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IRDiRC publishes Orphan Drug Development Guidebook

Over the past 2-3 years, IRDiRC has been working with their Therapies Scientific Committee, on a project called Orphan Drug Development Guidebook. The paper describing the work performed is now published in the format of a commentary in Nature Reviews Drug Discovery. This Guidebook aims to provide all types of developers (including Patient Organizations involved in R&D) with tools and recommendations in order to navigate the complexity of the medicines development ecosystem.

The Orphan Drug Development Guidebook Taskforce had been working with more than 20 experts from the field of Rare Diseases during 2 years in order to help reaching IRDiRC goals. Namely the goal 2 of the IRDiRC Consortium that is ‘1000 new therapies for rare diseases will be approved, the majority of which will focus on diseases without approved options’ by 2027. 

An illustration of the Orphan Drug Development Guidebook. Source: IRDiRC

To reach this goal, the Taskforce started from the idea that a change is needed in the way drugs are developed. To enable this change, new practices should become standard elements of the development framework. To fully integrate these new elements within a defined development plan generating better data quality, shorter development timelines and lower development costs/ better R&D efficiency, the entire development framework should be re-engineered. 

About the Guidebook

The “Orphan Drug Development Guidebook” is a guidebook for academic and industrial drug developers describing the available tools and initiatives specific for rare disease development and how to best use them. The project consisted in mapping the eco-system of research and drug development in rare diseases, defining Building Blocks (representing more than one hundred regulatory tools, HTA, reimbursement and early access tools but also development practices and development resources) and preparing a fact-sheet for each of them in which we included the advice from our group of experts. 

The expertise of the multi-stakeholder group has been gathered all along the duration of the project but also with the organization of a workshop where a series of standards cases of drug development have been used to define ‘how’ and ‘when’ to use these tools and engage in the various initiatives. 

The compilation of the Building Blocks, the knowledge generated through the case studies and a series of practical check-lists of what to consider at each phase of development constitutes the Orphan Drug Development Guidebook. The materials will evolve as we are also developing an interactive platform, jointly with the European Joint Programme on Rare Diseases, in order to allow a more user-friendly navigation through the various materials.