Drug development programmes in rare diseases 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. Moreover, academic sponsors and investigators end up getting involved not only in the scientific aspects of the research but also having to navigate the operational coordination and management themselves. 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.
The toolbox aims to collect the accumulated knowledge, experience, and [...]
