1. Medicines for Children Research Network Paediatric Clinical Trials: Principles, Practicality and the Medicines for Children Research Network Andrew Collinson Co-Director MCRN South West

2. Medicines for Children Research Network Principles – Scientific evidence RCTs: –Why “randomised” (minimises confounding) –Why “controlled” (comparison group) –Why “blind” (minimises recall/ observer bias) –Applicability and validity –Power

3. Medicines for Children Research Network Principles – Children are different Diseases Physiology Behaviour Pharmacokinetics Pharmacodynamics

4. Medicines for Children Research Network Principles - Ethics A brief history of disaster: –Nuremberg 1946 –Thalidomide 1977 –Tuskegee Syphilis Study And then there was… –The Declaration of Helsinki

5. Medicines for Children Research Network Practicality – Legislation and Standards International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) ICH Good Clinical Practice (GCP) The EU Clinical Trials Directive ‘Better Medicines for Children’: European Parliament and Council Regulation

6. Medicines for Children Research Network International Conference on Harmonisation (ICH) Addressed: –Need for new medicines in global market –Differing national requirements for approval –Need to reduce import delays –Efficient use of human, animal and material resource –Need to reduce repeat testing

7. Medicines for Children Research Network ICH Good Clinical Practice ICH Standards: –What do you think they should be?…

8. Medicines for Children Research Network ‘Better Medicines for Children’: European Parliament and Council Regulation

9. Medicines for Children Research Network Practicality: Research Infrastructure

10. Medicines for Children Research Network

11. UK Clinical Research Network Diabetes Dementias Stroke Diabetes Dementias Stroke

12. Medicines for Children Research Network Director Manager Research nurse Data entry staff Administrator Service support UKCRN MCRNCC Portfolio Development LRNx6CTU Clinical Studies groups Regulators Clinical experts Industry Consumers Funders

13. Medicines for Children Research Network MCRN Local Research Networks (LRNs)

14. Medicines for Children Research Network What are we trying to achieve? “To facilitate the conduct of randomised trials and other well-designed studies of medicines for children, including those for prevention, diagnosis and treatment”

15. Medicines for Children Research Network Clinical Studies Groups PROACTIVE ROLES: To identify research priorities To propose and develop trial proposals Representatives sit on MCRN Operational Group REACTIVE ROLES: To help investigators to develop ideas to successful funding application To provide subject specific and methodological advice to investigators Representatives sit on MCRN Trial Adoption Committee

16. Medicines for Children Research Network Clinical Studies Groups Anaesthesia, Intensive Care, Pain Control TBC and Cardiology Diabetes, Endocrinology & Metabolic Medicine Prof David Dunger Gastroenterology, Hepatology & Nutrition Dr Stephen Murphy General Paediatrics (including Dermatology)Dr Colin Powell Methodology Dr Peter Brocklehurst Neonatal Prof David Field Neurosciences Dr William Whitehouse Pharmacy & PharmacologyProf Ian Wong RANII (Rheumatology, Allergy, Dr Mike Sharland Nephrology, Infection & Immunity) Respiratory & Cystic FibrosisProf Jonathan Grigg

17. Medicines for Children Research Network MCRN and opportunities for Industry Access to opinion leaders in paediatric medicine Standing confidentiality agreements Swift assessment of site feasibility Consistent approach to costing studies & arranging contracts Central sign off for R&D approval at national level

18. Medicines for Children Research Network

19. MCRN Consumer Involvement open and transparent central to all aspects of MCRN activities all stages and levels of research cycle all key National and Local MCRN committees reflect and contribute to good practice guidelines on involving consumers, particularly children and families, in research impact of MCRN consumer involvement monitored and evaluated findings and outputs on consumer involvement strategy should be widely disseminated.

20. Medicines for Children Research Network MCRN LRNs Clinical Studies Groups Clinical Trials Units MCRN Portfolio Trial Adoption Committee MCRN Coordinating Centre Dr Vanessa Poustie Assistant Director Miss Jennifer Blakeburn CSG Administrator Portfolio Development

21. Medicines for Children Research Network Current portfolio

22. Medicines for Children Research Network