Clinical Trials

The overall objective of Work Package 20 is to foster the implementation of recently developed innovative methodologies for clinical studies in rare diseases in practical settings as well as to develop innovative tailored methodologies for clinical trials in rare diseases with unmet needs.

To realize the objective the following steps will be implemented:

  • Improving clinical studies methodology in Rare Diseases will come into force by a helpdesk supporting ERNs and other clinical teams involved in Rare Diseases
  • Disseminate of the latest development in trial methodology including the methodologies proven to be successful for clinical studies in Rare Diseases.
  • Implementation of innovative methodologies to Rare Disease clinical studies to derive experience in practical settings and understand the value of these methodologies.
  • Identify unmet needs for tailored methodologies and develop innovative methodologies for Rare Disease clinical studies.

Once validated, agreed and accepted by all stakeholders, the innovative methodologies will encourage the accomplishment of IRDIRC goals.