Moreover, Dr. Giannuzzi highlighted how this lack of harmonisation makes more difficult the approach in case of studies involving non-EU Countries, as DEEP, and especially ‘small populations’, such as children and patients affected by rare diseases, which typically are multi-centre and multi-national.

More details on the event and the list of all submitted abstracts are available here.

In such a scenario, “the development of a tailored oral chelator for children”- as explained by Prof. Ceci – “responds to a still unmet need in the treatment of haemoglobinopathies, including thalassaemia, which require chronic blood transfusions and, therefore, iron chelation”.

Prof. Della Pasqua clarified the innovative approaches, implemented to overcome sensitive issues and ethical concerns about the conduct of trials in small children, which have led to the development of a Pharmacokinetic study aimed at determining the appropriate dosing requirements in children below six years of age.

The achievement of the project objectives, as explained by Dr. Bonifazi, was made possible thanks to the joint collaboration of experts in the field of thalassaemia, haematology, paediatric clinical trial design and management, regulatory and ethics, who gathered together to form a network of research institutions, clinical centres and patients associations from both European and non-European countries.

In conclusion, Prof. El Beshlawy highlighted that, as reported by a recent cost-utility analysis “deferiprone still represents the most cost-effective treatment available for managing chronic iron overload patients”, if compared to the other available chelators, thus resulting in a more easily accessible, safe and efficacious treatment with substantial cost savings for health services.

The European Commission featured the DEEP Project in Issue #34 of the research*eu results magazine. With Prof. Ceci answering the editorial staff’s questions, the article focuses on the achievements of the project studies in the past three years, which led to a new formulation of deferiprone with a better taste suitable for children, as well as valuable data on the correct dosage to be administered for each age group.

The lessons learned so far? ‘The project takes advantage of the participation of private pharmaceutical companies experienced in developing medicinal products and making them available on the market. Without such collaboration it would be unlikely that the drug would be distributed to all children in need. The lesson learned is that public and private efforts should (and can) be brought together to address the patients’ best interest.’

The magazine can be found online here, and this specific issue can be downloaded here.

research*eu results features highlights from the most exciting EU-funded research and development projects. It is published 10 times per year in English, and covers:

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•    Social sciences and humanities
•    Energy and transport
•    Environment and society
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