Cystic Fibrosis Care in Ireland compared with other European Countries
Cystic Fibrosis care services and treatments in Ireland have progressed steadily over the past decade, but with still much more to do. Cystic Fibrosis is a life-long recessive genetic condition for which there is still no cure. Ireland has among the highest rates of CF in the world with among the most severe manifestations – in large part shaped by the genotype profile of the CF population in Ireland. CF primarily impacts on the respiratory and digestive systems, but also impacts on other organs of the body.
Rising care standards in CF care in Ireland have been shaped by access to new and innovative drug therapies; the work of the National Clinical Programme for CF; our expert doctors and their teams; the data and benchmarks provided by the Cystic Fibrosis Registry and of course the sustained advocacy by Cystic Fibrosis Ireland over 60 years.
Improved hospital infrastructure in Ireland, additional clinical staff and high quality research, especially over the past decade, has made a major contribution to the progress in CF services. However with this progress, CF care has also become significantly more complex. As predicted survival increases year on year, co-morbidities associated with growing older and CF related liver and kidney disease have emerged as more significant issues for an increasing number of our patient population.
Predicted survival rates of people with CF have been significantly boosted by the emergence of the new and innovative drug therapies that treat the underlying cause of CF, such as Orkambi and most recently Kaftrio. In paediatric care in particular there is a growing sense that while CF remains a life threatening disease, it can now be managed much more effectively. ‘Fire-fighting’ is now being replaced in large part with ‘fire prevention’ for many children and younger people. It is also important to emphasise that CF impacts on individuals differently and not all have being able to access or tolerate Kaftrio for example, especially those with rarer gene mutations.
However these wonderful new drugs cannot undo the damage to organs that has already occurred. For example, almost 100 people with CF in Ireland have a double lung transplant. Some now need a kidney or liver transplant. This cohort of patients were also most at risk from the recent COVID-19 pandemic. Further investment in post-transplant CF care in the Mater (MMUH) and other hospitals has commenced and this is to be welcomed but more needs to be done. Transplant services in general in Ireland are still recovering from the COVID-19 pandemic.
Rather than fighting for basic services, we are now in a position in Ireland to aspire to the higher level of care long enjoyed by some of our European Union neighbours, such as Belgium, Netherlands, France and Germany. Much of the work of Cystic Fibrosis Ireland over the past decade has been to fight for the full implementation of agreed government policy to enable us to meet accepted European standards. In some aspects we are now among the leading CF care providers in Europe but still with much more to do.
CF adult care in Ireland was given a significant boost in recent weeks as a result of green lights being given to the commencement of building of the long promised 20 bed inpatient unit in Beaumont Hospital and the new adult CF outpatient centre in Merlin Park Galway. CFI contributed ¤700,000 to the Galway Unit which will cost a total of ¤3.4m. CFI has also raised more than ¤1m for the Beaumont CF Unit, which will cost around ¤22m. We thank the hospitals and the HSE for providing the main capital funding for both of these important initiatives though delays have made these projects significantly more expensive.
Over the past decade Ireland has made strides to catch up with our European neighbours in other areas of CF care. For example in 2011, after 20 years of lobbying, CF was finally added to the list of conditions identified through the Heel Prick Test. This means that treatment for CF mow commences from birth rather than waiting months of inaction because of the absence of an accurate diagnosis.
Other patient groups, such as Sickle Cell and Thalassemia Ireland have been advocating for many years for similar inclusion in new born screening.
The new CF drug therapies are still not a cure and up to one quarter of people with CF in Ireland have presently no access to CFTR Modulator drugs such as Kaftrio. In this context it is welcome to see that a dossier has now been forwarded to the European Medicines Agency by Vertex which seeks to broaden the license for Kaftrio in Europe, including Ireland. In June 2023 I was elected as President of Cystic Fibrosis Europe (CFE), the umbrella group for 39 patient groups across greater Europe. From my increased interaction with other EU countries it is clear that CF care in Ireland is now aspiring to be in the top tier of CF care.
Some countries in eastern and southern Europe only have rudimentary CF services. Access to drug therapies such as Kaftrio remains an aspiration for such countries, However there are some recent shafts of light. For example, the decision by pharma company Vertex to provide Kaftrio free of charge to patients in Ukraine is to be warmly welcomed.
CFE continues to urge the global expansion of these ground breaking drugs through marketing strategies that takes into account regional levels of poverty and GDP. Individual governments must also be held to account for poorer CF care facilities.
Around 2009, the national lung transplant centre in the Mater Hospital was still largely in its infancy and depended on support from the Freeman Hospital in Newcastle for the few CF lung transplants then undertaken in Ireland. The lung transplant programme is now well established in Ireland and there is very little reliance on UK hospitals. However resourcing remains an issue.
Ireland continues to lag behind other Council of Europe Countries in respect of all organ donation and transplants. This deficit is likely through a combination of under-investment in some aspects of donation and transplant services, but is also related to our present and outdated opt-in form of organ donor consent, which is thankfully about to change.
Ireland should aspire to be in the top 10 organ donor and transplant countries in Europe, rather than our present position (28th place).
There are some 600 people (all diseases) waiting for a lifesaving transplant in Ireland (heart, lung, kidney, liver, pancreas). In this context, it is very welcome that the soft opt-out organ donor consent enabling provisions in the Human Tissue Bill, passed through the Report Stage in the Dáil on the 8th Of November 2023. Barring any last minute glitches the Bill should be enacted early in the new year. Soft opt out organ donor consent is the default in the rest of Europe including Spain which consistently has the highest levels of transplanting organs per head of population. It should also be cautioned recent experience in Wales shows that its takes 2-3 years for this improved form of organ donor consent to be fully functional.
In conclusion, much done, more to do. CFI marks our 60th year. We were formed on a dark November day in November 1963. Our 2 ‘guiding lights’, with the support of many, are AnneO’Dwyer, our founder and Bridie Maguire our first CEO. Bridie, a former Person of the Year, sadly passed away in October 2023. She was an amazing woman who lost 2 children to CF before they reached the age of 2 years of age. The fact that parents like Bridie and Anne continued to fight for better CF services and to support those in desperate need, even after the death of their own children, is a testament to the members and spirit of Cystic Fibrosis Ireland. It has been the priviledge of my life to had the opportunity to serve the CF community in Ireland and to have met people like Anne and Bridie. The story of CFI is available on our website in the Book ‘For the Roses’ see news item on Bridie Maguire. An 11 minute documentary to celebrate the 60th year of CFI is available on you tube ‘Someday, CF will stand for Cure Found’.
Written by Philip Watt CEO CF Ireland and President of CF Europe
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