c4c welcomes the International Rare Diseases Research Consortium (IRDiRC) Guidebook, which aims to facilitate drug development for rare diseases by organizing available tools into a standardized framework.


The guidebook can be found in Nature Reviews Drug Discovery, and provides a framework for optimal use of existing tools available in the USA, Europe and Japan, referred to the “building blocks” of development.


A taskforce for the Guidebook mapped 110 building blocks available to orphan drug developers to create concise factsheets for each supportive tool.


The taskforce selected the most relevant building blocks and defined their optimal use during rare disease drug development, placing them into a milestone-based drug development framework.


The Guidebook will reduce delays in developing drug development by enhancing the use of available tools that will also reduce risks and costs,and improve patient and regulatory acceptability.


The c4c project is one of the resources in this framework, the role of c4c is described here.