Medicines for children in Belgium:the way forward with BPCRNProfessor Jos Ramet
Studies on medicines in adults not sufficientSpecificity diseaseEffects on growth, development, maturationSpecific adverse reactionWhat the paediatricians know
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
de combinatie van ADMEbepaalt de expositie en dus hettijd/conc profiel
De expositie in combinatie metde interactie tgv therapeutischetarget (receptor) bepaalt deconc/effect relatie.
farmacokinetiek
DrugExposureResponseAbsorptieDistributieReceptoren InteractieBiotransformatieExcretieziektegroei ontwikkelingK. Allegaert
Ref: Nelsons textbook Pediatricslichaamsproporties
Ref: Kearns et al, NEJM 2003
Stakeholders
PartnersBalance 3 main stakeholdersEthical concerns
Partnership?Industry Role
Discovery of new medicinesHigh throughput screening of compoundsDevelopment of new medicinesPre-clinical toxicologyHuman evaluation of dose, safety, efficacyPaediatric Role
Define therapeutics needsDevelop validated end-points for efficacy and safetybridges adult studiesDevelop effective, efficient, ethically driven networks to conduct clinical studiesPerformed at all level of paediatric care
Objectives of BPCRN: Databaseexisting networks, experience, investigators, local facilitiesReference for health care professionals & pharmaceutical industry Interactionall stakeholdersbetween networks Training & educationnew centres & investigators Informationannual meeting stakeholdersprinted report of clinical research activities in various fields Role as 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 researchsupport major international research projects act on education , training , research stimulation
BPCRN
Belgian PediatricClinical Research Network
Rseau Belge de Recherche Clinique PdiatriqueBelgisch Netwerk van Klinische Research in de Pediatrie
Back up
ConclusionBelgian Paediatric Clinical Research NetworkObjectives 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. Consulting2. Feasibility: qualified study centres3. Regulatory: application to authoritiesand ethics committees (national legalaspects)4. Study coordination5. Pharmcovigilance: procedures (reportingto authorities and ethics committees6. Investigator Recruitment: recruitmentprocedures7. Study Assistance8. Monitoring9. Data Management: IT-support for trial database, communication, homepage10. Biostatistics11. Tools, Templates & examples of agreementsand contracts, specific paediatric SOPs(Standard Operating Procedures)(including templates for patient information,consent and assent), 12. Trial know-how: transfer of knowledgeand experience concerning planning andconducting of clinical trials13. Training courses14. 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 needsdefinecenters of excellence organize a research task distribution in Europebe present at EMEA & related advisory groupsFrom private practice to research centre
From clinical to fundamental
Major advantage ofpaediatrics
Existing EU paediatric networks
7 national networks public funding FR, NL, IT, DE ,..EU funded TEDDY sub-speciality ESPNIC, ESPN age-related neonatalstructure-related Pharmacovigilance networkspaediatric centres dedicated clinical research centres
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