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

To promote the development of therapies for rare diseases and the repurposing of existing medicines, new forms of regulation and collaboration in public-private partnerships (PPPs) are essential, focusing on both financial and societal value. All parties must optimally combine their individual strengths and expertise to create a value chain that spans the entire trajectory from fundamental research to approved medicines.

RARE-NL’s activities are designed to support academic institutions and foster socially responsible public-private partnerships, with a focus on:

• Drug repurposing: Developing existing medicines for new applications.
• Therapies for rare diseases: Developing treatments for rare conditions.