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