Orphan Drug Development Guidebook Materials

NEW! ODDG tutorial  & Interactive Section are available (see below)


For the creation of the guidebook, different types of documents were created:

  1. A list of all Building Blocks
  2. A fact sheet form for each Building Block 
  3. A power point presentation of the Guidebook, including different figures
  4. A tutorial, explaining the Guidebook in detail

Building Block List

A list of building blocks (BBs) (tools, incentives, resources, initiatives, and practices) either specific to rare diseases, or more often used in rare diseases but also available for common diseases were collected and analyzed. The full list of BBs available in Europe, United States of America and Japan is shown below (please note that this is list might not be complete and it will be regularly updated).

List of BBs

Building Block Forms

Based on a systematic review of websites, literature search and the expertise of the Taskforce members, we have created fact sheets describing each BB by including key information on its use, duration, pros and cons, and among other aspects the TF’s advice on the best time to use the BB. All fact sheets can be found below. You can access to the BB forms by type or by geographical scope.

By type

By geographical scope

Guidebook Presentation 

A more comprehensive representation of the guidebook and its methodology has also been created in following PDF file:

Publication in Nature Reviews Drug Discovery

Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook

NEW! Tutorial

In order to understand more on the guidebook, a ODDG tutorial can be watched, discussing all the different elements of the Guidebook, the methodology followed and the way to use it

NEW! Interactive Section

All the materials from the Orphan Drug Development Guidebook are now available on a user-friendly website. Go and check it out!