Rare Diseases Clinical Trials Toolbox
This Toolbox has been developed as a practical aid for developers of clinical trials on medicinal products for human use regardless of therapeutic area
Drug development programmes in rare diseases (RD) have many challenges, some of which differ from those facing researchers working on common diseases, like the lack of clinical research experts and the scarcity of patients. This Rare Diseases Clinical Trials Toolbox has been developed as a practical aid for developers of clinical trials on medicinal products for human use regardless of therapeutic area.
Originally developed under the European Joint Programme on Rare Diseases (EJP RD), the Rare Diseases Clinical Trials Toolbox is now actively maintained and further developed within the ERDERA partnership, building on the EJP RD foundation to strengthen innovation capacity, collaboration, and knowledge-sharing across the rare disease research community.
The objective of the Toolbox
The toolbox aims to collect the accumulated knowledge, experience, and resources (collectively termed as ‘tools’) generated by previous projects and/or research infrastructures and other organizations into a practical and guided toolbox to help clinical trialists and R&D managers understand the regulations and requirements for conducting trials, with special focus on investigator-initiated trials for rare diseases.
- You will find a guide on how to use the toolbox here (under the section « How should you use the toolbox »).
- If this is your first time using the Toolbox, we recommend watching our short introductory tutorial to help you navigate the toolbox and find what you need quickly: « IMT Use Case: A Toolbox to Support Rare Diseases Clinical Researchers – RD Clinical Trials Toolbox »