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European Congenital Heart Disease Organisation (ECHDO) Berlin 23 – 24 March 2007

H. W. Seyberth Chairman of the Commission on Drug Safety of German Society of Pediatrics (DGKJ) Member of the Paediatric Working Party (PEG) at the EMEA. European Congenital Heart Disease Organisation (ECHDO) Berlin 23 – 24 March 2007. Save Drugs for Children with CHD:

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European Congenital Heart Disease Organisation (ECHDO) Berlin 23 – 24 March 2007

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  1. H. W. Seyberth Chairman of the Commission on Drug Safety of German Society of Pediatrics (DGKJ) Member of the Paediatric Working Party (PEG) at the EMEA European Congenital Heart Disease Organisation (ECHDO)Berlin 23 – 24 March 2007 Save Drugs for Children with CHD: New European Regulation

  2. The labelled percentage of 41 “standard drugs” used in a neonatal and pediatric intensive care unit(Heidelberg, 1981). – newborns infants children 100% 23 5 13 – 50% 57% 32% 11% 0% H.W. Seyberth, Kinderarzt 1984

  3. Consequences of unlicenced and off label drug perscriptions: No dosing regimen No warning of possible adverse drug reactions No information about possible drug interactions No product liability for the drug by the sponsor No long term surveillance No adequate pediatric formulations

  4. When we choose a drug for a child ... We rely on: - Personal experience (trial/error)? - Advice from colleagues/mentors? - Anecdotal reports in the literatur? - Extrapolation from adult data? - Small „trending“/inclusive trials?

  5. Adverse Drug Reactions (ADR) of unlicensed and of label drug prescription in paediatrics ADR % In label and licensed - 10 Of label and unlicensed 6.0 - 5 3.9 3.4 1.4 0 In the outpatient clinics (FR) On the wards (UK) (Horen et al, Brit J Clin Pharm 2002) (Turner et al, Acta Paed. 1999)

  6. What has happened in the past ?Some examples !

  7. Adverse effects of geriatric heart failure therapy applied to the preterm infant with sPDA: marked volume depletionrenal hypoperfusion PG-stimulationnephrocalcinosis arrhythmias with cerebral bleeding intestinal perforation renal failure fluid restriction furosemide digoxin NSAID´s ACE- inhibitor

  8. 12/12 Polyhydramnios 0/13 Affected nephron segment thick ascending limb distal convolute Congenital salt losing tubulopathies (SLTs) Different age at manifestation and ontogeny of targets (Jeck et al., AJ P 2005) Furosemid-SLT : NKCC2 Thiazid-SLT : NCCT <1 year: 1/13 1-5 years: 4/13 6-13 years: 8/13 Age at first presentation antenatal: 12/12 polyuria hyponatremia hypotension (shock) hypercalciuria nephrocalcinosis hypokalemia carpopedal spasms hypomagnesemia hypocalciuria growth retardation Postnatal leading symptoms

  9. Medium analgesic dosage of morphine in children with an age between 0 and 6 years p < 0.01 40 dosis: 0.05 mg/kg/min infusion rate until painlessness 30 Morphine plasma concentration [µg/l] at time point of pain recovery 20 10 0 0 - ½ year n = 5 2 - 4 year n = 5 6 year n = 4 (Olkkola et al., CPT 1988)

  10. Legislative Incentives in Europe?

  11. Objectives of the EU-Regulation on Medicinal Products for Paediatric Use • To improve the health of the children of Europe, by: - increasing high quality research into medicines for them - promoting the development and authorization of such medicines - improving the information on medicines designed for children • While avoiding unnecessary studies in children and not delaying the authorization of medicines for adults

  12. Key measures for patent medicines Requirement at the time of applications for new medicines for: • Data in children as agreed by PCor • A waiver from requirementor • A deferralof the timing of the studies

  13. Key measures for patented medicines Rewards for studies conduced in children: - 6-months extension of the supplementory protectioncertificate (in-effect, a patent extension) - For orphan medicines, 2-years additional market exclusivity(10+2 years)

  14. Key measures for off-patent medicines The Pediatric Use Marketing Authorisation (P.U.M.A.) : • enabeling 10-years data protection - use of existing brand name(brand recognition) - amended data requirements

  15. Institutional or horizontal key measures of the EU-Regulation: • A new expert paediatric committe (PC) at the EMEA, which negotiates with the MAH the padiatric investigation plan (PIP) • Free scientific advice from EMEA • European network of experts • Information tools – inventory of therapeutic needs, new product labelling requirements, database of studies • Public funding for studies into off-patent medicines, e.g. accepted • application in the FP7 • Enhanced safety monitoring for marked products

  16. Specific paediatric needs and priorities in Europe (particular for the neonates): • Analgesics • Sedatives • Immunomodulators, e.g. rheuma and TPL • Antiepileptic agents • Anticongestive and antiarrhythmic agents • Antihypertensive agents • Antiobstructive agents • Cytostatic agents Not included: Paed. anaesthesiology and child psychiatry

  17. EMEA Priority-Listof Off-Patent Medicinal Products for Paediatric Studies in the FP7( http://www.emea.eu.int/pdfs/human/peg/49677706en.pdf )Methodology of Selection: • Severtity of the disease • Paediatric age groups affected (with special regard to the neonatal population) • Non-availability of treatment alternatives • High prevalence of the disease in the paediatric population • High level of evidence available and known or suspected efficacy or safety issues

  18. Some Examples from the EMEA Priority-List of Off-Patent Medicinal Products for Paediatric Studies( http://www.emea.eu.int/pdfs/human/peg/49677706en.pdf )

  19. Child Health in FPVII (2007-2013) 2nd call due June 07 and 3rd call due September 07Overarching topics on Child Health: • Adopting off-patent medicines to specific • needs of paediatric populations • Studies include the assessment of PK-data, efficacy, • safety and/or the development of age appropriate • formulation • Funding scheme: collaborative project with a • maximum of EC contribution of 3 000 000 € • for each (3 year) project ( 30 Mio € are available !)

  20. Members of the European Network Drug Investigation in Children(ENDIC1997 in the ESDP) Elisabeth Autret-Leca, Tours Jean-Paul Langhendries, Liege Maurizio Bonati, Milano Gérard Pons, Paris Imti Choonara, Derby Anders Rane, Stockholm Rafaël Gorodischer, Beer-Sheva Hannsjörg W. Seyberth, Marburg Kalle Hoppu, Helsiniki John N. van den Anker, Rotterdam Evelyne Jacqz-Aigrain, Paris Bart van Overmeire, Antwerp

  21. Thank you for your attention !

  22. Milestones of the EU Paediatric regulation • EC Round Table, EMEA - 18 December 1997 • EU Council resolution - 14 December 2000 • Public consultations - 2002 and 2004 • EU Commission proposal - 29 September 2004 • European Parliament 1. vote - 7. Sept. 2005 • Adoption by EU Council - 9 December 2005 • 2nd vote and adoption by EP - 1. June 2006 • Entry into force - 26. January 2007 EMEA=European Medicines Agency

  23. Anaesthesiology Antiinfectious therapy Cardiology Chemotherapy Diabetes Epilepsy Immunology Migraine Obstructive Lung disease Pain Rheumatology (Gastroenterology) (Child-Psychiatry) Assessment of Paediatric Needs by the Paediatric Expert Group at the EMEA in the last three years( http://www.emea.eu.int/htms/human/peg/pegassessment.htm )

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