Ms Avril Daly:

As Ms McGrath said, it is frustrating for our patients here, who are connected to patients in Europe. One must bear in mind that rare disease patients in Ireland, because of the fact they are small in number, are connected very much to their colleagues in Europe. Their frustrations are enormous. There are systems and discussions about a multiple-country approach to a health technology assessment process that would be uniform across Europe, speed things up and allow us to have our voice along with other countries, which is very important.

It goes back to the infrastructure. The infrastructure here is a problem from the very basics. We do not have the registers. There is a lot of talk about registers but there is no policy initiative stating that registers are a priority in this country. As I stated, we are relying on charitable organisations and ad hocsituations that may not be sustainable because they rely on various mechanisms to keep them funded. That really is not something that we, as a modern society, should condone. Our citizens do not deserve it. They want access to innovation and they want it quickly. The longer we delay, the more patients will develop further complications to their disease that will cost the country significantly more, so it is a completely false economy, as Ms McGrath stated.

As regards new recommendations, plans, strategies and frameworks for rare diseases, good and solid recommendations will come out of this if we all get involved in the discussion and making those changes, but it is also about transparency. It is very difficult for us, as patients, to see certain groups get access while others do not. We do not know why that is the case or on what the decisions are based. That is a challenge for us. What we see working in the rare disease community through Rare Diseases Ireland is that the various patients, from people who have a more common rare disease to those who have an ultra rare disease, are just completely lost. How are they supposed to campaign in respect of the way things are right now in this country and be the squeaky wheel when they are looking after their kids? We need a much more transparent and fairer system. It comes back to the basics of the infrastructure. If we get the infrastructure right, we will be in a much better position and engage much more effectively with our European partners and colleagues, which would speed things up enormously for us.