Rare Diseases Clinical Trials Toolbox

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.

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. 

How should you use the toolbox?

You will find a guide on how to use the toolbox here (under the section “How should you use the toolbox”).
You can download the full list of tools on the ECRIN website here
You can watch here the video “IMT Use Case: A Toolbox to Support Rare Diseases Clinical Researchers – RD Clinical Trials Toolbox”