Catherine Noone (Fine Gael)
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I thank the Minister of State, Deputy Catherine Byrne, for coming to the House on behalf of the Minister, Deputy Harris. I will be very happy to take her response and it is perfectly adequate to receive a response from the Department via the Minister of State.

The matter concerns the PKU issue and the drug Kuvan, on which there was a presentation in the AV room this morning. PKU is a rare blood disease that affects 13 or 14 babies per year in Ireland. It is known to be more prevalent in Ireland than in other countries - it is a Celtic gene, or so it was described this morning in the briefing I attended. The issue is that there is no known cure for this disease. It is diagnosed by a heel prick test and the Irish Government was revolutionary in introducing the heel prick test in the 1960s. As a young lad who suffers from PKU said this morning, we were really ahead of the game back at that time in dealing with PKU but, sadly, since then those affected by PKU have simply been advised to follow a strict no-protein diet. While that gives patients a certain amount of help and relief, if the levels of protein in their blood become too high, it affects them mentally and causes brain damage. We were shown videos this morning of young people who were behaving in a very distressed manner due to brain damage from high levels of protein in their blood.

The quality of life of patients and their families is a huge issue. PKU requires a highly regimented diet and lifestyle. The mother of the child who was at the presentation this morning described how they have to use a powder they buy in the pharmacy to make bread or an omelette. They colour it so the child knows it looks like bread, although the child has never tasted bread, but everything still tastes more or less the same. It is quite difficult for families to live with. The mother described how a sibling was very unhappy eating around the younger child because of the nice smells and so on. It is a difficult dynamic for a family to deal with.

Many Western countries have adopted the drug Kuvan, which has been described as a wonder drug. My question to the Minister of State is why Ireland has not decided that we should keep up with our intentions in the 1960s in being one of the forerunners on this. Kuvan received EU regulatory approval in 2009 and is currently approved for use and reimbursed in Austria, Belgium, Bulgaria, the Czech Republic, Estonia, France, Germany, Hungary, Italy, the Netherlands, Luxembourg, Norway, Portugal, Romania, Russia, the UK, areas of Sweden, Lithuania and Denmark. The Irish PKU community cannot see any justification for the drug not being available here.

While I know everything comes down to budgets, the harsh reality is that even when the gruelling PKU diet is maintained, people managing the condition still suffer with long-term permanent health conditions such as neurocognitive defects, neurological side effects, growth defects, bone mass reduction and obesity. This is very serious. I hope the Minister can find the budget to help the individuals with this disease. The eldest of the people who were diagnosed with this disease in Ireland are only in their 60s, so it is relatively new in terms of science and the medical area. I am very interested to hear the Minister's response.

Catherine Byrne (Dublin South Central, Fine Gael)
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I thank the Senator for raising this very important matter. I am sorry the Minister cannot be here but he sends his apologies.

Medicines play a vital role in improving the overall health of Irish patients. Securing access to new and innovative medicines in a timely manner is a key objective of the Irish health service. The challenge is to deliver on this objective in an affordable and sustainable way. Under the community pharmacy schemes alone, over 70 million prescription items will be dispensed in 2017 at an estimated cost of approximately €1.7 billion, taking account of fees and ingredients costs. In addition, the HSE will spend in excess of €500 million in 2017 on medicines through a range of other schemes, in hospitals and in other care settings, for example, in nursing homes.

Spending on medicines represents one of the largest areas of expenditure across the health service and it will continue to grow in the years ahead as our health service continues to meet the needs of our citizens. This is most welcome and can have a transformative impact on the health of patients in the future. However, as the Minister, Deputy Harris, has stated previously, the cost that some companies seek to charge for their medicines may result in a situation where certain treatments will never become available to patients. This is the case not only in Ireland, but across Europe. This is why it is essential that Ireland has a scientific, robust and evidence-based assessment process in place. This ensures that decisions in regard to the reimbursement of medicines are made on an objective and scientific basis, recognising the health needs of the population and other factors.

The Health (Pricing and Supply of Medical Goods) Act 2013 provides the legal framework in Ireland for such a process. This Act gives full statutory powers to the HSE to assess and make decisions on the reimbursement of medicines, taking account of expert opinion, as appropriate. The HSE follows the process set out in the Act for the assessment of all drugs. In reaching its decision, the HSE examines all the evidence which may be relevant, in its view, for the decision and will also take into account such expert opinions and recommendations it may have sought, including, for example, advice from the National Centre for Pharmacoeconomics, NCPE.

In December 2015, the company Merck Serono advised the HSE that the market authorisation for Kuvan was transferring to BioMarin in 2016. The HSE met with the new market authorisation holder in May 2016 and it advised the HSE that it would be submitting a health technology assessment dossier for Kuvan. The NCPE assessment of the new dossier was completed in September 2017 and the NCPE did not recommend Kuvan for reimbursement as it was not deemed cost-effective. The HSE assessment process is ongoing and the HSE will take into account the expert advice of the NCPE when making its decision, in line with the Health (Pricing and Supply of Medical Goods) Act 2013. The robust nature of this evidence-based assessment process helps to ensure that the health service makes the most efficient use of its finite resources.

Catherine Noone (Fine Gael)
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It is a disappointing answer which basically states: "We are not funding this." I cannot understand why, so I need to look into this further. I do not think we can leave it here, from what I heard earlier this morning. It is a disease that deserves attention. The individuals involved range from children to those aged up to 60. I need to get more information regarding the funding and how much the drug costs per patient, given the briefing did not go into that. I do not feel it is unreasonable to look for this and I am not going to leave the matter here.

Catherine Byrne (Dublin South Central, Fine Gael)
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I note the concerns of the Senator and will bring back to the Minister the points she has made. At present, all I can give her is the information I have been given, although I wish I could give her more. I understand the difficulties people have, particularly when children at a very young age are experiencing difficulties within families, and in terms of how this affects them when they cannot participate, even by having a meal with their family.I will bring the Senator's concerns to the Minister, Deputy Harris, and ask him to respond to the Senator on the cost if it is made available.

Catherine Noone (Fine Gael)
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I thank the Minister of State.