NIHR Medicines for Children Research Network (MCRN)

1. NIHR Medicines for Children Research Network (MCRN) Prof Ian Wong Chair, MCRN Pharmacy & Pharmacology Clinical Study Group Associate Director of London Local Research Network

2. Medicines for Children • Historically, 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

3. 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 (7th Framework funding) • European Paediatric Research Network (EnprEMA) More research on paediatric medicines

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

5. 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

6. 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)

7. NIHR Clinical Research Networks

8. Clinical Studies Groups (CSGs) • Key opinion leader advisory groups • Proactive Roles: • To identify research priorities within specialty areas • To propose and develop trial ideas and proposals • Representatives on MCRN Board provide strategic direction • Reactive Roles: • To work with investigators to develop study ideas to successful funding application • To provide subject specific and methodological advice to industry and other investigators (protocols, PIPs etc) • Representatives sit on MCRN Study Assessment Committee (SAC)

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

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

11. Growth of Study Portfolio

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

13. Feasibility Feasibility request <5 working days Level 1 Feasibility (Top level review by CSGs) Level 2 Feasibility (Investigator identification by LRNs) 10 working days Data sent to company • Additional company feasibility • (Investigators/LRNs) • Company specific CDAs • Full feasibility questionnaires

14. Clinical Research Facilities (CRFs) • Children’s CRFs facilitate the conduct of very complex/invasive studies, which might be impossible to perform elsewhere • Specialist Children’s Research Nurses and other staff • Advanced equipment, lab and other facilities • Purpose built for children • Close collaboration between MCRN and UK CRFs

15. Site Setup • LRNs support: • Financing (NIHR CRN Costing template) • Local R&D/ethics approvals • Contracting (mCTA) • Staff contracting and recruitment • Training • All company employees working on adopted studies can attend free NIHR CRN/MCRN training courses • MCRN support helped sites setup an average of ~2 months earlier than other sites

16. Recruitment to target • LRNs support recruitment - strategy, staff support, new site identification • Recruitment continually monitored by MCRN Industry Team, LRNs and Executive • If site not recruiting as expected, investigator/site staff contacted to resolve issues

17. Industry Successes “Congratulations on your first screened subject!!! This is excellent news and I can confirm that this is the first screened subject globally.” Charlotte Vigor PhD Study Manager, Clinical Operations Takeda Global Research and Development (Europe) Ltd

18. Industry Portfolio Adopted industry studies (n=98; as of September 2010)

19. 98 Industry Studies Adopted

20. Example Industry Study (Vaccine)

21. Industry Portfolio Number of studies Adopted Number of Children Recruited

22. Conclusions • MCRN is committed to meeting recruitment targets for the both academic and pharmaceutical industry • The Network provides support during all study stages to ensure that targets will be met • Excellent recruitment to studies • Successful even in rare paediatric conditions • Positive feedback from companies

23. To discuss support that MCRN / Pharmacy and Pharmacology CSG can provide please contact: Prof Ian Wong ian.wong@dpp.pharmacy.ac.uk