FP7 Projects in Rare Anaemias : DEEP - Deferiprone Evaluation in Paediatrics

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FP7 Projects in Rare Anaemias : DEEP - Deferiprone Evaluation in Paediatrics. Adriana Ceci DEEP Scientific Coordinator, on behalf of DEEP Consortium. OUTLINE. 1- Project Specificities : 2- The Regulatory steps : PIP Clinical Trials in the project - PowerPoint PPT Presentation

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<p>FP7 Projects in Rare Anaemias: DEEP - Deferiprone Evaluation in Paediatrics</p> <p>FP7 Projects in Rare Anaemias: DEEP - Deferiprone Evaluation in PaediatricsAdriana CeciDEEP Scientific Coordinator, on behalf of DEEP ConsortiumOUTLINE1- Project Specificities:</p> <p>2- The Regulatory steps:PIP Clinical Trials in the project</p> <p>3- The DEEP CT facilitating strategy</p> <p>4- Status of the projects and preliminary results</p> <p>Project Specificity: Responding to EU Policy Needs(FP7-HEALTH-2010. 4.2.)only ~ 30% of marketed drugs are paediatric in Europea large Paediatric off-label use occurs as:Unapproved formulationsDrugs for adults not tailored for childrenAgeUK(%)It(%)NL(%)&lt; 233.020.032.12-110.41.626.412-172.02.042.5Total4.77.632.4Neubert and al, on behalf of TEDDY NoE,Pharmacol Res. 2008 Nov-Dec;58(5-6):316 </p> <p>Less than 50% of Paediatric Medicines have been studied in children</p> <p>Increase drugs and Trials in childrenGrant a Paediatric Investigational Plan before the trials will startIdentify therapeutic needsand Priority for fundingMeasures in the Paediatric Regulationensuring that new products (or variations) will be developed to meet paediatric needs according to PIPs agreed by the Paediatric Committee (art.7- art.8)Give a new MA (PUMA) to the existing medicines (OFF-PATENT) willing to developing at least one paediatric study (art.30). PIP is neededNewMAFunding of studies into off-patent medicinal products should be provided through the EU FRPs (art. 40) with the aim to develop a PUMA</p> <p>All the needs: ~ 20 therapeutic classes~ 400 active substancesProducts into the Priority List~ 13 therapeutic classes,~100 active substances</p> <p>Project Specificity: Responding to EU Policy Needs(HEALTH-2010-4.2-1 : Off-patent medicines for children)The Paediatric Regulation (Regulation (EC) No 1901/2006 of the European Parliament and of the Council on medicinal products for paediatric use was adopted on 12 December 2006 and entered into force on 26 January 2007. Its main provisions started to apply from July 2008 (Article 7) and January 2009 (Article 8), respectively.</p> <p>4Why Ferriprox was included in the Priority List-thalassaemia major and other genetic haemoglobinopathies The legal statusFerriprox obtained the EU MA under exceptional circumstances in Oct. 1999Off-patent drugThe relevance of the therapeutic AreaTo be used in rare and more severe forms of anaemia in the worldThe scarsity of approved chelators in some paediatric ages: Therapeutic NeedAge: &gt;2 and &lt; 6y SmPCs information: the only approved drug in this group of age is DFO. Oral chelators can be used if DFO is refused, inadequate or contraindicatedThe scarsity of clinical evidenceFew data in children </p>

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