Transcript BVK
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 Child... not a small adult Infant... not a small child Preterm...not a small newborn BPCRN “De bijzonderheden van klinische studies voor de pediatrische klinische praktijk” farmacokinetiek de combinatie van ADME bepaalt de expositie en dus het tijd/conc profiel Absorptie Distributie expositie De expositie in combinatie met de interactie tgv therapeutische target (receptor) bepaalt de conc/effect relatie. Eliminatie Metabolisme ziekte groei ontwikkeling genetica omgeving Drug K. Allegaert Absorptie Distributie Receptoren Interactie Biotransformatie Excretie Exposure Response lichaamsproporties Ref: Nelson’s textbook Pediatrics Ref: Kearns et al, NEJM 2003 Stakeholders Academia Paediatric societies Patients parents orgs. National Competent Authorities Paediatric providers Stake holders others Pharma. industry Govt. funded research centres Partners Balance 3 main stakeholders Ethical concerns Pharmaceutical Industry • Frequent & chronic diseases • Reputation • Return of investment Regulatory Authorities Paediatricians • Societies • Research centres • Clinical centres • Medical needs • Clinical and/or scientific questions • Orphan drugs Partnership? Paediatric Role Define therapeutics needs Develop validated endpoints for efficacy and safety “bridges” adult studies Develop effective, efficient, ethically driven networks to conduct clinical studies Performed at all level of paediatric care Industry Role Discovery of new medicines High throughput screening of compounds Development of new medicines Pre-clinical toxicology Human evaluation of dose, safety, efficacy Objectives of BPCRN: Database existing networks, experience, Reference investigators, local facilities… for health care professionals & pharmaceutical industry Interaction all stakeholders Role as between networks Training & education « facilitator » new centres & investigators Information annual meeting stakeholders printed report of clinical research activities in various fields « 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 BPCRN Belgian PediatricClinical Research Network Réseau Belge de Recherche Clinique Pédiatrique Belgisch Netwerk van Klinische Research in de Pediatrie Academia Paediatric societies Patients parents orgs. National Competent Authorities Paediatric providers Stake holders others Pharma. industry Govt. funded research centres Back up Conclusion New Substance Clinical trial project EMEA Academia Paediatric societies Patients parents orgs. Pharma. industry Stake holders Belgian Paediatric Belgian Paediatric ClinicalNetwork Research Network Proposal of competent trial centres Trial Govt. funded research centres National Competent Authorities others PIP / waiver / deferral Paediatric providers 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 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 Network Virtual structure , defined by formal agreement between individuals , organisations or structures sharing and collaborating towards the same objectives , goals and quality standards Challenges What could Paediatric European networks contribution be to paediatric research? be able to respond to increased research needs From private define practice to “centers” of excellence research centre organize a “research task distribution” in Europe be present at EMEA & related advisory groupsFrom clinical to fundamental Major advantage of paediatrics Existing EU paediatric networks 7 national networks public funding FR, NL, IT, DE ,.. national EU funded Paed. centres age related funded TEDDY sub-speciality ESPNIC, ESPN… networks sub speciality EU age-related structure related neonatal structure-related Pharmacovigilance networks paediatric centres dedicated clinical research centres