Reinvigorating the possibilities: Drug repurposing as a strategic access pathway for rare diseases

With over 8000 rare diseases identified and treatments available for only a fraction, the urgency for accelerating therapeutic development is paramount. Drug repurposing – the process of identifying new uses for existing drugs – may offer a faster, more cost-effective approach than de novo drug development. Thus, the concept of drug repurposing for rare diseases was a hot topic during the RE(ACT) Congress and IRDiRC Conference held in Brussels on March 5–7, 2025. This article draws from the expert panel presentations and discussions, exploring the landscape of drug repurposing in rare diseases, including the opportunities, challenges, and examples of success. Key themes include collaborative infrastructure, data-driven innovation, patient-centricity, and the need for tailored regulatory and commercial strategies.