Pat Deering (Carlow-Kilkenny, Fine Gael)
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PKU is a rare genetic disorder that affects a person's metabolism. People with PKU are unable to break down an amino acid called phenylalanine, phe, a natural substance found in food. This results in a build up of phe in the blood and in the brain, which can cause serious health problems, including, if untreated, severe brain damage. There is much more to it than the dietary effects. It also involves irritability, lack of concentration, stomach pains and just not being like every other child.

The only medical treatment for PKU is the drug Kuvan, which can vastly improve the quality of life of some people with PKU. The drug enables more "normal" food to be eaten and critically improves cognitive functioning thus reducing the real threat of long-term irreversible brain damage. 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 and the UK and certain areas in Sweden, Lithuania and Denmark so why is this not the case in Ireland?

The PKU community cannot see any justification for the drug not being available in Ireland on such a relatively small scale when it is widely available across EU, the rest of Europe and worldwide. Since 2010, Kuvan has been assessed by the National Centre for Pharmacoeconomics, NCPE, twice and rejected on both occasions in 2009 and 2017. The PKU community is frustrated with this process and feels it is unfair as it is designed to fail orphan drugs, which do not have the necessary data. It is an expensive and lengthy process for small organisations such as the PKU Association of Ireland, PKUAI, while also being costly for the HSE. The drug review with the NCPE is set up to fail orphan drugs. These drugs are put through endless assessments which they can never win due to their poor scoring on quality of adjusted life, QALY. To get a reliable QALY, one needs big clinical trials, which are common for things such as oncology or heart disease. In simple terms, for example, a cancer drug could give an additional year of life for €10,000 so the cost of that QALY is €10,000. Sadly, PKU and other rare diseases cannot show examples for orphan drugs because one simply cannot do the clinical trials to produce good QALY. There is a misconception that these orphan drugs are expensive. While this sometimes is the case, the reality is often the fact that the QALY is unreliable. However, this will always be the case in orphan drugs because one cannot get reliable clinical trials with small amounts of patients and data.

The PKUAI strongly urges the HSE to engage with the manufacturers of Kuvan and approve funding for the only drug treatment available for PKU. It is already available in nine other EU countries. Giving access to this life-changing drug for all those eligible could transform the future health of people with PKU throughout Ireland and would be of huge benefit to everybody in the community

Gino Kenny (Dublin Mid West, People Before Profit Alliance)
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Like all Deputies in this House, we received an email from an impassioned mother whose six year old daughter Maria lives with PKU. I had not heard of PKU until I read the email. Ireland has the highest rate of PKU in the world. As Deputy Deering said, the drug Kuvan has been approved by over 20 countries in Europe. This drug has been proven to be medically beneficial for those living with the condition. Sadly, Kuvan has been rejected twice by the NCPE in the past eight years. This has had a devastating effect on the PKU community. My question is fundamental to many things that happen in this House, to orphan drugs and to people who have rare conditions, not because of their choosing but because of circumstances. The NCPE has said twice that the drug is not to be approved. Obviously, the reason it has not been approved is because the NCPE thinks it is not cost-effective. Will the Minister override its decision and tell the manufacturer of this company to sit down and agree a price? It has been done previously with the cystic fibrosis community regarding the drug Orkambi, so I am asking the Minister to override the NCPE's decision because this drug can greatly benefit those suffering from PKU.

Jim Daly (Cork South West, Fine Gael)
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I wish to thank Deputies Dino Kenny and Deering for raising this issue. 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. However, the challenge is delivering 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 ingredient cost. 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.

Expenditure on medicines represents one of the largest areas of expenditure across the health service and 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. However, as the Minister has stated previously, the cost that some companies are seeking to charge for their medicines may result in a situation where certain treatments may never become available to patients. This is the case not only in Ireland but across Europe. That is why it is essential that Ireland has a scientific, robust and evidence-based assessment process in place. This ensures that decisions relating to the reimbursement of medicine 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 take into account such expert opinions and recommendations which may have been sought by the HSE, including, for example, advice from the NCPE.

Sapropterin, or Kuvan, was previously considered under the national pricing and reimbursement processes in 2009. At that time, insufficient evidence was available to support the pricing and reimbursement application submitted by Merck Serono for Kuvan. In December 2015, 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 Biomarin in May 2016 and was advised that it would be submitting a health technology assessment dossier relating to Kuvan. The NCPE assessment of the Biomarin dossier was completed on 15 September 2017. The NCPE did not recommend Kuvan for reimbursement as it was not deemed to be 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.

Pat Deering (Carlow-Kilkenny, Fine Gael)
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I thank the Minister of State for his reply. Like Deputy Gino Kenny, a constituent of mine has a child with this disease. Obviously, it is a hugely debilitating disease and the family is very concerned in that regard. Fortunately, it was picked up by the heel prick test, which is carried out after the child is born. I disagree with one point made by Deputy Gino Kenny. I am not looking for the Minister to overrule the decision. I am asking him to change the assessment process because this drug will never succeed in being passed under the assessment process at present. As I mentioned in my initial contribution, the assessment process at present only suits tests relating to oncology or other more prominent diseases. This is a rare disease and the structure and the criteria that are there will never allow this drug to be used, which is the crucial point. The criteria for testing drugs dealing with these very rare diseases need to be changed. The process of assessing the drugs needs to be changed in order for this drug to be approved. I suggest that the Minister goes back and looks at the criteria.

Gino Kenny (Dublin Mid West, People Before Profit Alliance)
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This statement does not really mean anything. It is hollow, to say the least. What this comes down to is putting a price on somebody's life. Somebody has made a decision - I do not know if it is the Minister of State - that the price of somebody's life is not worth the price of medication for that. This goes further than providing Kuvan for people with PKU; it involves other orphan drugs like Translarna for people with muscular dystrophy, as was mentioned by Deputy Adams yesterday.

Will the Minister of State override its decision and review the assessment done by the NCPE on orphan drugs? The assessment is fatally flawed. People who have conditions not of their choosing but because of circumstances are being medically discriminated against because of price. I ask the Minister of State to review and override the NCPE's decision.

Jim Daly (Cork South West, Fine Gael)
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I am acutely conscious that there are real lives behind these speeches. Sometimes these speeches can come across as very harsh departmental responses but I am acutely aware that real people and children are involved, including mums, dads, brothers and sisters. We are talking about real life and I am not going to forget that for a second. I will have the assessment process looked at and will ask my officials to review it. There is a difficulty in that very clear guidelines are set out on how to assess the process and that is done in law. The process for it is under Schedule 3 of the 2013 Act. The NCPE has made a recommendation but the HSE has not yet made a decision and I want to clarify that. Some recent media reports have presented it in such a way as to indicate that the decision has been made. The decision has not been made, just the NCPE recommendation. It has looked at it under a variety of criteria to assess it, as it is duty-bound to, under the Act, and it has not recommended it but the HSE has yet to make a decision on it and that assessment process is still ongoing. I appreciate the opportunity that both Deputies have given to give air time to the matter and to add a human side to ongoing deliberations.

We spend €1.7 billion on prescribed drugs in this country and that is a challenge. We cannot have an open chequebook scenario when an international pharmaceutical giant comes to Ireland to offer prescription drugs at sometimes twice the rate they are sold for in other countries. There is a negotiation process and unfortunately a business model underpins it. It is of course about human life at all levels, but I reassure the Deputies that the final decision has not been made and the HSE's deliberations are ongoing. I welcome the Deputies' contributions to that process.