7. Deputy Shane Moynihan asked the Minister for Health if the Belgian model is being examined in the context of the programme for Government commitment to investigate early access schemes for rare disease treatments; and if she will make a statement on the matter. [34617/25]

Sentiment score: 0.13

The Minister is no doubt aware of the cases of those who suffer with Duchenne muscular dystrophy, a matter which has been raised in the Dáil a number of times, as well as with the Minister directly, not least because of the Thompson boys in my own constituency. This has brought into focus the need for an early access scheme for rare disease drugs. I know there is a commitment to this in the programme for Government that has been discussed before. What considerations is the Department is taking into account? Is it looking at other models internationally of how such a scheme might be applied?

Sentiment score: -0.06

It is great to hear about the progress that has been made in implementing the recommendations of the Mazars report on this subject that was published some years ago. That goes to the heart of the extra resourcing that the Minister spoke about. She is right; there needs to be a partnership between industry and the State to make sure that these applications are put through immediately and that the HSE can adjudicate on them accordingly. I am very encouraged to hear that the Department is considering other systems internationally, like the Belgian model. This model is particularly interesting because it shows a way in which timely access to these drugs that is not necessarily contingent on EMA approval can be provided. That is based on EU Regulation No. 726/2004. Article 83 of the regulation provides access to medications that are in late stage clinical development, expected to receive EU marketing authorisation and targeting patients with serious conditions. If we had those sorts of parameters for a scheme focused on early access to rare disease drugs, it would be a game-changer for many families that are afflicted with these conditions.

Sentiment score: 0.24

I appreciate the efforts of the Minister in this regard and I have conveyed that to the families involved. I am very grateful for the Minister's proactive approach. The beauty of getting an early access scheme right is that it does not necessarily rely on the proactivity of the State to be involved in that process but, rather, the partnership is hardwired into every piece of the system and the State is empowered to get value for money but also to ensure timely access to these drugs. In many cases of patients with rare diseases, time is the issue when it comes to stopping the deterioration of their conditions. I ask the Minister to keep us updated on the Department's progress in looking at these international models and to ensure that the best practice we learn from those is applied in any such Irish case.

Sentiment score: 0.53