EMA public workshop on extrapolation of efficacy and safety in medicine development

• Date:
  Mardi, 17 mai 2016, Journée Entière
• Lieu:
  European Medicines Agency, Amsterdam, the Netherlands

In 2013, the European Medicines Agency (EMA) published a concept paper on extrapolation of efficacy and safety in medicine development. It aims to discuss the need and possibility of developing a framework for extrapolation approaches that are considered scientifically valid and reliable to support medicine authorisation. The framework will set out a structured approach for each extrapolation exercise, in order to improve interactions with stakeholders and to standardise decision making across committees. Further to the extrapolation expert meeting that took place in September 2015, EMA will publish a reflection paper on extrapolation across age groups that will include considerations specific to global product development and licensing of paediatric medicines. This workshop will present the reflection paper and seek feedback from developers.

Documents

EMA public workshop on extrapolation of efficacy and safety in medicine development - Outcome of a multi-stakeholder meeting with experts and regulators

Reference Number: EMA/478467/2016

Agenda - Workshop on extrapolation of efficacy and safety in medicine development across age groups

Reference Number: EMA/12429/2016

Registration request for the workshop on extrapolation of efficacy and safety in medicine development across age groups

Presentation - Quantitative challenges of extrapolation

Presentation - EMA extrapolation framework (Christoph Male)

Presentation - When do clinicians usually extrapolate in their practice? (Daniel Brasseur, Christoph Male)

Presentation - Paediatric medicines development - The need for different approaches (Sam Maldonado)

Presentation - Extrapolation: which is the patient perspective? (Marco Greco)

Presentation - A modelling and simulation perspective on extrapolation (Ine Skottheim Rusten)

Presentation - (One) Statistician(’)s perspectives on extrapolation (Rob Hemmings)

Presentation - Methodology for extrapolation in the EU FP7 ASTERIX, IDeAl and InSPiRe projects (Nigel Stallard, Ralf-Dieter Hilgers, Kit Roes)

Presentation - POS (Partial onset seizures) Extrapolation from adults to children Clinical setting (Catherine Chiron, Gerard Pons)

Presentation - Case study Brivact UCB

Presentation - Bayesian prior elicitation: an application to the MYPAN trial in childhood polyarteritis nodosa (Lisa Hampson, John Whitehead, Despina Eleftheriou, Paul Brogan)

Presentation - Model Informed Drug Discovery and Development (MID3) Good Practice: use of prior knowledge and setting up assumptions (Scott Marshall, Lutz Harnisch)

Presentation - Extrapolation of dosing, efficacy and safety of biologics in JIA, IBD and psoriasis - EMA history (Richard Veselà½)

Presentation - Extrapolation & Pediatric Development: A case study from pediatric Ulcerative Colitis (Richard Strauss)

Presentation - Extrapolation in paediatric juvenile idiopathic arthritis: case study (Ruth Oliver, Laura Shaughnessy, Philippa Charlton)

Presentation - Design of PK/PD Studies (Mats Karlsson)

Presentation - Session 3: Extrapolation plan and PK/PD studies Panel Discussion (Martin Posch)

Presentation - Supporting a Pediatric Investigational Plan for Everolimus - Defining the extrapolation plan (Thomas Dumortier, Mick Looby)

Presentation - On the road to clinical extrapolation (Kristina Weber, Armin Koch)

Presentation - Paediatric Pulmonary arterial hypertension - Regulatory Perspective (Amany El-gazayerly)

Presentation - Extrapolation challenges in pediatric PAH - Possible solutions for a feasible, global study (M. Bacchi, A. Morganti, P. Cornelisse, J. Bolognese, C. Lesage, A. Kà¼mmel, P. Nilsson)

Presentation - Extrapolation plan and statistical tools - Panel discussion (Flora Musuamba, Alexander Staab)

Presentation - Validating the extrapolation concept – methodological considerations (Andrew Thomson)

Presentation - Confirmation of extrapolation based on PK/PD data and modeling (Jacob Brogren)

Presentation - General framework on managing uncertainties (Kevin Blake)

Presentation - Decision making, revisited

Presentation - Regulatory interactions: Expectations on extrapolation approaches (Lynne Yao)

Presentation - Considerations for Implementing the EMA Extrapolation Approach: A Global Drug Developer’s Perspective (Christina Bucci-Rechtweg)

Presentation - EMA Extrapolation Framework Regulatory tools (Paolo Tomasi)

Multimedia