IRDIRC Orphan Drug Development Guidebook

The commentary ‘Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook’ has been published in Nature Reviews – Drug Discovery. This guidebook, which has been authored by the IRDiRC Task Force “Orphan Drug Development Guidebook (ODDG)”, is aimed at facilitating drug development for rare diseases by organizing available tools into a standardized framework. The Task Force worked for 2 years with more than 20 experts from the field of RD to help to 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.

In the current context, where developing drugs for rare diseases is a challenge due to several limitations and obstacles, the ODDG provides an innovative and unique model to expedite this process by systematically organizing the resources and tools available in Europe, US, and Japan. The project mapped the ecosystem of research and drug development in rare diseases, defining a compilation of Building Blocks and preparing factsheets for each of them with correlated recommendations and advice. By enhancing the use of the available tools, delays in development timelines can be avoided, risks and costs reduced, and patient and regulatory acceptability improved.

We invite the community at large to use this guidebook.

For more info, please visit the IRDiRC website.