medicines for children in belgium: the way forward with bpcrn professor josé ramet
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Medicines for children in Belgium:the way forward with BPCRN
Professor José Ramet
Studies on medicines in adults not sufficient Specificity disease Effects on growth, development,
maturation Specific adverse reaction
What 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”
AbsorptieAbsorptie
Metabolisme
Metabolisme
EliminatieEliminatie
DistributieDistributie
expositieexpositie
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
DrugExposure
Response
AbsorptieDistributieReceptoren InteractieBiotransformatieExcretie
AbsorptieDistributieReceptoren InteractieBiotransformatieExcretie
omgeving genetica
ziektegroei ontwikkeling
K. Allegaert
Ref: Nelson’s textbook Pediatrics
lichaamsproporties
Ref: Kearns et al, NEJM 2003
Stakeholders
Paediatric societies
Patients parents
orgs.
National Competent Authorities
others
Govt.funded
research centres
Pharma. industry
Paediatric providers
Academia
Stakeholders
PaediatriciansPaediatricians• Societies • Research centres• Clinical centres
PharmaceuticalPharmaceutical
IndustryIndustry
RegulatoryAuthorities
• Frequent & chronic diseases
• Reputation • Return of
investment
• Medical needs• Clinical and/or
scientific questions• Orphan drugs
Partners Balance 3 main
stakeholders Ethical concerns
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
Objectives of BPCRN:
Databaseexisting networks, experience, investigators, local facilities…
Reference 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 »
« « facilitatorfacilitator » »
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
Réseau Belge de Recherche Clinique Pédiatrique Belgisch Netwerk van Klinische Research in de
Pediatrie Paediatric societies
Patients parents
orgs.
National Competent Authorities
others
Govt.funded
research centres
Pharma. industry
Paediatric providers
Academia
Stakeholders
Back up
Conclusion
Paediatric societies
Patients parents
orgs.
National Competent Authorities
others
Govt.funded
research centres
Pharma. industry
Paediatric providers
Academia
Stakeholders
New Substance
EMEA
Clinical trial project
PIP / waiver / deferral
Belgian Paediatric NetworkProposal of competent trial centres
Trial
Belgian Paediatric
Clinical Research Network
Objectives of the network
« facilitator » « facilitator »
To do listdefine interested stakeholdersproposal of procedurefind support of the network: 2 yearsfirst 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 betweenindividuals , organisations or structures sharing and collaborating towards the same objectives , goals and qualitystandards
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 ofpaediatrics
Existing EU paediatric networks
EU funded
subspeciality
agerelated
structurerelated
Paed. centres
national
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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