Prof. Adriana Ceci, President of Fondazione Gianni Benzi, participated in the Thalassemia International Federation World Congress 2013, that has been held in Abu Dhabi from 20 to 23 October 2013, with a presentation entitled ‘A multi-ethnic multi-national approach to Ethical approval of clinical trials involving Thalassaemia patients: the DEEP lesson’ (Authors: Giannuzzi V., Cavallo M., Fattoum S., Nastas E., El-Beshlawy A., Ceci A.).

The DEEP project foresees the conduction of clinical trials to test the deferiprone drug in children affected by beta thalassaemia major and other hereditary haematological rare diseases, which obliged them to frequent transfusions. The experimental centres belong to European countries (Cyprus, Greece, Italy) and extra-European countries (Albania, Egyptum, Tunisia).

Fondazione Gianni Benzi, partner of the project, is actively involved in the regulatory aspects, included the submission of the studies to the ethic committees, facing the numerous challenges of clinical trials in DEEP. It has been necessary to take into account the regulatory, methodological and ethical aspects linked to the conduction of ‘research-driven’ clinical trials, that involve children affected by a rare disease living in countries with a different legislative asset and an heterogeneous ethnic and cultural context. It is for these reasons that an ad hoc approach has been adopted, evaluating all the following characteristics:

• a single submission dossier of the trials
• a trial management plan including numerous aspects, like the drafting of operative protocols (the so called SOP), data management, drug supply, pharmacovigilance, etc.
• ad hoc strategies to allow the “empowerment”, that is the active involvement, of little patients and their families, realising informative instruments for the children.

These documents, which have been submitted to the ethic committees, have been created to inform children of the objectives, benefits and risks of the study in which they are involved, taking into account the understanding capacities and the curiosity of the addressees.

The results achieved in DEEP represent a valuable example not only for the method of overcoming the difficulties linked to the conduction of clinical trials, but also because it has been proposed an innovative model of ‘patient-driven’ clinical trial.

The scientific material about DEEP presented during the Thalassemia International Federation World Congress 2013 is available in the attached documents.