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!
The hearing, which aligns with the EU4H-2024-PJ-03 Call under the EU4H Action Grants 2024 and forms part of the public consultation launched by the European Parliament’s Committee on Public Health (SANT), served as a multi-stakeholder dialogue aimed at strategically positioning research and innovation within EU life science and health policy.
The Brains4Brain Foundation is delighted to announce the launch of an ambitious initiative aimed at addressing one of the most critical challenges in healthcare today: the establishment of a concrete ecosystem for rare diseases. To this aim, we are organising a preparatory event to present our project to selected policymakers -other MEPs but also representatives from the European Commission and the Member States – and a wider range of stakeholders within the rare disease ecosystem.
The challenges faced by healthcare providers and patients affected by rare diseases are multifaceted, and fragmented approaches have proven inadequate. There is a pressing need to align financial and non-financial incentives to secure sustainable, long-term investment in rare disease research and development.
We are excited to announce that this page will feature all the videos and pictures taken by participants during the B4B Annual Workshop and Special Celebration, held at Sidney Sussex College, Cambridge from September 9th to 11th, 2024.
In the spirit of David’s vision and dedication, we aim to once again breathe life into the B4B initiative.
