NIHR Medicines for Children Research Network (MCRN)

1. NIHR Medicines for Children Research Network (MCRN) Prof Ian C K Wong Chair, MCRN Pharmacy & Pharmacology CSG Associate Director, London LRN NPPG conference 2010 “A cheerful heart is good medicine, but a crushed spirit dries up the bones. (Proverbs 17:22)”.

2. Contents • Background of the Medicines for Children Research Network (MCRN). • MCRN Local Research Networks (LRNs). • MCRN Pharmacy and Pharmacology Clinical Study Group (CSG) – Functions – What can the CSG for you – What can you do for the CSG

3. Medicines for Children • Historically, there have been limited numbers of paediatric clinical trials conducted. • High proportion of off-label/unlicensed medicine use. • Potentially leads to over/under dosing. • Increased chance of adverse events. • Requirement for: – More clinical trials in children – Better age-appropriate formulations – More authorised medicines and treatments

4. EU Regulation “Better medicines for children” • 26 January 2007 • Paediatric Investigation Plan (PIP) for all new MA’s and variations – Agreement with Paediatric Committee – Waivers and deferrals – 6 month patent extension – Information included in SmPC • PUMA (data protection) – for off-patent developments • Research on older drugs (7thFramework funding) • European Paediatric Research Network (EnprEMA) More research on paediatric medicines

5. MCRN - Vision To improve children’s health and alleviate suffering through the provision of better and safer medicines.

6. MCRN - Aims • To provide the best infrastructure in the world to support studies which address the safety and efficacy of medicines for children. • To provide an excellent base for clinical research for children, which is an attractive resource for the global pharmaceutical industry. • To ensure that world-leading research addresses the needs of children.

7. MCRN – Adding Value to Study Delivery • Large network supporting randomized and other studies of medicines for children in UK NHS sites serving approx 12 million children • Supporting: – All phases (I-IV) – All therapy areas (except oncology) • Studies supported (adopted) by MCRN – Publicly-sponsored – Industry-sponsored – Investigator-initiated (industry funded) Research (IIR)

8. NIHR Clinical Research Networks

9. Local Research Networks (LRNs) • Local Research Networks (LRNs) ensure that clinical trials involving children are performed efficiently to the highest standards across NHS sites. • In total, there are 6 LRNs that cover over 100 NHS sites. • LRNs assist those wanting to conduct studies by improving set-up time and reducing administrative burdens. • The Networks helps with detailed feasibility assessments.

10. Local Research Networks (LRNs) LRN staff support the conduct of paediatric research in the NHS and work to Good Clinical Practice (GCP) standards • Directors • Manager • Research Nurses • Pharmacists • Data entry staff • Administration

11. Clinical Study Groups (CSG) MCRN CSGs are panels of stakeholders relevant to medicines for children who have the general remit to: - Provide expert advice to investigators, the pharmaceutical industry and to the MCRN Study Adoption Committee (SAC). - Identify gaps within current research and develop studies to successfully address these gaps.

12. Clinical Studies Groups (CSGs) Allergy, Infection and Immunity Anaesthesia, Pain, Intensive Care and Cardiology Diabetes, Endocrinology and Metabolic Medicine Gastroenterology, Hepatology and Nutrition General Paediatrics (including Dermatology) Inherited Metabolic Disorders (MCRN/UK LSD) Methodology Neonatal (MCRN/Action Medical Research) Nephrology (MCRN/BAPN) Neurosciences Pharmacy and Pharmacology Respiratory and Cystic Fibrosis Rheumatology (MCRN/Arthritis Research UK) Dr Mike Sharland Dr Robert Tasker Prof David Dunger Dr Nick Croft Dr Colin Powell Dr Chris Hendriksz Prof Peter Brocklehurst Dr Mark Turner Dr Moin Saleem Dr Timothy Martland Prof Ian Wong Dr Paul Seddon Dr Michael Beresford

13. Pharmacy and Pharmacology CSG • The focus of the Pharmacy and Pharmacology CSG has been to establish formulation, pharmacokinetics and pharmacogenomics research priorities in relevant paediatric fields and to promote the translation of quality pharmaceutical and pharmacological research into well designed clinical studies in children.

14. Chairs • The Group is Chaired by Professor Ian Wong. • Dr Catherine Tuleu is the Chair of the formulation subgroup. • Professor James McElnay Chairs the pharmacokinetic subgroup.

15. Formulation subgroup • Dr Catherine Tuleu (Chair). • Ms Jacqueline Childs (Consumer Representative). • Ms Kate Gandhi (Paediatric Pharmacist). • Professor Richard Guy (Academic Formulation Scientist). • Dr Olufemi Rabiu (Hospital Formulation Scientist). MCRN members: • Dr Mine Orlu Gul (MCRN Formulations Research Fellow). • Dr Utpal Shah (MCRN Formulations Research Fellow). • Professor Tony Nunn (MCRN Associate Director).

16. Pharmacokinetics • Professor James McElnay (Chair). • Ms Jacqueline Childs (Consumer Representative). • Professor Atholl Johnston (Academic Pharmacokineticist). • Professor Tony Nunn (MCRN Associate Director). • One Specialist Member (to be appointed).

17. Role of MCRN CSGs Reactive Role Proactive Role To provide advice to investigators for the development of research proposals and applications. To research within specialty areas and determine priorities. identify gaps in current funding research To provide advice to commercial companies through the MCRN Industry Liaison Team. To develop clinical trials or other well designed studies to address research gaps. To Adoption Committee the quality of studies submitted to run through the network. advise the MCRN Study To decisions of the MCRN through representation Board. participate in strategic regarding on the MCRN

18. Reactive Role “Provision of advice to investigators and Commercial Companies” **October 2008**

19. Proactive Role (1) “Identifying gaps in research and working to address them” IDENTIFICATION OF RESEARCH PRIORITIES Development formulation and pharmacokinetics – April 2007. ERANET Project (A EU project to identify paediatric research priorities) – January 2008. of research priorities in

20. Proactive Role (2) DEVELOPMENT OF RESEARCH PROPOSALS Global €10million funding application to the European Commission FP7 Research Programme. The formulation workpackage comprises Prof Tony Nunn, Dr Catherine Tuleu and Prof Ian Wong from the Pharmacy & Pharmacology CSG. €500,000 was successfully obtained for the formulation workpackage. Research In Paedaitrics (GRIP)

21. Proactive Role (3) OTHER KEY ACTIVITIES/ACHIEVEMENTS Pharmacy and Pharmaceutical Conference (April 2ndto Presentations are http://www.mcrn.org.uk/ Methodology + Pharmacology CSG joint conference in Non- Adherence in children (May 11th2010). Pharmacology Translational CSG Research – April 3rd2009). available from: the Pharmacy and

22. Balanced Portfolio Adopted studies (n=198; as of July 2010)

23. What can we do for you? • If you have a research idea. • Contact us – We can help you to develop your research idea. – Link you up with the appropriate experts or other CSGs. – Assist you in developing a proposal. – Advise you on how to apply for funding. – Peer-review your proposal prior to your funding application

24. What you can do for us? • We need new members. Please join us. • You can give us paediatric pharmacy practice research ideas. • Let your colleagues know about our work. • We are open to suggestions.

25. Professional Group Liaison • All other CSGs are linked up with the RCPCH and/or their professional/consumer group. • The Pharmacy and Pharmacology CSG is not currently linked with any professional group. • Should the Pharmacy and Pharmacology CSG link up with the Neonatal and Paediatric Pharmacists Group?

26. Questions and Discussion?

27. To discuss support that the MCRN can provide please contact: Prof Ian Wong Chair of the Pharmacy and Pharmacology CSG ian.wong@.pharmacy.ac.uk Laura Pilkington Clinical Studies Groups Administrator laura.pilkington@liverpool.ac.uk