Drug repurposing for rare: progress and opportunities for the rare disease community

• DOI: 10.3389/fmed.2024.1352803
• Publication Date: 2024-01-17T04:33:11Z
• Authors: Anneliene Hechtel...; Daniel O’Connor; Maria Cavaller-Be...; Christine Fetro; Maria Gogou; Peter A. C. ’T Hoen; Martin de Kort; Heather Stone; Nivedita Valentine; Anna Maria Gerdin...
• Keywords: Medicine (General); 0303 health sciences; 03 medical and health sciences; therapy development; R5-920; drug repurposing; orphan drugs; rare diseases; Medicine; Medical Biosciences - Radboud University Medical Center; unmet need; innovation

Repurposing is one of the key opportunities to address the unmet rare diseases therapeutic need. Based on cases of drug repurposing in small population conditions, and previous work in drug repurposing, we analyzed the most important lessons learned, such as the sharing of clinical observations, reaching out to regulatory scientific advice at an early stage, and public-private collaboration. In addition, current upcoming trends in the field of drug repurposing in rare diseases were analyzed, including the role these trends could play in the rare diseases’ ecosystem. Specifically, we cover the opportunities of innovation platforms, the use of real-world data, the use of artificial intelligence, regulatory initiatives in repurposing, and patient engagement throughout the repurposing project. The outcomes from these emerging activities will help progress the field of drug repurposing for the benefit of patients, public health and medicines development.