IRDiRC Drug Repurposing Guidebook

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The International Rare Diseases Research Consortium (IRDiRC) has developed a new guidebook to invigorate drug repurposing for rare diseases.

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.
With the aim of addressing the challenges in drug research and development (R&D) for rare diseases, IRDiRC developed the Orphan Drug Development Guidebook (ODDG).
This resource, which was launched in 2020, provides a set of materials that describe the available tools and initiatives for rare disease drug development (called building blocks) and how best to use them; factsheets for each of them with correlated recommendations and advice and a specific checklist for drug repurposing were prepared. This ODDG guidebook supports 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.
Moreover, the ODDG guidebook focused on a set of development cases, including traditional, well-understood drugs (such as small molecules) and highly innovative technologies (such as cell and gene therapies), but did not take drug repurposing into consideration. So, the IRDiRC’s Therapies Scientific Committee set out to develop this new Drug Repurposing Guidebook (DRG).
The new DRG builds on the ODDG and has been created for researchers and developers involved in drug repurposing in the rare disease field. The DRG project was set up as a task force of 23 experts, representing patients, regulators, academics, industry, health-care professionals, public funders, intellectual property experts and consultancy firms.
The task force conducted a gap analysis of the current ODDG to confirm which of the materials could support a repurposing programme and which elements were missing. Such exercise resulted in an overview of the most important tools and practices currently available for sponsors of drug repurposing projects. The ODDG mapped the ecosystem of drug repurposing in rare diseases, defining a compilation of Building Blocks,

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