'?-thalassaemia major is one of the most severe forms of chronic congenital anaemia. The recommended treatment consists in regular blood transfusions combined with chelating therapy to remove harmful iron accumulation in the body. The use of deferoxamine, the first chelating agent only available for subcutaneous administration is limited due to toxicity and the lack of compliance, despite its satisfactory therapeutic effects. An oral iron chelating agent, deferiprone, was authorised in Europe in August 1999 and recommended for the treatment of iron overload in patients with thalassaemia major when deferoxamine is contraindicated or inadequate. Despite a wide experience of the administration of deferiprone for thalassaemic patients, limited data are available on its use in children below 10 years and the need for additional data in this age subset was clearly indicated in the 2009 priority list approved by the Paediatric Committee at the European Medicines Agency (PDCO). In addition, according to the recent scientific advancements and in consideration of the anticipated benefit of this chelator in controlling cardiac iron overload, studies evaluating the effects of the deferiprone in all the paediatric ages and in all transfusion-dependent chronic congenital anaemia (including Sickle Cell Diseases) were also considered a critical therapeutic need. The DEEP project, in line with these premises, has been funded with the specific aim to produce a new oral liquid formulation of deferiprone suitable for the paediatric use and to provide evidences for the use of this chelator as first line therapy in the whole paediatric population (from 1 month to 18 years) affected by transfusion-dependent chronic anaemia. The condition under study in the DEEP project is rare. This poses special difficulties in the conduct of the studies due to the small patient population and the need to involve a large number of recruiting centres . However, being dedicated to develop an orphan drug, DEEP has been also recognised in the context of IRDiRC, the International Rare Diseases Research Consortium devoted to repurpose/develop 200 new drugs for Rare Diseases by the end of 2020. Main features of the DEEP project are: -The innovative design of the clinical studies including pharmacokinetic modelling for the definition of the most appropriate dosage of deferiprone in younger children, the cardiac MRI T2* evaluation as primary endpoint, a three years safety study aimed at evaluating deferiprone, in monotherapy or in combination, in the real world's setting and, for the first time, a comparative efficacy-safety trial to compare the two existing oral chelators: deferiprone and deferasirox. -The DEEP Consortium including European and non-European Countries from the Mediterranean region where the transfusion-dependent congenital anaemia, in particular ?-thalassemia major, is particularly widespread: the collaboration within a multinational and multicultural network makes the Project extremely challenging due to many different ethical, methodological and social approaches to be explored and positively addressed.'

Chronic pain is estimated to affect 15-20% of children with varying disabilities. Due to the paucity of clinical information and appropriate medicinal products, in clinical practice paediatric patients are often under-treated causing profound impact on their QoL. To date, opioids, non-steroid anti-inflammatory drugs (NSAIDs), antidepressants and anticonvulsants are used to treat pain, but only few of these include a paediatric authorisation. In addition, the few available paediatric drugs have a very different authorisation profile across Europe and, when not approved in paediatrics, they are used off-label to cover the high therapeutic need, thus exposing children to unnecessary risks of dosing errors and increasing ADRs. The GAPP Project is focused on the development of gabapentin for the treatment of paediatric chronic pain, a condition where gabapentin, as demonstrated in adults, is expected to bring great benefit to children. To this end, a large international scientific Consortium is set up, with experienced professionals in the field of pain that will work together with a Pharmaceutical Company committed to apply for a PUMA using the project results. Within GAPP, age-appropriate formulation will be developed and two randomised, comparator-controlled clinical trials and a bridging study will be conducted in compliance to a submitted Paediatric Investigation Plan (PIP, Summary Report Day 90 - EMEA-001310-PIP01-12) in order to investigate appropriate dosages, efficacy and safety of gabapentin in the paediatric population. Further to this, in order to ensure the safety of gabapentin in very young children, a non clinical study will be conducted to evaluate the neurotoxic potentil of the drug. At the end of the project, gabapentin can be proposed for treatment of chronic pain in children both as monotherapy and as adjuvant therapy.