Tommy Broughan (Dublin Bay North, Independent) | Oireachtas source

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.

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