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 need to optimally combine their individual strengths and expertise to create such a value chain covering the entire pathway from basic research to approved drugs.

RARE-NL’s activities aim to support academic parties and get socially responsible public-private partnerships off the ground and focus on:

Drug repurposing: Het ontwikkelen van bestaande geneesmiddelen voor nieuwe toepassingen.
Therapieën voor zeldzame ziekten: Het ontwikkelen van behandelingen voor zeldzame aandoeningen.