NEW! ODDG tutorial & Interactive Section are available (see below)
Introduction
For the creation of the guidebook, different types of documents were created:
- A list of all Building Blocks
- A fact sheet form for each Building Block
- A power point presentation of the Guidebook, including different figures
- 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).
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
- BB Forms – Development practices
- BB Forms – Development resources
- BB Forms – Early access
- BB Forms – HTA and reimbursement
- BB Forms – Regulatory
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!