Joe McHugh (Donegal North East, Fine Gael)
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I am raising this issue on behalf of some parents who have boys with Duchenne muscular dystrophy, a rare disease that affects approximately 100 boys in Ireland. Before I discuss the specific circumstances of the parents on whose behalf I am speaking, I wish to put on record the good work and good service delivery at the Central Remedial Clinic, CRC, in respect of various sectors. Some parents send their boys with Duchenne muscular dystrophy to the clinic. It is important to record that I am not speaking on their behalf but on behalf of parents of boys who do not attend there. Approximately a dozen parents bring their boys to Newcastle, the reason being that bisphosphonates are available there on prescription. In conjunction with steroids, this treatment is producing very positive results for the boys. According to one of the parents, the survival rate is staggering. Another parent has said to me that all parents are trying to do is keep their children on their feet for as long as possible.

With Duchenne muscular dystrophy, best practice changes nearly every six months. It is a new area. There is a clinical trial site in Newcastle but none here. This presents a difficulty for the dozen or so families who are sending their boys to the United Kingdom.

There is a new administration temporarily in charge of the CRC. We are all aware that the board has stepped down and that the chairman is no longer in situ. There is a new administrator. All I am asking on behalf of the dozen or so affected parents is that the new administrator meet the parents and listen to them. They have experience of very good service delivery in Newcastle. They want to bring something to the table that might be of value and help in the delivery of services here. This is not about saying one service is better than the other; it is required of the Government to be big enough to say that if there are new ideas being applied in Newcastle, it is willing to learn from them. All I ask is that the dozen or so parents be listened to because they could bring value to the debate and be of benefit to the other parents in Ireland whose affected boys are just on steroids and who do not have the opportunity to take bisphosphonate.

For many years, I have been advocating the location of a clinical trial site in this country.

I am aware of the current economic constraints, but we should still focus on the possibility of developing a clinical trial site in this country.

Kathleen Lynch (Cork North Central, Labour)
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I thank the Deputy for raising this issue and his positive comments on the incredible service provided by the CRC. The clinic has been embroiled recently in the controversy over pay and top-up payments, which is unfortunate because 99% of those attending the CRC are extremely happy with the service being provided.

The Department of Health has been unable to obtain the material to answer the specific questions raised by the Deputy in the time given. However, it undertakes to follow up with the Health Service Executive and the CRC and write to the Deputy with the information as a matter of urgency.

The issue of standards of care is at the top of the Minister's and the HSE's agenda. The HSE has established a quality and clinical care directorate to help to improve patient care throughout the health system. Clinical governance will ensure patients can get the right treatment at the right time. Clinical audit programmes will measure and audit standards to ensure they are delivered across the health system, including through agencies such as the CRC.

I will deal with the Topical Issue raised by referring to work under way on rare diseases and the services provided at the CRC. Duchenne muscular dystrophy is a rare, chronic, debilitating and life threatening condition. Most rare diseases have a genetic origin and the life expectancy of persons with rare diseases such as Duchenne muscular dystrophy is significantly reduced. Scientific knowledge of rare diseases, including Duchenne muscular dystrophy, is limited.

Ireland is well advanced in developing a strategy for rare diseases. A national steering group is developing a policy framework for the prevention, detection and treatment of rare diseases. The principles are high quality care and equity and centred on the patient. An integral part of an effective policy framework will be the development of a dedicated HSE clinical programme for rare diseases. This programme will aim to improve and standardise patient care by bringing together clinical disciplines and enabling them to share innovative solutions to deliver greater benefits to HSE service users. It is envisaged that a national rare diseases clinical programme would, over time, assist in developing national treatment guidelines, standard operating procedures and care pathways for many rare disorders, including Duchenne muscular dystrophy, in collaboration with designated reference centres. It would also develop care pathways with European reference centres for those ultra rare disorders on which there may not be sufficient expertise in Ireland.

The CRC provides a comprehensive range of services for almost 4,000 children and 500 adults throughout the country with physical conditions ranging from the very rare to the more familiar such as cerebral palsy, spina bifida and muscular dystrophy, including Duchenne. The CRC services assess, diagnose and treat children and adults with a wide range of physical conditions. In addition, the clinic offers a range of training, development and ongoing support options to adults with a physical disability. The CRC received funding of approximately €16 million from the HSE in 2013. It is also worth noting that it has been designated as a centre of excellence by the national accreditation committee for its high standard of delivery of training in adult services. It also recently received a HSE innovation award for its mobile gait laboratory services.

Dr. Brian Lynch, a consultant at the CRC, has provided additional information for the HSE. He has pointed out that children with Duchenne muscular dystrophy attend the CRC which has run a neuro-muscular clinic for over 25 years. At the CRC, children attend multidisciplinary clinics where they receive input from paediatricians, neurologists, orthopaedic specialists, physiotherapists, occupational therapist and dieticians. They also engage with clinical psychologists and social workers, where necessary. The treatment provided for these children at the CRC is in line with best international standards of care.

I will pass on the Deputy's specific request regarding the new director of the CRC, whoever that may be, meeting the families. Ireland is too small to develop the required research capacity in the area of rare diseases. We will have to continue to co-operate with others across Europe.

Joe McHugh (Donegal North East, Fine Gael)
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I thank the Minister of State for her reply and commitment to try to facilitate a meeting between the new director of the CRC and the dozen or more families concerned. Several aspects of her response are encouraging, particularly the reference to the development of care pathways, in co-operation with European reference centres, for those ultra rare disorders for which there is insufficient expertise in Ireland. The EU patient mobility directive is due to be transposed into Irish law next year and it will benefit the parents of children with Duchenne muscular dystrophy and many others. If the Acting Chairman needs a hip replacement, under the new directive, he will be able to travel to Italy, Germany or France to obtain one.

Bernard Durkan (Kildare North, Fine Gael)
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That is most reassuring.

Joe McHugh (Donegal North East, Fine Gael)
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It will change the way services are delivered in Europe. The parents of the children to whom I refer will continue to travel to Newcastle, regardless of when the directive is transposed. They were a little sore when the E112 forms were withdrawn, but I hope changes will happen early next year in that regard. Sometimes policy makers and politicians sit around the table and make decisions in the absence of the most important people - service users. The parents to whom I referred who travelled to Newcastle had an overwhelmingly positive experience, although that is not to say some of the parents of the children attending the CRC have not had a positive experience, too. The service in Newcastle has given these parents hope.

I do not want to put a figure on life expectancy for those suffering from this debilitating disease because parents have been given hope their children can live longer than expected. The children will be able to stand for longer and will not have to use wheelchairs when they are eight or nine years old. They are living longer and able to play with their peers in the school yard. Their parents want the best for them and we can learn so much from both the parents and children. They have had a great experience and have been given hope. Sometimes we ignore the important things in life like listening to people. The parents of these children have something important to bring to the table. We must sit down and listen to them because we will learn more from them than from the experts with PhDs and other letters after their names.

Kathleen Lynch (Cork North Central, Labour)
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The Deputy will find no disagreement from me in that regard.