ERDERA Management of multinational clinical trials for rare diseases

The ERDERA Training on Management of Multinational Clinical Trials for Rare Diseases, developed by ECRIN, is an online training course designed to support clinical research teams involved in planning and managing multinational rare disease trials. The course builds on ECRIN’s extensive experience in coordinating multinational clinical studies and focuses on practical solutions to common barriers affecting the initiation and implementation of rare disease clinical trials. It is aimed at principal investigators, sub-investigators, study coordinators, clinical trial unit personnel and project managers involved in rare disease research.

The training addresses key aspects of multinational trial management, including early planning of clinical studies, identification of stakeholder roles and responsibilities, coordination between countries and sites, regulatory and ethical considerations, funding sources and budget preparation. It aims to help trial teams anticipate operational challenges and develop strategies to improve harmonisation and efficiency across participating centres.

This resource is particularly relevant for rare disease clinical trials because these studies frequently require multinational collaboration due to limited patient populations, geographically dispersed participants and the need to access specialised clinical expertise. Effective trial management, coordination between countries and early identification of operational bottlenecks are critical factors influencing recruitment, timelines and overall study success.

The training also incorporates aspects of responsible research practices and patient engagement, recognising the importance of involving patients and stakeholders in rare disease clinical research. By strengthening the skills of research teams, this resource contributes to improved trial readiness and supports the successful delivery of complex multinational rare disease clinical studies.

Although developed within the European ERDERA and ECRIN framework, the principles and operational approaches presented are broadly applicable to multinational rare disease trials internationally, particularly studies requiring collaboration between multiple institutions, countries and healthcare systems.