Orkambi

Orkambi

  • 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.

     

  • Calling all people with CF, parents, carers & family members - we want to hear your views!!

    Please complete our survey by clicking HERE

     

    Why is your participation so important?

    As many of you are aware, two new possible treatment options for some people with CF (PWCF) are currently being assessed for both clinical and cost-effectiveness by the National Centre for Pharmacoeconomics (NCPE). 

    1. Orkambi, which seeks to correct the basic underlying genetic defect in 57% of people with CF in Ireland, people with two copies of F508del mutation. 
    2. The expanded use of Kalydeco for mutations:
          • G551D & other non-G551D gating mutations (expanded use to PWCF aged 2-5 years)
          • R117H (for PWCF over 18 years)

    As with all new therapies, they must undergo Health Technology Assessments (HTA) which is

    "A form of research that generates information about the clinical and cost-effectiveness of health technologies. These technologies can include drugs, medical devices, diagnostic techniques, surgical procedures and public health programmes such as cancer screening programmes. A Health Technology Assessment (HTA) may also look at the social, ethical, medicolegal and organisational aspects associated with use of a technology including its resource implications and budget impact.

    The information provided by the HTA is used to inform health policy decisions regarding the investment in (or disinvestment from) these health technologies."

    Cystic Fibrosis Ireland (CFI) are putting together a submission which will be made to the National Centre for Pharmacoeconomics (NCPE). This primary aim of this submission is to ensure the patient & family perspectives are formally taken into consideration during the Health Technology Assessment of the new therapies: Orkambi & Kalydeco (for expanded use).

    The purpose of this survey is to gather information from both people with CF and their families about experiences & views of treatment options for cystic fibrosis & ultimately, 'what matters to you'.

    It is so important that the voice of the CF community is heard loud and clear throughout, so please fill in the survey to have your say today.

    This process and the arrival of similar innovative therapies in the future will challenge the HSE & Irish Government to look at what 'value for money' really means in the context of CF care. The whole community must speak up and make sure these decisions are guided by the unique knowledge and experience of people living with CF.

    This survey has been shared by the Cystic Fibrosis Trust, UK, who have kindly allowed CFI to use & edit to questionnaire to meet the needs of the Irish CF population.

    The survey will take approximately 15-20 minutes to complete & we ask you to provide as much detail as possible.

    The results of this survey will be used to represent the views of the CF community & will primarily be used for advocacy purposes, and to inform policy decisions.

    Your responses will be completely confidential and any published results will be entirely anonymous.

    If you have any questions about the research, please contact Katie, Research & Development Officer, CFI at This email address is being protected from spambots. You need JavaScript enabled to view it. or locall 1890 311 211

     

  • Statement from Cystic Fibrosis Ireland - Orkambi (and Kalydeco)

    Cystic Fibrosis Ireland (CFI) is concerned that, according to a recent statement by Vertex Pharmaceuticals, there has been no contact between the Health Service Executive and Vertex in recent weeks, despite repeated public assurances by the Minister for Health, Simon Harris TD, and An Taoiseach, Enda Kenny TD, that a deal on important new drug therapies is imminent.

    On behalf of our patients, CFI calls on Minister Harris and the Government to do what they have promised to do – to make an agreement for the provision of Orkambi and Kalydeco for the 600 patients that would benefit from these two vital drug therapies.

    The negotiations for these ground-breaking drugs are now more than nine months old. There have been repeated assurances given in recent weeks which CFI has welcomed. Cystic Fibrosis patients would be very grateful for the promised announcement on a final agreement.

  • Statement from Minister for Health 

    Cystic Fibrosis Ireland welcomes the statement made by Minister for Health, Simon Harris TD today in relation to the extension of Orkambi to children aged 6 - 11 years. We thank the Minister Harris and the HSE for their on going commitment to CF care in Ireland. 

    Below is the statement from the Minister for Health, which can also be viewed online by clicking here.

    Good news for Cystic Fibrosis patients and their families with the extension of ORKAMBI availability to children aged 6 – 11 years

    Ireland becomes one of the first countries in the European Union to provide access to ORKAMBI for children in this age category

    The Minister for Health, Simon Harris, has welcomed the extension of the availability of ORKAMBI to eligible children with Cystic Fibrosis, aged 6-11 years.

    This comes following EMA approval in January of this year. The 2017 agreement between the manufacturer, Vertex Pharmaceuticals and the HSE included the extension to other age groups, subject to market authorisation in Europe.

    The Minister said “It’s very positive news that ORKAMBI is now licenced and available for reimbursement in Ireland for CF patients aged 6 to 11.

    “ORKAMBI was already licenced and available for reimbursement for children aged 12 years and over. The extension to the younger children became possible following EMA approval in January of this year. This quick turnaround between approval and availability was possible because we ensured the younger age group was included in the agreement with the company Vertex last year, subject to market authorisation in Europe.”

    “I am very pleased that Ireland is one of the first countries in the European Union to provide access to ORKAMBI for children in this age category.”

    ENDS

     

  • Sweden adapts Irish drug therapy deal to make Orkambi available

    Philip Watt, CEO of Cystic Fibrosis Ireland welcomes the decision to make the ground-breaking CF drug therapy Orkambi available to CF patients in Sweden. In doing so, the Swedish authorities and Vertex (pharmaceuticals) have adapted the pioneering long term access programme that was approved in Ireland in April 2017. This long term access programme not only provides patients access to Orkambi but also CF drugs not yet approved but which are in at advanced clinical trial stage and which may be even better than Orkambi.

    The CF drug Orkambi is now available in Austria, Germany, Ireland, Italy, the Netherlands, Sweden and the U.S.

    Read the full statement from Vertex below:


    Vertex Announces Long-Term Access Agreement in Sweden for Cystic Fibrosis Medicine ORKAMBI® (lumacaftor/ivacaftor) 

    June 18, 2018

    - The agreement allows for reimbursement of ORKAMBI for people who have two copies of the F508del mutation from July 1 -

    - A framework for assessment and access to our future cystic fibrosis medicines is included as part of the agreement -

    LONDON--(BUSINESS WIRE)--Jun. 18, 2018--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of theF508del mutation, ages six and older, will be reimbursed in Sweden after concluding the three-party negotiations with TLV and the county councils. Reimbursement is effective from July 1. The innovative, long-term access agreement also provides a framework for the assessment and access of our future CF medicines.

    “We are delighted that people with CF in Sweden will join the thousands of others around the world who are already benefitting from our CF medicines,” said Simon Bedson, International General Manager at Vertex. “We commend the Swedish authorities for partnering with us on an innovative, long-term access agreement. In countries where Vertex remains actively involved in reimbursement discussions, we encourage these health authorities and governments to match the commitment to innovation shown in Sweden to secure access for all patients who may benefit.”

    CF is a devastating rare disease that causes continuous damage to multiple organs from birth. In the lungs, a build-up of sticky mucus causes progressive and permanent damage, severe infections and ultimately premature death. In addition to Sweden, countries where lumacaftor/ivacaftor is available to all eligible patients include Austria, Germany, Ireland, Italy, the Netherlands and the U.S.

    About Cystic Fibrosis 

    Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

    CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

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

    In people with two copies of theF508del 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 theF508del 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. Lumacaftor/ivacaftor 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 onwww.ema.europa.eu.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

    Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for eight years in a row. 

    For additional information and the latest updates from the company, please visitwww.vrtx.com.

    (VRTX-GEN)

    View source version on businesswire.com: https://www.businesswire.com/news/home/20180618005495/en/

    Source: Vertex Pharmaceuticals Incorporated

  • Symkevi likely to be available in Ireland in early 2019 

    As predicted in the Autumn issue of Spectrum, the European Medicines Agency have now fully approved the third CFTR drug from Vertex called 'Symkevi'. 

    Those that stand to benefit from this new drug are the same group of patients that benefit from Orkambi (double Delta F508). It is likely that a protocol for deciding which patients will remain on Orkambi and which may switch to Symkevi will be developed.  

    The PR Company for Vertex Pharmaceuticals have informed Cystic Fibrosis Ireland of additional updates, as below:

    • The European Commission has granted Marketing Authorization for SYMKEVI® (tezacaftor/ivacaftor), a new treatment option for patients with two copies of the F508del mutation, the most common mutation in cystic fibrosis.
    • SYMKEVI® is the first medicine in the EU to treat the CFTR protein defect in patients who have one copy of the F508del mutation and one copy of one of 14 mutations that result in residual CFTR activity
    • The agreement that Vertex agreed in 2017 with the HSE included facilitates an expedited review of our reimbursement application for certain treatments and extensions to age cohorts to specific CF mutations, subject to market authorisation by the European Commission.
    • Accordingly, Vertex will work closely with the HSE and relevant officials to secure timely access for these patients, and we hope it will be made available shortly

    As part of the pipe-line/portfolio deal agreed between the HSE and Vertex, CFI predicts that Symkevi will be available in Ireland in early 2019.

    We will keep you updated.


    Statement from Vertex

    Vertex Announces European Authorization for Third Cystic Fibrosis Medicine SYMKEVI® (tezacaftor/ivacaftor), to be used in combination with ivacaftor (KALYDECO®), for People with CF Aged 12 and Older with Certain Mutations in the CFTR gene

    - A new treatment option for patients with two copies of the F508del mutation, the most common mutation in cystic fibrosis -

    - First medicine in the EU to treat the CFTR protein defect in patients who have one copy of the F508del mutation and one copy of one of 14 mutations that result in residual CFTR activity -

    LONDON--(BUSINESS WIRE)--Nov. 1, 2018-- Vertex Pharmaceuticals (Europe) Limited, today announced that the European Commission has granted Marketing Authorization for SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO®) for the treatment of people with cystic fibrosis (CF) aged 12 and older who either have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. In addition, the European Medicines Agency’s Committee for Orphan Medicinal Products recently recommended the maintenance of orphan designation for tezacaftor/ivacaftor in combination with ivacaftor.

    “The authorization of tezacaftor/ivacaftor in combination with ivacaftor is welcome news for European CF patients, their families and everyone involved in their treatment and care. This new medicine is especially important for patients with residual function mutations and those who do not tolerate ORKAMBI® (lumacaftor/ivacaftor),” said Harry Heijerman, Professor and Head of Department of Pulmonology at University Medical Centre Utrecht, The Netherlands.

    The EU Marketing Authorization was based on results from two pivotal Phase 3 studies, EVOLVE and EXPAND, published in the New England Journal of Medicine in November 2017. Results showed treatment with tezacaftor/ivacaftor in combination with ivacaftor provides benefits across different CF populations, including statistically significant improvements in lung function, as determined by absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1); with a generally well tolerated safety profile and a lack of increased respiratory adverse events compared to placebo. The improvements in lung function showed a mean absolute change in ppFEV1 compared to placebo of 4.0 percentage points (P<0.0001) and 6.8 percentage points (P<0.0001) in EVOLVE and EXPAND respectively. The most common adverse reactions experienced by patients who received tezacaftor/ivacaftor in combination with ivacaftor in pooled, placebo-controlled Phase 3 studies were headache and nasopharyngitis.

    “Today marks an important milestone for many CF patients in Europe, including those who so far have had no available option to treat the CFTR protein defect responsible for their disease,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “With today’s Marketing Authorization, we are rapidly moving towards treating 90 percent of CF patients.”

    Tezacaftor/ivacaftor in combination with ivacaftor was approved by the U.S. Food and Drug Administration (FDA) in February 2018 and by Health Canada in June 2018. It is marketed as SYMDEKO™ in the U.S. and Canada.

    About CF
    Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

    CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

    About tezacaftor/ivacaftor and ivacaftor
    Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface where ivacaftor can increase the amount of time the protein stays open.

    For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu once posted.

    About EVOLVE and EXPAND
    Data from the two Phase 3 studies EVOLVE and EXPAND were published in the New England Journal of Medicine in November 2017, the studies enrolled approximately 750 people with CF ages 12 and older with two copies of the F508del mutation or with one F508del mutation and a second mutation associated with residual CFTR activity. Across both studies, patients treated with tezacaftor/ivacaftor in combination with ivacaftor experienced statistically significant improvements in lung function, as determined by absolute change from baseline in ppFEV1. The treatment was generally well tolerated; the most common adverse reactions (≥10%) experienced by patients who received tezacaftor/ivacaftor with ivacaftor in the pooled, placebo-controlled Phase 3 studies were headache (14% versus 12% on placebo) and nasopharyngitis (12% versus 10% on placebo).

    About orphan designation for medicines
    Orphan designation is granted by the European Medicines Agency’s Committee for Orphan Medicinal Products to treatments which either address an existing unmet need or can provide significant benefit for people with life-threatening or chronically debilitating diseases, affecting a small number of patients.

    About Vertex
    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

    Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for eight years in a row.

    Special Note Regarding Forward-looking Statements
    This press release contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995, as amended, including the quotes in the second and fourth paragraphs of this press release. While the company believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, risks related to commercializing SYMKEVI in Europe and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN)

    Source: Vertex Pharmaceuticals Incorporated

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

    PurpleRoses

    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."

    Ends

     

  • 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 extension for Children with CF aged 6-11 years

    Cystic Fibrosis Ireland understands that the HSE is aiming to make Orkambi available to children with CF from the beginning of February 2018. This is very welcome and we thank the HSE and the Minister for Health, Simon Harris TD, for their on going commitment to CF care in Ireland. 


    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


  • 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.



    REPLY.
    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.

  •  

    Very encouraging Late Stage Study Results for forthcoming CF Triple-Combination drugs

    On 27 November Vertex pharmaceuticals released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination drugs that are undergoing late stage clinical trials (Phase 3 trials). These are further part of the CFTR family of drugs that includes Klaydeco; Orkambi and most recently Symkevi.

    ‘The results are very encouraging’  said Philip Watt, CEO Cystic Fibrosis Ireland (CFI). 'They are still at clinical trial stage but they point to the increasingly strong possibility of further drugs coming down the line for those already on a CFTR drug and for some of those that are not. It continues to be a time of hope for people with CF. We are determined to try and ensure that no one gets left behind and that there will be a CFTR drug available to as many patients as possible in Ireland’

    In further, unconnected news - Novartis has sold TOBI Solution and Tobi Pod inhaler to US pharma firm, Mylan which also has a significant presence in Ireland (see information at end of this article).

    The results
    Positive results were announced from late-stage studies of a potential triple-combination CFTR drug for people with cystic fibrosis. Vertex Pharmaceuticals released Phase 3 clinical trial data for two studies of the next-generation modulator VX-659 in combination with ivacaftor and tezacaftor. 

    The first study tested this triple-combination drug on people with CF ages 12 years and older who have one copy of the most common gene alteration (mutation), F508del, and one minimal function mutation

    Results showed that those who received VX-659 combined with ivacaftor and tezacaftor had a 14 percent increase in lung function compared to participants taking a placebo. Researchers believe that anyone with at least one copy of the F508del mutation -- regardless of their second mutation -- could benefit from next-generation modulators.

    The second study included people with CF ages 12 years and older who have two copies of the F508del mutation. The trial compared the effectiveness of Symkevi (tezacaftor/ivacaftor) to VX-659 combined with tezacaftor and ivacaftor. Participants who were given VX-659 combined with tezacaftor and ivacaftor had a 10 percent improvement in lung function over those who were only given Symkevi® (tezacaftor/ivacaftor).

    Just as important are the wider impacts that these drugs will likely have including overall quality of life and reduction in exacerbations and weight gain. It’s too early to be definitive but the signs at this stage are very encouraging.

    Vertex will decide which of the two next-generation drugs they will submit to the U.S. Food and Drug Administration (FDA) for potential approval as a new drug. If approved, it is estimated that triple-combination CFTR modulators could potentially bring the benefits of therapies that treat the underlying cause of the disease to more than 90 percent of people with CF.

    Michael P. Boyle, M.D., senior vice president of therapeutics in the US Cystic Fibrosis Foundation stated:

    ‘It is an exciting time for the CF community, as we approach a milestone that seemed impossible even just a few years ago. Today's announcement represents an important step in our journey to developing treatments for the underlying cause of this disease for all people with cystic fibrosis. We are enthusiastic about the clinical benefit VX-659 demonstrated in these studies and look forward to seeing how these compare to the other next-generation modulator in clinical trials, VX-445.’

    CF Hospital Centres in Ireland have played a key role in supporting clinical trials for these medications. It is unclear yet when they will be submitted for approval to the FDA in the US and the EMA in Europe.

    There are presently 3 Vertex CFTR drugs approved for reimbursement in Ireland. These are ground-breaking drugs that treat the underlying cause of CF and include:

    Kalydeco (2013) primarily for those patients with the G551D gene alteration

    Orkambi (2017)for those patients with two copies of the F508del gene alteration

    Symkevi (2018) for those patients with two copies of the F508del gene alteration (alternate drug to Orkambi)

    In relation to Symkevi, CFI understands that a protocol is being developed. It is likely that those who are doing well on Orkambi (and the feedback to date is generally very positive) will remain on Orkambi.

    The impact of these drugs are closely monitored in Ireland. CFI will continue to provide impartial and informed information about these and future CF Drugs

    Further Drugs News: Novartis sells Tobramycin to Mylan
    In September 2018 Mylan, a Pennsylvania pharma company paid Novartis, the Swiss based pharm-company, 463 million dollars for its CF therapies including TOBI solution and TOBI pod-inhaler. This coincides with TOBI coming off patent. Nebulised Tobramycin was introduced in 1998 and has proven especially effective against Pseudomonos. Meanwhile Teva an international Pharma company has introduced a generic form of Nebulised Tobramycin.  These developments will likely result in significant price reductions in Nebulised Tobramycin to the HSE. 

  • VX-661 news

    Vertex Pharmaceuticals issued a statement on Monday 15th August that it is ending a clinical study testing a two-drug combination therapy on a small group of cystic fibrosis patients after an independent board concluded the experimental treatment wasn’t showing meaningful benefit.

    Executives had previously warned that the late-stage clinical study combining its drug candidate, called VX-661, with its approved drug Kalydeco might not prove effective for about 150 patients enrolled in the study. Each of those patients has two separate genetic mutations linked to CF.

    In a statement, Vertex chief medical officer Jeffrey Chodakewitz said that this CF population has “a form of the disease that is particularly difficult to treat.” But he said Vertex remains hopeful those patients will be able to benefit from a three-drug combination -- including VX-661 and Kalydeco -- that Vertex is also developing.

    Background Note: Vertex have two approved drugs, Kalydeco and Orkambi, which treat CF patients with certain mutations. Kalydeco has been approved and is reimbursed in Ireland. Orkambi is not yet available in Ireland, except on compassionate grounds as the price is currently being negotiated. These are the first drugs that treat the underlying cause of CF