Donato Bonifazi, DEEP Project Manager presented the project in the context of the international paediatric trials that have to face a default level of complexity, due to regulatory, methodological, ethical and administrative issues, especially when they are registrative and international as DEEP is.

In particular, the specific challenges that DEEP has addressed and is still facing with reference to the recruitment and patient’s retention have been described, since DEEP is a ‘Registrative’ CTs with economic burden, ethical stringent provisions and GCP-ICHE11 obligations that involves not only a paediatric multi-ethnic population with different cultures, laws and children of different ages but also a rare and disperse population involving different Rare Congenital Anaemia.

Moreover, the DEEP multisteps strategy to address these issues has been presented, resulting from the following key aspects:

• the organisation of a ‘trials management plan and infrastructure’ including SOPs preparation, data management, drug management, pharmacovigilance, monitoring, etc;

• the implementation of a unique procedure and a unique CTA ‘package of documents’;

• the developments of a ‘patients tailored approach’ including children, families and association.

To conclude Donato Bonifazi updated on the patients’ recruitment that is currently proceeding successfully thanks to a continuous and coordinated activity of the TMT and the PIs and the continuous discussion of the Protocol-related issues among Principal Investigators, Trial Management Team and all the study key contacts that has led the inclusion of many new centres.

The event was also the occasion to discuss, in the framework of an international multi-stakeholders debate, the present and the future of the paediatric research including the efforts made by the scientific community and the ongoing projects such as the proposed Paediatric Research Infrastructures (EPCTRI) and the global networks.

The ‘Foresight Training Courses’ (FTCs) are short intensive courses devoted to create a critical mass of expertise in the different areas of Regulatory Sciences (medical devices, foods, medicinal products), with particular reference to the innovative advancement of the European Pharmaceutical and Biological products sectors. The meeting was the occasion to present DEEP with its achievements and results, and to focus the attention of the audience on congenital haemoglobinopathies, the most common congenital rare diseases in the world.

The importance of an international network and the innovative nature of the project have been highlighted by Dr. Donato Bonifazi, Project Manager of DEEP and CEO of CVBF: “DEEP belongs to the small number of ‘investigator-driven’ paediatric clinical trials; a study based on a recognised ‘therapeutic need’ and sponsored by a research group that will lead to the collection of information intended for regulatory purposes; a multi-ethnic Network in which the complexity of conducting paediatric clinical trials has to take into account the existing rules at European and non-European level. The biggest challenge of the project is to perform its clinical trials in accordance with the European ethical requirements for studies involving the paediatric population”.

The experience of Policlinico of Bari, as Coordinating Centre of the DEEP-1 Clinical Trial and host of the Drug Management Service for all Italian recruiting centres, was presented by Dr. Bonifazi, with particular reference to its key-role in performing management and clinical activities. The innovative role and tasks of the Hospital Pharmacy of the General Hospital of Bari were described by Director Dr. Lattarulo.

“The pharmacokinetic study allowed us to identify the optimal dose of deferiprone for children under 6 years and ongoing studies will provide us with important additional information on the efficacy and safety of this drug in pediatric patients,” stated Dr. Giovanni Carlo Del Vecchio, Unit of Paediatrics, while Mr. Sebastian Botzenhardt University Hospital Erlangen (Germany) provided updates on DEEP-3.

The meeting provided an opportunity to present DEEP, a European project specifically designed for children and aimed to better define the safety, the tolerability and the efficacy of this drug in order to make its anticipated therapeutic benefits available to the entire paediatric population.

In this occasion, the project poster was distributed among participants and exposed during the whole course. The full programme of the DEEP meeting and the list of speakers are available here. Here you can also download all the presentations of the speakers.

For further information on all the scientific events, please refer to the brochure of the VII Foresight Training Course, that can be browsed even through mobile devices.