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Dáil
‹ Questions on Promised Legislation

Orphan drugs for rare diseases

Summary

Deputy Brassil argued that rare and ultra-rare disease drugs need a different approach because patient numbers are too small for normal evidence standards. The Taoiseach said any system still needed a way to test effectiveness and value for money.

I will take the few remaining Deputies if they are brief.

Comment on this

I will be brief. In terms of the programme for Government, Deputy Gino Kenny raised the issue of the provision of orphan drugs and drugs for rare diseases. In the Taoiseach's reply, he referred to establishing the efficacy of such drugs, but I remind him that, in some circumstances, there are only three, four or five patients affected by the disease in question. As such, establishing that type of information is next to impossible. We need to consider separately drugs for rare diseases, which affect approximately 0.05% of the population, and ultra rare diseases, which affect less than 0.02% of the population. If we do not, we will never get drugs that are badly needed for a very small population group.

I wish to ask about the drug Respreeza, which we have discussed many times in the Chamber. The company has agreed to supply it for the next six months free of charge, but the HSE is now in a battle with the company about who will pay for the drug's distribution over those months. The H3N2 virus, or the so-called Aussie flu, is out there. If it comes to these shores, the Respreeza patient group will be extremely vulnerable. We need to get this issue resolved. I call on the Taoiseach to intervene with the HSE.

Comment on this
Leo Varadkar The Taoiseach Fine Gael

It is a matter that would best be answered by way of reply to a parliamentary question to the Department of Health rather than by me as I do not have the up-to-date information.

Comment on this

It would be a good Topical Issue to table.

Comment on this
Leo Varadkar The Taoiseach Fine Gael

The Deputy makes a valid point on orphan drugs and ultra-rare diseases and I recognise his expertise in this matter. No matter what we do with orphan drugs or ultra-rare diseases, we need some mechanism to establish whether the medicine is effective and what represents a fair price as we need to ensure that patients receive drugs which are effective and which taxpayers can afford.

Comment on this