I am also voting "Yes" and calling for a "Yes" vote on Friday but I want to raise another health matter of public importance. The Taoiseach set up the National Rare Diseases Office when he was Minister for Health in 2015. By definition, rare diseases are rare but the office estimates that something like 300,000 Irish people will develop a rare disease at some stage in their lives. Recurrent complaints to us concern the lack of clinical supports and the tortuously slow process for the approval of orphan drugs for these diseases. A case in point is the failure since 2009 to approve the drug Kuvan, or sapropterin dihydrochloride, under the process for national pricing and reimbursement. Kuvan was developed to assist citizens with the rare metabolic condition phenylketonuria, PKU, where patients have difficulty processing the amino acid phenylalanine and must live on very restricted low-protein diets. Kuvan helps many of those affected to live more normal lives. PKU was first identified in the 1930s, although the widespread heel prick test screening did not start until 1960s. Failure to give treatment to a child after the first 48 hours results in serious cognitive impairment and often severe brain damage.

In 2009, the European Medicines Agency and 20 countries approved the use of Kuvan. The Minister for Health told me that at that time, insufficient evidence was submitted by Merck Serono, the manufacturers. It passed on the market authorisation to BioMarin in 2016. BioMarin submitted a new health technology assessment for sapropterin to the HSE. Nine months ago, I asked the Minister for the results of the assessment by the National Centre for Pharmacoeconomics. My constituents and I were very disappointed to find that Kuvan was not recommended for reimbursement last September. It was said to be not cost-effective, although even a 100% uptake has been estimated to cost only around €2.5 million per year. The figure for the per capita cost, the Taoiseach may note from his past work, is relatively small. In several Dáil replies, the Minister has told me that the assessment process is ongoing and that the rare diseases medical products technology review committee is engaged with prescribers, specialist centres and the patient representative group to try to bring forward clear reimbursement guidelines that would be expected to target sapropterin to the patient cohort for which it may be a cost-effective intervention. I appreciate the Minister's diligent responses relating to Kuvan over the past two years but after nearly ten years of assessment and as one of the countries most affected by this condition, is it not time to approve it? As the Taoiseach may be aware, one in 4,500 babies is born with PKU in Ireland. This is double the European average. I think only Italy and Turkey are somewhere near us. It is said to result from one of our Celtic genes. In 2015, the Minister told me that there were something like 350 or so referrals and 20 new PKU patients per year in the metabolic disorders unit at Children's University Hospital, Temple Street. It is estimated that around 750 patients with PKU are receiving hospital treatment. Representatives from the PKU Association of Ireland, the European association and other patients and relatives have been in regular contact so there is a very strong request that this drug would be approved.

The Deputy will be aware that we have a rare disease strategy. I think it was published by the then Minister for Health, James Reilly, a few years ago. One of the recommendations in that was to establish the National Rare Diseases Office, which I had the pleasure of opening during my time as Minister for Health. If one takes rare diseases on their own, they are rare but when one adds them up together, quite a lot of people have one. They include diseases like PKU, which the Deputy mentioned, haemophilia, cystic fibrosis and others. When one adds up all of the people who have a rare disease, it comes up to a couple of hundred thousand people. It does not seem so rare when one does that.

In terms of any new medicine, including Kuvan, a couple of things must be assessed. First, its effectiveness must be assessed. Does it actually do what the manufacturers say it does in terms of modifying disease and improving quality of life and life expectancy? Second, is the price being put forward by the manufacturer fair because even the richest countries are budget-limited and if we pay too much for something, there is an opportunity cost relating to the other things we cannot do as a result? Another factor is whether alternative treatments are available that might be as effective or most cost-effective. I do not think there is in this case. I believe it is an orphan drug. The assessment is done by the National Centre for Pharmacoeconomics in St. James's Hospital. It carries out a detailed assessment of the medicine and makes a recommendation to the HSE's national drugs committee, which makes the final decision. It is not and should not be a decision that is made by politicians for lots of reasons we all understand. It is always the case that if the manufacturer has new information and data, it can submit that and it will be examined again. I understand that when it comes to Kuvan, that is currently being done and that the application is currently being reassessed. I know the Minister has met with the parents of children who have the disease and he will, of course, provide the Deputy with an update as soon as possible.

Kuvan is actually a synthetic copy of the BH4 deficiency, which causes PKU. There are many heart-rending accounts of the daily struggles of families with children with PKU to stay within their low-protein allowance, particularly given that there are few low-protein foods available in Ireland. As I said, it is ten years since the European Medicines Agency approved it for market authorisation. The health systems in most of our EU colleague countries have supported it. The Orphanet Journal of Rare Diseases, of which the Taoiseach will be aware, published the complete European guidelines on PKU diagnosis and treatment last year. The journal says that diet plus medicine is the best control of the condition.

On the wider issue of orphan drugs, including Kuvan, does the Government foresee making any changes to the quality of adjusted life measurements in the National Centre for Pharmacoeconomics assessments? I have put that question to the Minister several times because it seems to be the key point when one starts talking about cost-effectiveness. Are officials in the Department of Health and the HSE working on a fairer way to assess the efficiency of treatments for rare diseases? The Taoiseach mentioned the national rare disease plan from 2014 to 2018. Are we preparing a new plan from 2019 onwards? I know that when the Minister set up the National Rare Diseases Office, it started with five part-time staff. I know the Government did not provide any additional funding in 2017 so are further funding and resources being provided for that? Above all, it is now time, as the Minister for Health will probably agree, to make sure Kuvan is approved.

The Minister has met the patients affected and their families.

He has committed to giving the Deputy an update on the status of approval for Kuvan specifically and also to give an update on funding for the rare diseases office.

As a broader issue, the Government acknowledges we have a problem here in that the process by which we approve drugs generally may be inappropriate for orphan drugs because it is the only one available; that is the nature of an orphan drug. However, the pharmaceutical industry takes advantage of the situation. When a number of medicines are available for a particular condition, pharmaceutical companies cannot overcharge for them. However, a sole producer of the only medicine can charge whatever it likes. That is an enormous problem that we and other countries are facing.

The process we have for assessing new medicines is robust and good. It is scientific and independent. However, I am concerned when I see medicines available in Britain that are not available here. The NHS is not renowned for approving new medicines quickly - for example, it has not approved Orkambi for patients with cystic fibrosis. I am concerned when I see medicines available in Britain but not available here. I find it very hard to justify. The Minister, Deputy Harris, and I are in conversation with the Minister for Finance as to how we might improve that in the near future.