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The Paediatric Regulation as an instrument for European paediatric research ENPREMA, London 10/11 March 2011 Presented by: Paolo Tomasi, MD PhD Head of Paediatric Medicines, European Medicines Agency An agency of the European Union Why is


  1. The Paediatric Regulation as an instrument for European paediatric research ENPREMA, London 10/11 March 2011 Presented by: Paolo Tomasi, MD PhD Head of Paediatric Medicines, European Medicines Agency An agency of the European Union

  2. Why is there a EU Paediatric Regulation? = for 2 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  3. Objectives of the EU Paediatric Regulation • Improve the health of children: – Increase high quality, ethical research into medicines for children – Increase availability of authorised medicines for children – Increase information on medicines • Achieve the above: – Without unnecessary studies in children – Without delaying authorization for adults 3 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  4. New Drug Development Process: US vs EU EC/EMA PIP (all inclusive) Required for Filing Marketing Approval Preclinical Phase NDA Submission Phase One Phase Three Postmarketing Phase Two PREA*/WR Written Request FDA From Dianne Murphy, FDA

  5. Paediatric Investigation Plan Basis for development and authorisation of a • medicinal product for all paediatric population subsets Includes details of the timing and the • measures proposed, to demonstrate: – Quality Marketing – Safety Authorisation Criteria – Efficacy To be agreed upon and/or amended • by the PDCO Binding on company  compliance check • (but modifications possible, at the company ’ s request) 5 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  6. PDCO Opinions on Paediatric Investigation Plans and Clinical Trials 6 After the PIP Decision: modification of the agreed PIP, validation, compliance check

  7. PDCO opinions on applications 2008-2010 300 250 200 PIP agreed ( ± deferral) 201 150 waiver granted 122 negative 100 81 50 67 52 48 13 7 4 0 2008 2009 2010 7 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  8. PDCO opinions on applications 2007-2010, total negative 24 4% waiver granted 176 29% PIP agreed Deferral: 82% of trials ( ± deferral) (until after adult 406 development) 67% 8 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  9. Therapeutic areas of applications (all applications, 2007 – today) Uro-nephrology Cardiovascular Diseases Anaesthesiology Vaccines 2% 9% 1% 4% Dermatology Psychiatry 4% 2% Diagnostic Endocrinology- 1% Gynaecology-Fertility- Pneumology - Allergology Metabolism 17% 10% Pain Gastroenterology-Hepatology 4% 3% Oto-rhino-laryngology Haematology- 2% Hemostaseology Other 4% 4% Immunology-Rheumatology- Transplantation Ophthalmology 7% 3% Infectious Diseases Neonatology - Paediatric Oncology Nutrition 6% Neurology Intensive Care 0% 10% 5% 1% 9 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  10. Deferrals (Study in the first 96 PDCO procedures) • At least one deferral granted in: –91% of applications for new products –64% of applications for already authorised products • Usual deferral duration: 3-5 years from MAA in adults • Deferral is the instrument to avoid delaying marketing authorisation in adults Olski TM et al., Three Years of Paediatric Regulation in the European Union. Eur J Clin Pharmacol 2011 (in press) 10 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  11. Deferral granted in 91% of new medicinal products and 64% of authorised (2007-2009) Olski TM et al., ib. authorised new 11 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  12. Clinical trials design (Study in first 96 PDCO procedures) • PDCO increased studies in neonates from 15% to 26% (subsequent data showed an increase in proposed neonatal studies) • Staggered approach proposed, encouraged and occasionally imposed • N. of patients required: PDCO requested an  Increase in 5/54 (9.3%)  Decrease in 8/54 (14.8%) Olski TM et al., ib. 12 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  13. Clinical trials design (study in the first 96 PDCO procedures) • Primary endpoint accepted in 90% of cases • General design endorsed in almost all cases (97%):  2/96: double-blinding imposed  23 additional trials imposed (20 comparative, 6 active-controlled, 12 placebo-controlled, 2 dose- comparison) Olski TM et al., ib. 13 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  14. Clinical trials in Europe (data from EudraCT) 14 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  15. Clinical trials involving children (<18) 2010, EEA including <18 years, 949, 10% adults only, 8252, 90% 15 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  16. Clinical trials in children (2010, EEA) paediatric only vs. paed. + adult 949 trials including children (<18) <18 and adults, 413, 44% only <18, 536, 56% 16 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  17. Paediatric clinical trials by age groups (2010, EEA) Note: Multinational trials are counted in each country 1100 1000 949 900 753 800 700 586 600 500 400 261 300 200 78 100 37 6 0 total <18 in utero preterm newborns infants and children adolescents newborn toddlers infants 17 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  18. Is the number of paediatric trials increasing in the EEA? 1200 1000 800 600 400 200 0 2005 2006 2007 2008 2009 2010 18 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  19. The % of paediatric trials is substantially stable (appr. 9-10%, EEA) 100% 90% 80% 70% 60% adult only 50% 40% 30% 20% 10% paed 0% 2005 2006 2007 2008 2009 2010 19 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  20. Are clinical trials moving outside the “first world”? 20 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  21. A substantial proportion of patients in adult CTs is from outside the EU 21 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  22. Are clinical trials moving outside the “first world”? 22 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  23. The role of ENPREMA Key operational goals • Collaboration and communication (between existing networks and stakeholders) • Facilitation of recruitment in clinical trials (providing expertise and access to infrastructure for industry to conduct studies in children) • Building competences (defining strategies for resolving major challenges) • Avoiding unnecessary studies • Stimulating high quality research (consistent and transparent quality standards; harmonising clinical trial procedures) • Strengthening the foundations of the European Research Area

  24. What ENPREMA is NOT supposed to do • To fund studies • To conduct studies • to decide on areas of paediatric research, which is under the responsibility of: – the Member States – the Commission through the Community programmes – each individual network

  25. Paediatric Regulation and Clinical Trials in Children - Conclusion • Paediatric development has gone from very very optional to compulsory in principle (unless a waiver is granted) • Paediatric Investigation Plans, agreed between EMA and pharmaceutical companies, require a substantial increase in n. of clinical trials in paediatric age groups • Unless capacity is sufficient in Europe, companies will need to find alternatives 25 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

  26. Thanks for listening PDCO meeting at EMA 26 The Paediatric Regulation as an instrument for paediatric clinical research in Europe - P Tomasi

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