Together for Rare Diseases is a multi-stakeholder alliance supporting ERNs and industry to collaborate for the benefit of the rare disease community. Recently, the Together4RD Steering Group has published a new position statement on ‘Collaboration between European reference networks and industry’.
The paper highlights that, despite two decades of policies and legislation in Europe to promote research and development for rare diseases, only 5–6% of such diseases have dedicated treatments. This poses a significant unmet need for over 30 million patients in the EU alone. The pace of research and innovation in Europe lags behind other regions, necessitating a fundamental shift in research planning and delivery to bring meaningful treatments to patients. The European Reference Networks have the potential to address these challenges, but they require adequate financial, technical, and policy support. There is a need for robust collaborations, including with the industry, to effectively tackle rare diseases. Despite existing barriers, the Together4RD initiative aims to overcome these obstacles, learning from case studies, exploring collaboration frameworks, and launching pilots to address real research needs.
Access the full position statement here.