Debate: A European Action Plan Against Rare Diseases
Date: WEDNESDAY, 24 NOVEMBER 2021
Speaker: Stella Kyriakides
Language: EN

Mrs Kopacz, you said that all patients, no matter where they live, should have equal access to treatment, and I would agree totally that this is not negotiable. And Mrs Guteland said it’s not easy to build up expertise and competence in all Member States and that shows exactly the importance of European reference networks. Several of you, I think it was Mrs Kopcińska and others, mentioned the importance that we support a European reference networks, and I totally agree. I’m working on this too. They deserve the support. I am working to find the solution. We will be supporting their further activities and looking how to do this within our EU4Health programme.  Mrs Ries, you mentioned Red 2030 and I think I need to just mention here that we are already looking at the outcomes of this study, and I am pleased to note that many of these recommendations of the study are already being taken on board.  Mrs Metz also mentioned the European reference networks, and several of you, and I made some notes here, spoke about rare diseases in the forms of paediatric cancers. I know that Madame Véronique Trillet-Lenoir is an expert in the area, and this is why we dedicated a specific area of Europe’s Beating Cancer Plan to paediatric cancers. And this is why we are, in conjunction with the plan, looking at the revision of the orphan and paediatric regulations, which aim to address the problems that we now face in terms of treatments and innovation. I want to assure you that all medicinal products, which could be efficacious in treating paediatric cancers, are effectively studied in children. And we would look at the experience acquired in the US with their recent change of legislation for medicines for children to address this issue. And Mr Fourlas, we will be giving incentives to industry to boost innovation in areas where we have medical needs. But we will also impose obligations to ensure better access to medicines, to ensure that inequalities disappear within the EU.  Several of you also mentioned the issue to do with stigma and awareness and the need to really change it, and I can’t agree more with this. And Mrs Moretti also mentioned the issue to do with the (Red) 2030 project and the need to have the evaluation of this, but we need to widely consult and I just wanted to say that before deciding the way forward. So I just wanted to say a few words in terms of where we are with the European Reference Networks. We will also grow the ERNs and these are already providing answers and diagnosis and treatment plans for more than 1.7 million patients who before only had, as many of you said, uncertainty and doubt. And in January 2022, the new ERNs will be invited to join the existing 24 networks, almost doubling their size to comprise around 1500 specialised healthcare units across the EU.  And so we are consistently moving forward with this. We’re supporting Member States in the development of their national rare disease plans. We’re working within the Pharmaceutical Strategy for Innovation and increase support for our medical needs. And as many of you mentioned, we’re preparing the proposal for the European Health Data Space and through the European Platform on Rare Diseases Registration. We’re working towards a framework to counter data fragmentation.  So, all I can say in ending is that we as a Commission and myself, as Commissioner of Health and Food Safety, we are committed. We’re committed to continuing to make concrete and tangible improvements and changes to the lives of rare disease patients, of their families, of those who work in the areas of rare disease, with commitment and dedication, as we have already been moving for the last 20 years and I look forward to working very closely with this Parliament and having the opportunity to stand before you again in the not so distant future and share with you how we have moved on with this.