Leo Varadkar (Dublin West, Fine Gael) | Oireachtas source

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.

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