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Medicines for children in Belgium: the way forward with BPCRN

Medicines for children in Belgium: the way forward with BPCRN. Professor José Ramet. What the paediatricians know. Studies on medicines in adults not sufficient Specificity disease Effects on growth, development, maturation Specific adverse reaction.

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Medicines for children in Belgium: the way forward with BPCRN

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

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

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

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

  5. Absorptie Distributie expositie Eliminatie Metabolisme farmacokinetiek 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.

  6. genetica omgeving ziekte groei ontwikkeling Absorptie Distributie ReceptorenInteractie Biotransformatie Excretie Exposure Response Drug K. Allegaert

  7. lichaamsproporties Ref: Nelson’s textbook Pediatrics

  8. Ref: Kearns et al, NEJM 2003

  9. Stakeholders

  10. Paediatricians • Societies • Research centres • Clinical centres Regulatory Authorities Pharmaceutical Industry • Medical needs • Clinical and/orscientific questions • Orphan drugs • Frequent & chronic diseases • Reputation • Return of investment • Partners • Balance 3 main stakeholders • Ethical concerns

  11. 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 Partnership?

  12. 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 »

  13. « 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

  14. Belgian PediatricClinical Research Network Réseau Belge de Recherche Clinique Pédiatrique Belgisch Netwerk van Klinische Research in de Pediatrie BPCRN

  15. Back up

  16. New Substance Clinical trial project EMEA PIP / waiver / deferral Belgian Paediatric Network Proposal of competent trial centres Trial Conclusion Belgian Paediatric Clinical Research Network Objectives of the network « facilitator » • To do listdefine interested stakeholdersproposal of procedurefind support of the network: 2 yearsfirst things first: database

  17. 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

  18. Network Virtual structure , defined by formal agreement betweenindividuals , organisations or structures sharing and collaborating towards the same objectives , goals and qualitystandards

  19. 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

  20. Existing EU paediatric networks • 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

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