The High-level meeting „HLM rare 2025“ was held in Brussels from 9 to 11 December 2025 and was hosted by the European Reference Networks, MEP Vytenis Andriukaitis, and the Brains for Brain Foundation. The meeting aimed to strengthen a European Innovation and Care Ecosystem for Rare and Complex Diseases, addressing the needs of approximately 30 million people living with a rare disease in Europe.
Each day of the meeting focused on a specific theme. The first day concentrated on research and innovation, highlighting the need to support clinical trials and translational research and to help Europe regain leadership in rare disease science through an ambitious Research and Innovation Union. The second day focused on infrastructure and skills with particular emphasis on strengthening the European Reference Networks, expanding EU capacity, and using tools such as newborn screening, shared data spaces, and artificial intelligence to shorten diagnostic pathways. The third day addressed policy and funding, exploring how these can accelerate the translation of innovation into timely and equitable access to care and treatments across Europe. A key outcome of the meeting was the launch of the Declaration on the European Innovation and Care Ecosystem for Rare and Complex Diseases. This Declaration represents political and strategic commitment to fundamentally transform the ecosystem by setting out a shared vision, clear strategic priorities, and a roadmap for action. It also forsees the establishment of a Consultative group and the development of a concrete and measurable action plan. In the medium to long term, the Declaration aims to accelerate progress in rare and complex diseases through increased availability of treatments, universal newborn screening and improved diagnostic capabilities. The Declaration contains 8 major priorities that were agreed upon and discussed and which will be the roadmap to the next steps and activities.
