We are proud to share with you the newly released informative video detailing the challenges and strategies involved in conducting the DEEP- 2 clinical trial across multiple countries for a rare paediatric disease. The video features insights from Mariagrazia Felisi, our Clinical Projects Director.

The DEEP- 2 trial investigated oral iron chelation therapy in transfusion-dependent hemoglobinopathies, enrolling 393 patients from 23 sites across Europe, North Africa, and the Mediterranean region. The researchers expanded their reach outside the EU to ensure an adequate sample size for this rare condition, allowing them to study potential ethnic/racial differences in drug response. However, operating across numerous countries presented substantial regulatory hurdles. These challenges included a lack of harmonized approval procedures, cultural differences around informed consent processes, and varying clinical practice guidelines to navigate.

The measures taken by the research team to overcome these obstacles, such as providing extensive training, adopting standardized materials while allowing national adaptations, and taking a centralized oversight approach through the DEEP consortium.

While the new EU Clinical Trials Regulation aims to streamline applications within member countries, multinational trials for rare diseases may still require expanding beyond EU borders to achieve adequate enrolment.

Overall, CVBF’s insightful video sheds light on the enormous collaborative efforts required to conduct large-scale multinational studies for rare diseases. It provides a first-hand look at the strategic approaches taken by researchers to navigate the various geographical and cultural complexities involved.

This video was produced as part of the European Joint Program for Rare Diseases project and will be available in the Innovation Management Toolbox (IMT), a reference library of resources for translational medicine in rare diseases.

We invite you to watch the video by clicking here.