Tools for EU clinical studies: the rare disease clinical trials toolbox

Published on 28/02/2024
Rare diseases toolbox

February 29 is World Rare Disease Day, and at ECRIN we fully support all action taken by the rare disease community to support everyone involved in the battle against rare diseases. For this matter, we developed and published the Rare Diseases Clinical Trials Toolbox. This is a freely accessible ECRIN tool to facilitate clinical research in Europe, with a special focus on rare diseases. 

Drug development programmes in rare diseases have many challenges, some of which differ from those facing researchers working on more common diseases, like the lack of clinical research experts and the scarcity of patients. The 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. It aims to collect the accumulated knowledge, experience, and resources 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. 

The use of the Rare Diseases Clinical Trials Toolbox is easy as it is divided into 5 sections, representing the sequential phases in the development of a clinical trial: Research Question, Plan, Execute, Analyse, and End of Trial. Each section is then further subdivided into subsections to facilitate information retrieval.  The importance of each sub-section is the explained and followed by a list of identified tools. 

Explore the Rare Diseases Clinical Trials Toolbox for free on the ECRIN website 

Discover the tool 

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