A collaborative group interview of four international researchers of the DEEP Network (Dr. Donato Bonifazi, Prof. Adriana Ceci, Prof. Oscar Della Pasqua and Prof. Amal El Beshlawy) appeared in Issue 162 of the International Innovation magazine in order to discuss the objectives and the outcomes of the European project DEferiprone Evaluation in Paediatrics (DEEP). The interview opens with an intervention of Prof. Adriana Ceci, who gave an overview on the DEEP project, and the advantages of a multinational program, describing also the background that led to the conception of the three clinical studies (DEEP-1, DEEP-2, DEEP-3), in a context characterised by the lack of sufficient knowledge on safety and efficacy data about the use of deferiprone for the treatment of children with β-thalassaemia major and other genetic haemoglobinopathies (some of the most worldwide severe forms of anaemia).
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.