We are delighted to invite you to the upcoming High-Level Meeting (HLM) on a European research and innovation ecosystem for rare diseases, taking place on December 9-11, 2025, in Brussels.

This three-day event will gather key stakeholders, including EU policymakers, industry leaders, patient advocacy groups, researchers, and healthcare providers. Our goal is to identify concrete actions and incentives to enhance innovation across the rare disease ecosystem. By involving all actors in the development, approval, and access to new therapies, we aim to establish a predictable regulatory pathway, ensuring that the benefits of scientific advancements reach all European citizens, particularly those often left behind.

Inspired by the recent recommendations from the Draghi, Letta, and Heitor reports, our agenda will focus on the following key topics:

  • Day 1: Fostering competitive excellence in science and innovation through support for fundamental research, clinical trials, and translational research.
  • Day 2: Building pan-EU infrastructure to strengthen European Reference Networks and enhance capacity, including newborn screening and the use of data and AI to expedite diagnosis and treatment initiation.
  • Day 3: Overcoming fragmentation by establishing a coherent policy and funding regulatory framework specific to rare diseases, highlighting the importance of cross-border care.

We are pleased to announce that the list of confirmed participants is growing week by week. Notably, we look forward to the presence of Mr. Letta, and we are in discussions with Mr. Heitor and Mr. Draghi to secure their attendance as well.

A key objective of the HLM will be to advocate for the EU’s adoption of a European Declaration on Rare Diseases. This formal commitment aims to create a sustainable Research and Innovation Ecosystem for rare diseases, ensuring ongoing dialogue and collaboration among all relevant stakeholders. Additionally, we will push for dedicated funding to be allocated within the next Multiannual Financial Framework (MFF) 2028-2034.

Please save the date for this important event. Further details regarding registration and the detailed agenda will follow soon.

We look forward to welcoming you to Brussels!