BVK

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Medicines for children in Belgium the way
forward with BPCRN
Professor José Ramet
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• 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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Child... not a small adult Infant... not a
small child Preterm...not a small newborn
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BPCRN

• De bijzonderheden van klinische studies voor de
  pediatrische klinische praktijk

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(No Transcript)
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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.

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ziekte groei ontwikkeling
Absorptie Distributie Receptoren
Interactie Biotransformatie Excretie
Exposure Response
Drug
K. Allegaert
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lichaamsproporties
Ref Nelsons textbook Pediatrics
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Ref Kearns et al, NEJM 2003
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Stakeholders
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• Partners
• Balance 3 main stakeholders
• Ethical concerns

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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

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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 
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 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

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BPCRN

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

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• Back up

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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

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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

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Network

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

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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
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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