Together4RD position statement on collaboration between European reference networks and industry

Area of Science:

• Rare disease research and development
• European health policy
• Multi-stakeholder collaboration

Background:

• Limited treatments (5-6%) exist for rare diseases, impacting over 30 million EU patients.
• European research and innovation lag globally, necessitating a paradigm shift in rare disease R&D.
• European Reference Networks (ERNs) show promise for rare diseases but need robust support and collaboration.

Purpose of the Study:

• Analyze barriers limiting European Reference Network (ERN) and industry interactions.
• Propose strategies for enhanced multi-stakeholder collaboration in rare disease research.
• Introduce Together4RD's initiatives to foster effective partnerships and address unmet needs.

Main Methods:

• Analysis of existing barriers to ERN-Industry engagement.
• Review of case studies and collaborative frameworks.
• Development and piloting of multi-stakeholder interaction models.

Main Results:

• Identified tangible and perceived barriers hindering ERN-Industry collaboration.
• Explored potential frameworks and pilot initiatives for effective partnerships.
• Highlighted the need for financial, technical, policy, and infrastructural support for ERNs.

Conclusions:

• Overcoming ERN-Industry barriers is crucial for advancing rare disease R&D in Europe.
• Together4RD aims to facilitate collaboration through case studies, frameworks, and pilot projects.
• Strengthening multi-stakeholder interactions is essential to deliver life-altering treatments for rare conditions.