Taoiseach, Enda Kenny TD; Minister for Health Simon Harris TD and Finian McGrath Minister for Disabilities 
        Fianna Fail leader, Michael Martin TD; Health spokesperson Billy Kelleher TD; Marc MacSharry TD
        Sinn Fein leader, Gerry Adams TD and Health spokesperson Louise O Reilly 
        Labour spokespersons, Jan O’Sullivan TD and Brendan Ryan TD 
        Solidarity–People Before Profit
        Independents 4 Change, in particular Joan Collins TD 
        Rural Alliance TD’s

     

    There are many more not included on this this who supported us over the past 10 months – many thanks to all.

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

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

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

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

    Philip Watt

    CEO Cystic Fibrosis Ireland

  • 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.'

    ENDS

    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

    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

    CEO 

    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.’ 

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

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

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

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

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

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

     

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

     

  • 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


  • Extension of Orkambi to children aged 6-11

    The recent decision of the European Medicines Agency (EMA) to extend the CF drug “Orkambi” to children aged 6 to 11 years old, on the basis of clinical research emerging from two Phase III studies (NCT02514473 and NCT01897233), means that this ground breaking drug which had previously only been available to those aged 12 years and older with two copies of the F508del mutation, will now also be available to children aged 6-11 (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 two copies of the F508del alteration. 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 (including this EMA decision to extend Orkambi to children aged 6-11), would be made available to people with CF in Ireland.

    Commenting on the extension of Orkambi to children aged 6-11, the Minister for Health Simon Harris said:
    “It’s very positive news that Orkambi is now licenced and available for reimbursement in Ireland for CF patients aged 6 to 11.”

     

  • Germany approves award winning Cystic Fibrosis drug ‘Orkambi’

    Philip Watt CEO of Cystic Fibrosis Ireland welcomes today’s news (19 December 2016) that a pricing and reimbursement agreement for the ground breaking Cystic Fibrosis drug Orkambi has been reached with the German Statutory Health Insurances (GKV-SV).

    According to a press release issued by Vertex today, ‘the German Federal Joint Committee (G-BA) recognized the “considerable additional benefit” of Orkambi for people with CF who have two copies of the F508del mutation’.

    Orkambi has been available to eligible patients in Germany under their approval system (different to Ireland) that allows a drug to be made available on a one year trial basis once it has been approved by the European Medicines Authority (EMA). The EMA approved Orkambi in November 2015. Under the German system, authorities have a 12 month opportunity to assess the effectiveness and proposed pricing of a drug.

    Orkambi is now available to eligible people with CF in Germany, the United States and Austria. France has a similar system to Germany and is due to consider Orkambi in March 2017. CFI further notes that Orkambi has recently received the ‘Drug Discovery of the Year Award’ award from the British Pharmacological Society and the French ‘Prix Galien’ award for the most promising rare disease medicine in 2016.

    Cystic Fibrosis Ireland recently welcomed the news that the HSE and Vertex have entered into renewed negotiations on the potential of making Orkambi available in Ireland. CFI has urged that these negotiations are carried out in private and not over the airwaves and a fair agreement is reached as soon as possible. Philip Watt stated ‘both sides should make compromises in order to reach a fair agreement, including a considerable reduction in price from the manufacturer, Vertex and a greater acknowledgment by the HSE of the very high costs and risks involved in developing a drug for rarer diseases such as CF’.

    Philip Watt further stated: ‘CFI calls for an overhaul of the system of assessing drugs for orphan diseases (rare diseases) in Ireland, including consideration of adopting the French or German drug approval systems and/or adopting a similar approach to the way cancer drugs are assessed and approved in 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.



  • 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 www.ema.europa.eu


  • 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.”

    ENDS

    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.

     

  •  International study published in Lancet shows that Orkambi improves the lung function of children with CF in less than 2 weeks.

    24 July 2017

    The international study, which included researchers from Imperial College London at the Royal Brompton Hospital, showed that Orkambi can improve the lung damage caused by the genetic disease in under two weeks.

    The findings of the phase 3 trial involving 204 patients aged 6 to 11 years old were recently published in The Lancet Respiratory Medicine. Half the patients received Orkambi for six months, while the other half received a placebo.

    At the end of the treatment period, researchers found two key improvements in markers of health amongst those who received the drug. Firstly, the children’s airways were significantly healthier based on a sensitive test of gas mixing in the lungs. Secondly, there was a 20 per cent reduction in the amount of chloride in their sweat – a common symptom of the disease.

    Lead author Professor Felix Ratjen, of The Hospital for Sick Children at the University of Toronto, told the New Scientist: “We’ve shown that even in younger patients who have relatively mild disease, Orkambi led to improvements in lung function.”

    Unlike Ireland, Orkambi is not yet available on the UK under the National Health Service.

     Cystic fibrosis (CF) is caused by a defect in the gene cystic fibrosis transmembrane conductance regulator (CFTR), which codes for a protein that maintains the balance of salt and water in and out of cells. This leads to a build-up of thick and sticky mucus in the lungs and digestive system. Orkambi is one of the first therapies to target the commonest form of the underlying faulty CFTR protein rather than the symptoms.

  •  

    Letter to the Minister for Health
    Leo Varadakar TD

    Dear Minister, 

    We write to you to express our dismay that a statement has been made by the HSE to a Sunday newspaper which appears to state that a forthcoming groundbreaking drug ‘Orkambi’ will not be funded by the Irish government, irrespective of how good it is.

    We seek a meeting with you to discuss this issue urgently.

    This statement has caused considerable anxiety among the CF population in Ireland. Around 60% of people with CF in Ireland stand to benefit from this drug which is effective for the most common CF gene alteration in Ireland, which is Delta F508. It is the first drug to impact on the underlying cause of CF among people with two copies of Delta F508. 

    We welcome the subsequent statement made by you as Minsiter that this drug will be assessed and additional funds will be sought to fund the drug, if it is approved.

    Cystic Fibrosis Ireland urges you to ensure that this drug is assessed fairly and without the prejudice that appears to be have been demonstrated on this occasion. The statement made by the HSE comes before a Rapid Review or Health Technology Assessment was undertaken by the National Centre for Pharma-economics (NCPE).

    This drug is vital for people with CF in Ireland as:

    • It is the first drug to impact on the underlying cause of CF for 60% of the CF population in Ireland.
    • The drug has shown up to a 40% reduction in worsening of CF requiring hospital admissions
    • It has shown a sustained increase in lung function
    • It has shown a sustained increase in weight, an important issue in CF care

    The drug has the potential to lenghten lives; increase quality of life and delay the need for lung transplantation. We know this from first hand as Irish patients have been part of the trials for this important drug. 

    Yours sincerely, 

    Philip Watt

    CEO Cystic Fibrosis Ireland 

     

  • 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 Simon Harris' comments

    Cystic Fibrosis Ireland (CFI) warmly welcomes the comments by Minister Simon Harris on RTE’s Sean O Rourke programme Monday 6 March 2017 which signifies that further significant progress has been made towards the approval of Orkambi and the extension of Kalydeco. While we will remain cautious until a final decision is made, we look forward to the Minister making a final ‘yes’ decision within the next few weeks. 

    You can watch the video on Facebook here.

  • 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