Stuart Elborn

Stuart Elborn

  • Orkambi Update - 30th November 2016

    The following are points made by Cystic Fibrosis Ireland in response to the public debate on Orkambi over the past few days.


    1. The Urgent need For Ministerial Intervention and Positive Response by Vertex

    2. The Efficacy of Orkambi

    Cystic Fibrosis Ireland urges the HSE to provide a more comprehensive and fairer public statement on the efficacy of Orkambi, particularly in relation to recent research that has shown that CF progression is slowed down by Orkambi and the impact of the 40% reduction in exacerbations.

    Orkambi (also known as ‘lumacaftor/ivacaftor’) is for people aged over 12 with two copies of F508-del gene alteration, which about 550 people in Ireland (compared with around 3,200 people across the whole of the UK).

    Declining lung function and exacerbations of Cystic Fibrosis is ultimately the main cause of death among people with cystic fibrosis. By reducing this decline, precision medicines like Orkambi can help people with cystic fibrosis stay healthier for longer. The gap in lung function widens for every year that someone eligible is not on Orkambi. This is particularly poignant where one sibling is on Orkambi and one is not.

    Orkambi reduces exacerbations by up to 40%- that is worsening of a condition that results in hospitalisations. Every time a person with CF experiences an exacerbation research has shown it can reduce their survival expectancy from 3-6 months.

    Cystic Fibrosis Ireland urges the Department of Health/HSE to take into account a recent study (28th October 2016) carried out over 96 weeks.

    Analysis of the 96-week PROGRESS extension study of the pivotal phase 3 TRAFFIC and TRANSPORT studies of Orkambi confirmed the safety profile, and mean lung function was maintained above baseline for up to 120 weeks and the 40% reduction in exacerbations.

    Professor Stuart Elborn formerly of Belfast City Hospital and now lead Cf adult consultant in the Brompton hospital commented:
    "Long-term follow-up data has indicated that this treatment can prevent disease progression.

    "Initially, we were able to show that you can make people a bit better. Now we're seeing exciting and reassuring long-term improvement."

    "We hope this will lead to a further rethink about the long-term benefits," said Professor Elborn. "There are likely to be reductions in the cost of hospital visits and other treatments."

    "I'm really excited by the therapy and also the pipeline of other powerful drugs that could get us closer to a cure."

    3. Meeting the Cost of Orkambi from Savings in the Drugs Budget

    CFI has acted responsibly at all times. We did not engage in advocacy over the 25 weeks that the HSE negotiated with Vertex, the pharmaceutical company. This was a deliberate strategy to support the HSE to obtain the best price possible for this important drug. We have called on Vertex to make significant reductions in price and to enter into shared risk agreements with the HSE, if these are the sticking points on which the negotiations have foundered.

    We would not expect the HSE to pay for a drug that does not work on some patients. We would question recent assertions that Orkambi only works on about one quarter of those taking part in clinical trials and we have sought expert opinion on this.

    IPHA agreement and related savings

    CFI notes from the Minister’s statement to the Joint Oireachtas Committee meeting in September 2016 re the IPHA agreement
    ‘The States team objective was to improve the assessment and reimbursement process for new drugs and to secure significant price reduction so as to enable continued access to new and existing drugs for Irish patients while reducing growth in the HSE’s overall drugs bill…..The new Agreement, which runs to the middle of 2020, is projected to result in savings – that is, expenditure foregone – of some €600 million from IPHA companies, with a further €150 million in savings anticipated from non-IPHA companies over the lifetime of the deal’.

    The Fight for Orkambi will continue

    CFI will continue to lobby the HSE/Department of Health and Vertex to reach a fair price on Orkambi.

    Philip Watt

    CEO, Cystic Fibrosis Ireland