Workshop on Extrapolation and Evidence Synthesis in the Development and Therapeutic Use of Medicines in Children - 11th June 2013 – Strathclyde University, Glasgow, UK

*Dear All,* * * *On behalf of GRiP – Global Research in Paediatrics, we are* pleased to announce the *one-day Workshop* on *Extrapolation and Evidence Synthesis in the Development and Therapeutic Use of Medicines in Children*, which will be held the University of Strathclyde, Glasgow on *Tuesday, 11th June, from 09:00 - 17:15 h.* * **Among the various activities in the scope of the GRiP network focus is given to *the requirements for the use of novel trial designs, extrapolation as well as modelling and simulation. The primary objective of this workshop is therefore to review the current methodologies for evidence generation and evidence synthesis in paediatric research, including opportunities for extrapolation and optimisation of experimental protocols. Attention will also be given to issues such as the choice of comparators when defining efficacy and safety in children. These topics have become increasingly important with the recent changes in the legislation, which acknowledges the need for appropriate assessment of the efficacy and safety of paediatric medicines prior to regulatory approval and therapeutic use. See the programme below for further details. The workshop will be held as a satellite meeting of PAGE. PAGE participants are welcome to join the meeting and contribute to the discussion. A reflection document will be prepared based on the points raised during the workshop and submitted as a report to regulators and other relevant stakeholders. The *workshop is free of charge*, but *registration is required due to venue capacity (max 85)*. Please contact Sven van Dijkman ( [email protected]) providing *your name, email, organisation, date of arrival in Glasgow and a short description of your motivation (e.g., specific interest or experience in paediatric drug development)*. There are 30 places available. Registration will be on a first-come first-served basis. Best regards, Sven van Dijkman On behalf of the GRiP Organising Committee -------------------------------------------------------------------------------------------------------------------------------------------------------- *Workshop on Extrapolation and Evidence Synthesis in the Development and Therapeutic Use of Medicines in Children* * 11th June 2013 – Strathclyde University, Glasgow, UK* PRELIMINARY PROGRAMME 09:00 - Welcome and objectives of the workshop 09.10 – Introduction to the GRiP Network of Excellence Dr Paola Baiardi (CVBF, Pavia, Italy; GRiP- WP4 Leader) 09.30 – GRiP perspective on evidence generation and synthesis Dr Oscar Della Pasqua (University of Leiden, The Netherlands) * * *SESSION 1 - EVIDENCE GENERATION* 09.45 - Use of nonlinear mixed effects modelling and simulation for data analysis and study design optimisation. Dr Ann Vermeulen (J&J, Beerse, Belgium) 10.15 - Incorporation of historical data as priors in hierarchical models for the evaluation of treatment effect Dr Alberto Russu (J&J, Beerse, Belgium) 10:45 - Panel Discussion - Q&A Discussant: Prof Gérard Pons (University Paris Descartes, France. Paediatric Committee, EMA, UK) (to be confirmed) 11:15 - Coffee break *SESSION 2 - EVIDENCE SYNTHESIS* 11.35 - Role of meta-analysis in evidence synthesis: an example based on dose selection for antibiotics Dr Charlotte Barker (St George's Hospital, London, UK) 12.05 - Defining the level of evidence in the evaluation of risk Dr Yoon Loke (University of East Anglia, UK) 12.35 - Panel Discussion - Q&A Discussant: Dr Katja Verhamme (Erasmus Medical Centre, Netherlands) (to be confirmed) 13:05 – Lunch *SESSION 3 - FOCUS ISSUES - RARE DISEASES AND NEONATOLOGY* 14.05 - Requirements for the evaluation of rare diseases Elin Haf Davies (European Medicine Agency, UK) 14.35 - Clinical trials and evidence generation in neonates Hitesh Pandya, University of Leicester, UK 15.05 - Panel Discussion - Q&A Discussant: Prof Evelyne Jacqz-Aigrain (Hôpital Robert Debré, Paris) 15.35 - Tea break *SESSION 4 - REGULATORY and PATIENT PERSPECTIVES* 15.50 - The Paediatric Investigational Plan and the Pediatric Study Plan Dr Christoph Male, (European Medicine Agency, UK) 16.20 - Patient/parents insight on novel methodologies and regulatory requirements Representative TBC 16.45 - Panel Discussion - Q&A Discussant: GRiP representative (to be confirmed) 17.15 – Closure Oscar Della Pasqua (University of Leiden, GRiP W4)