RARE-NL aims to bring expertise together. By pooling our efforts, we aim to accelerate progress and improve outcomes for people affected by rare diseases. Embracing a ‘case to system’ approach, RARE-NL connects expertise, fosters collaboration and translates insights from individual cases into systemic solutions for the availability of rare disease therapies and drug repurposing. Lessons and knowledge from these examples are disseminated at systemic level through (academic) publications to improve patients’ sustainable access to repurposed medicines and treatments for rare diseases.

To promote the development of therapies for rare diseases and repurposing of existing drugs, new forms of regulation and cooperation in public-private partnerships (PPPs) are essential, aimed at both financial and societal added value. All parties should optimally combine their individual strengths and expertise to create such a value chain that covers the entire pathway from basic research to approved drugs.

RARE-NL is a collaborative initiative in which several university medical centres join forces as the ‘hub’ of FAST , the centre for Future Affordable Sustainable Therapy Development. FAST is an independent national centre of expertise and collaboration, promoting innovation in therapy development. FAST acts as a dynamic network infrastructure, bringing together knowledge, promoting dialogues and engaging a wide range of experts. The centre identifies key opportunities and challenges and addresses them through a comprehensive approach, such as use cases and pilot studies.