Pipeline drugs

Pipeline drugs

  • Drug Iceberg 2.0 Published

    In February 2018 the Irish platform for patient organisations, science and industry (IPPOSI) and the Medical Research Charities Group (MRCG) announced the publication of the second “Drug Iceberg” Report. This report lay out key recommendations including a call for the development of a new national strategy on access to medicines. 




  • The Pipeline Aspect of the Recent Vertex and HSE Agreement

    A couple of CFI members have asked us to help clarify the ‘pipeline aspect’ of the recent HSE/Vertex agreement. This is an innovative aspect of the deal and we understand it has never being implemented before in Ireland and may even be unique in Europe. CFI pushed for and supported the pipeline dimension during the negotiations and we acknowledge the positive role of Vertex, the HSE and Minister Harris in this development. 

    It is important to recognise that while this is a very positive and innovative development, it will not (of course) be a panacea for resolving all future CF drug therapy reimbursements. 

    CFI understands the HSE/Vertex agreement currently covers:

    • Orkambi for all CF patients 12 years and older who have two copies of the F508del alteration 
    • Expands access to Kalydeco for children aged 2 to 5 with any approved gating alteration (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D) 
    • Access to Kalydeco for people aged 18 and older who have an R117H mutation
    • Rapid access for people with all the above alterations (ie: two copies of the F508del mutation, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D, R117H) if the labels of the existing medicines are expanded to cover additional age groups and if new Vertex medicines are approved for these populations

    If, for example, a drug therapy is developed (either by Vertex, or indeed another pharma company) for the gene alterations not listed above, there would have to be further discussions between the company and the HSE as and when required.

    The bottom line is that we estimate that as a result of the Orkambi and the previous Kalydeco agreement of 2013 that around 65% of the CF population in Ireland (and probably more) will have now access to a bespoke CFTR drug therapy, with more pipeline drugs for those groups to follow. This is important as the impact of Orkambi can vary from one individual to another. 

    Needless to say, CFI will continue to adopt a robust approach to potential reimbursement of future drug therapies including in particular for those who do not have a bespoke drug at present, provided the CFI Board is convinced on the efficacy, safety and impact of such drugs. In short, the 35% of CF patients without a CFTR drug in Ireland will remain an absolute priority for CFI. It is worth noting the indirect impact of the reimbursement of Orkambi will be to encourage further research into ground breaking CF drugs. Further, the CFF in the US continues to invest millions of dollars into further drug therapies, including those without a bespoke therapy at present. In short this agreement is a major step forward but there remains so much more to do. 

    In consultation with expert clinical advice, CFI will also be also publishing important advice to our members about Orkambi and the need to continue other treatments and the need to keep up a good diet and exercise regime.