HLM RARE

Hosted in Brussels from 9-11 December 2025 by the European Reference Networks, MEP Vytenis Andriukaitis (S&D, Lithuania) and the Brains for Brain Foundation, this three‑day High‑Level Meeting on a European Innovation and Care Ecosystem for Rare and Complex Diseases (HLM Rare 2025) was a truly pivotal moment for the 30 million people living with a rare disease in Europe.

Each day of this meeting had a clear focus. Day 1, centred on research and innovation: how Europe can regain leadership in rare disease science by supporting clinical trials, translational research, and an ambitious Research and Innovation Union. Day 2 moved to infrastructure and skills: strengthening the European Reference Networks and EU capacity, including newborn screening, data spaces, and the use of artificial intelligence to shorten the diagnostic journey. Day 3 focused on policy and funding and how these can accelerate to turn innovation and infrastructure into timely, equitable access to care and treatments for all people living with rare and complex diseases across Europe.

A key milestone of this meeting was the launch of the Declaration on the European Innovation and Care Ecosystem for Rare and Complex Diseases. This Declaration is a political and strategic commitment to fundamentally transform our ecosystem. It sets out a bold shared vision, clear strategic priorities, and a roadmap that foresees, in the short term, the establishment of a Consultative Group to develop a concrete, measurable Action Plan, and in the medium to long term, accelerated progress for rare and complex diseases—through more treatments, universalised newborn screening, and advanced diagnostics.

Over the three days of our event we discussed and tested specific priorities and recommendations that the Declaration puts forward, so that this document becomes not just a statement of intent, but a practical guide for joint action in the years ahead.

Stay tuned as the next phase of this journey begins, with concrete steps to translate the Declaration’s priorities and recommendations into an actionable roadmap.

On behalf of the hosts, heartfelt thanks to all those who made HLM Rare 2025 a reality – in particular the members of our Steering Committee, our sponsors, the endorsing European Parliament political parties, and all our partner organisations whose expertise, trust, and support have underpinned this ambitious endeavour.