• 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.