Kalydeco extension

Kalydeco extension

  • ‘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’. 

  • CF Drugs Update: January 2019

    Orkambi, Kalydeco and Smykevi: More Good News


    Cystic Fibrosis Ireland welcomes the news that Orkambi has been approved by the EMA (European Medicines Agency) for children aged 2-5 years old with 2 copies of the F508del gene alteration. 

    Orkambi was originally approved for reimbursement in Ireland for 12 years and older in 2017. Orkambi was subsequently extended to 6-11 year olds in 2018. The further extension of Orkambi to 2-5 years olds approved by the EMA is consistent with the drugs portfolio deal agreed in April 2017 between the HSE and Vertex Pharmaceuticals. 

    CFI will seek clarification from the HSE on timing of availability of the latest Orkambi extension in Ireland. 


    CF Ireland understands that the introduction of Symkevi (approved on 1 January 2019) is going very well. A small number of patients have been switched to Symkevi who were originally on Orkambi, based on clinical criteria and expert advice from Consultants. However CFI also understands that the vast majority of those on Orkambi will remain on Orkambi as CFI predicted in earlier news updates.

    Symkevi is a CFTR medication that is a possible alternate for those eligible for Orkambi and also for a small number of other linked gene alterations


    The EMA have also recently approved the extension of Kalydeco to 1-2 year olds. 

    Cystic Fibrosis Ireland is also seeking clarification if Kalydeco will be extended to those under 18 years of age with the R117H gene alteration. 

    Kalydeco was the first CFTR drug introduced to Ireland in 2013 of which Orkambi and Symkevi have since followed (2017 and 2019). These are the first drugs that treat the underlying cause of CF. They are personalised drugs in the sense that they are tailored for those with certain gene alterations.

    Kalydeco impacts on those with the G551D gene alteration (15% of the CF population in Ireland) but also other named gene alterations including R117H (6% of the CF population in Ireland). The most common gene alteration in Ireland and worldwide is people with CF with 2 copies of the F508del gene alteration (about 57% in Ireland). This is the group of patients eligible for Orkambi and Symkevi

  • 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.’ 

  • 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.

  • 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.

  • 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




  • Orkambi and Kalydeco Update

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

  • Orkambi and Kalydeco 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. 

  • Protest and March

    Wednesday 12th April Kildare Street, Dublin at 1pm

    To support access to Orkambi and Kalydeco Cystic Fibrosis drug therapies


    Assemble 1pm sharp outside the Dail

    Wear something purple and bring a whistle

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

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

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

    The protest will also include a short march.

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

  • Protest outside the Dail Wednesday 1 March 1.15pm

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

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

  • 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. 

  • Update on Orkambi/Kalydeco drug therapies 23 February 2017

    Many thanks to Joan Collins TD who sent these answers to parliamentary questions to CFI. 



    QUESTION NOS: 197 to 199

    DÁIL QUESTIONS addressed to the Minister for Health (Simon Harris T.D.)
    by Deputy Joan Collins
    for WRITTEN ANSWER on 22/02/2017

    * To ask the Minister for Health if the HSE high level officials group has made a recommendation on the reimbursement of Orkambi and the extension of Kalydeco for the two to five age group and the R117h group of persons with cystic fibrosis eligible for these vital drugs; and if he will make a statement on the matter.

    - Joan Collins T.D.

    For WRITTEN answer on Wednesday, 22nd February, 2017.

    * To ask the Minister for Health if the HSE approval on the reimbursement of Orkambi and the extension of Kalydeco for the two to five age group and the R117h group contingent on additional funding is being provided by government to the HSE..

    - Joan Collins T.D.

    For WRITTEN answer on Wednesday, 22nd February, 2017.

    * To ask the Minister for Health if the Cabinet will be making the final decisions relating to the reimbursement of Orkambi and the extension of Kalydeco for the two to five age group and the R117h group; if so, when; and if not, the body that is making the final decision.

    - Joan Collins T.D.

    For WRITTEN answer on Wednesday, 22nd February, 2017.

    The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drugs schemes, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013.

    In reaching its decision, the HSE examines all the evidence which may be relevant in its view for the decision (including the information /dossier submitted by the Company) and will take into account such expert opinions and recommendations which may have been sought by the HSE at its sole discretion (for example, from the National Centre for Pharmacoeconomics).

    In considering an application, the HSE will also have regard to Part 1 and Part 3 of Schedule 3 of the 2013 Act. Part 3 requires the HSE to have regard to the following criteria:

    1. the health needs of the public;
    2. the cost-effectiveness of meeting health needs by supplying the item concerned rather than providing other health services;
    3. the availability and suitability of items for supply or reimbursement;
    4. the proposed costs, benefits and risks of the item or listed item relative to therapeutically similar items or listed items provided in other health service settings and the level of certainty in relation to the evidence of those costs, benefits and risks;
    5. the potential or actual budget impact of the item or listed item;
    6. the clinical need for the item or listed item;
    7. the appropriate level of clinical supervision required in relation to the item to ensure patient safety;
    8. the efficacy (performance in trial), effectiveness (performance in real situations) and added therapeutic benefit against existing standards of treatment (how much better it treats a condition than existing therapies); and
    9. the resources available to the HSE.

    I am informed that, following a request from the HSE, the NCPE carried out an assessment of the manufacturer's economic dossier submitted in March 2016 on the cost effectiveness of Orkambi and Kalydeco. This dossier included details on all relevant costs and relevant cost offsets including those associated with hospitalisation, disease management costs, intravenous antibiotics, adverse events and any additional costs arising in patients not taking Orkambi or Kalydeco.

    The NCPE has completed its Health Technology Assessment and this is available on its website. It was submitted to the HSE in June 2016. The NCPE determined, following an evaluation of the economic dossier, that the manufacturer failed to demonstrate cost-effectiveness or value for money from using the drug. The NCPE have confirmed that all relevant costs were included in the analysis. In line with the HSE's assessment process, the HSE Drugs Committee considered the NCPE recommendation, the manufacturers submissions and commercial offer and other information. Following this consideration the Drugs Committee did not recommend reimbursement at the current price. The HSE Directorate considered the Drugs Committee’s recommendation in December and the Directorate took the decision not to reimburse at the current price offered by the company.

    The HSE has since re-entered into negotiations with Vertex, the manufacturer of Orkambi and Kalydeco, with a view to significantly reducing the cost of both drugs. A meeting was held in December and again in early January. Following completion of the negotiation process, I am advised that the HSE’s Drugs Committee met at the end of January and considered the manufacturer's latest price offerings. No decision has been reached regarding Orkambi and discussions are ongoing with regard to Kalydeco. The matter is currently under consideration by the HSE Directorate.

    I understand how patients and their families must feel in these circumstances, as they await the decisions by the HSE in relation to reimbursement. However, as with all new drugs developed, the HSE must follow a statutory process, as set out in the 2013 Act. 



    Delegation that met with TD's and Senators in the Dail on 22 February to give an update on Orkambi/Kalydeco.