The commentary ‘IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases’ has been published in Nature Reviews Drug Discovery (https://www.nature.com/articles/d41573-023-00168-9). This guidebook, authored by the IRDiRC Task Force “Drug Repurposing Guidebook”, facilitates drug repurposing for rare diseases by organizing available tools into a standardized framework. The Task Force worked for 18 months with more than 25 experts from the field of RD to help reach the IRDiRC goal 2: ‘1000 new therapies for rare diseases will be approved, the majority of which will focus on diseases without approved options’ by 2027 (https://irdirc.org/about-us/vision-goals/).
Drug repurposing has often been suggested as a critical solution for developing more therapies for rare diseases. However, repurposing approaches for rare diseases has not been as impactful as anticipated. Intending to address challenges in drug repurposing, and building on the Orphan Drug Development Guidebook, the project mapped the ecosystem of drug repurposing in rare diseases, defining a compilation of Building Blocks, by preparing factsheets for each of them with correlated recommendations and advice and a specific checklist for drug repurposing. This guidebook can support a wide group of stakeholders, such as academics, clinicians, small and medium-sized enterprises, and patient-led groups wanting to start a repurposing programme for a rare disease.
Read the full paper: here.