The use of an existing medicine for a new indication is known as drug repurposing. The potential of this relatively new route for innovative applications is enormous, as the development trajectory is typically shorter and more cost-effective than that of traditional innovative medicines. Modern technology enables rapid screening for possible new uses. At the same time, however, this route to the patient still faces many obstacles. A viable business model is often lacking, and limited regulatory pathways frequently hinder the journey from initial discovery to consistent patient access. The few successful examples of drug repurposing illustrate both the immense untapped potential and the absence of a standardized development pathway.

RARE-NL is committed to breaking down the barriers surrounding drug repurposing. As a national collaboration hub, RARE-NL brings together academic institutions, industry, patient organizations, insurers, and government bodies to jointly work on concrete repurposing cases. By promoting knowledge sharing, encouraging public-private partnerships, and fostering sustainable business models, RARE-NL aims to establish a structural pathway from scientific discovery to accessible therapy. The goal: to fully harness the potential of drug repurposing for patients with rare diseases.