1. Medicines for children in Belgium: the way forward with BPCRN Professor José Ramet

2. Studies on medicines in adults not sufficient  Specificity disease  Effects on growth, development, maturation  Specific adverse reaction What the paediatricians know

3. Child... not a small adult Infant... not a small child Preterm...not a small newborn

4. BPCRN “De bijzonderheden van klinische studies voor de pediatrische klinische praktijk”

6. Absorptie Metabolisme Eliminatie Distributie expositie de combinatie van ADME bepaalt de expositie en dus het tijd/conc profiel De expositie in combinatie met de interactie tgv therapeutische target (receptor) bepaalt de conc/effect relatie. farmacokinetiek

7. Drug Exposure Response Absorptie Distributie Receptoren Interactie Biotransformatie Excretie Absorptie Distributie Receptoren Interactie Biotransformatie Excretie omgeving genetica ziekte groei ontwikkeling K. Allegaert

8. Ref: Nelson’s textbook Pediatrics lichaamsproporties

9. Ref: Kearns et al, NEJM 2003

10. Stakeholders Stake holders Academia Paediatric providers Pharma. industry Govt. funded research centres others National Competent Authorities Patients parents orgs. Paediatric societies

11. Paediatricians Societies Research centres Clinical centresPharmaceuticalIndustry Regulatory Authorities Frequent & chronic diseases Reputation Return of investment Medical needs Clinical and/or scientific questions Orphan drugs Partners Balance 3 main stakeholders Ethical concerns

12. Partnership? Industry Role Discovery of new medicines  High throughput screening of compounds Development of new medicines  Pre-clinical toxicology  Human evaluation of dose, safety, efficacy Paediatric Role Define therapeutics needs Develop validated end- points for  efficacy and safety  “bridges” adult studies Develop effective, efficient, ethically driven networks to conduct clinical studies Performed at all level of paediatric care

13. Objectives of BPCRN: Database  existing networks, experience, investigators, local facilities… Reference for  health care professionals & pharmaceutical industry Interaction  all stakeholders  between networks Training & education  new centres & investigators Information  annual meeting stakeholders  printed report of clinical research activities in various fields Role as « facilitator »

14. « facilitator » « facilitator »  recruitment  in various and/or dedicated centres  attainment of required sample sizes  increased performance of trials  through inter-investigators exchange of experience  circulation of information  consolidated effort of all stakeholders to communicate  practitioners in the field : aware of those new opportunities  develop scientific excellence  stimulate gifted medical students: consider careers in paediatric research  support major international research projects  act on education, training, research stimulation

15. BPCRN Belgian PediatricClinical Research Network Réseau Belge de Recherche Clinique Pédiatrique Belgisch Netwerk van Klinische Research in de Pediatrie Stake holders Academia Paediatric providers Pharma. industry Govt. funded research centres others National Competent Authoritie s Patients parents orgs. Paediatric societies

16. Back up

17. Conclusion Stake holders Academia Paediatric providers Pharma. industry Govt. funded research centres others National Competent Authorities Patients parents orgs. Paediatric societies New Substance EMEA Clinical trial project PIP / waiver / deferral Belgian Paediatric Network Proposal of competent trial centres Trial Belgian Paediatric Clinical Research Network « facilitator » Objectives of the network « facilitator »  To do list define interested stakeholders proposal of procedure find support of the network: 2 years first things first: database

18. How to achieve this? 1. Consulting 2. Feasibility: qualified study centres 3. Regulatory: application to authorities and ethics committees (national legal aspects) 4. Study coordination 5. Pharmcovigilance: procedures (reporting to authorities and ethics committees 6. Investigator Recruitment: recruitment procedures 7. Study Assistance 8. Monitoring 9. Data Management: IT-support for trial data base, communication, homepage 10. Biostatistics 11. Tools, Templates & examples of agreements and contracts, specific paediatric SOPs (Standard Operating Procedures) (including templates for patient information, consent and assent), 12. Trial know-how: transfer of knowledge and experience concerning planning and conducting of clinical trials 13. Training courses 14. support services for investigators

19. Network Virtual structure, defined by formal agreement between individuals, organisations or structures sharing and collaborating towards the same objectives, goals and quality standards

20. Challenges What could Paediatric European networks contribution be to paediatric research? be able to respond to increased research needs define “centers” of excellence organize a “research task distribution” in Europe be present at EMEA & related advisory groups From private practice to research centre From clinical to fundamental Major advantage of paediatrics

21. Existing EU paediatric networks networks national Paed. centres structure related age related sub speciality EU funded 7 national networks  public funding  FR, NL, IT, DE,.. EU funded  TEDDY sub-speciality  ESPNIC, ESPN… age-related  neonatal structure-related  Pharmacovigilance networks paediatric centres  dedicated clinical research centres