Seventh Framework Programme (FP7) small-population research methods projects and regulatory application workshop

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TitleSeventh Framework Programme (FP7) small-population research methods projects and regulatory application workshop
Date29/03/2017 - 30/03/2017
LocationEuropean Medicines Agency, London, UK
SummaryThe European Union funds three projects, Asterix, IDeAl and InSPiRe, within the Seventh Framework Programme (FP7) to develop new methodology on design and analysis of small population clinical trials since 2013. The projects are in their final phase. The workshop will discuss their results to date. The aim is to translate and promote results and novel methodologies into tangible recommendations to advance the clinical research and development of medicines and new treatments for patients with a rare disease and personalised medicine. The results to date and draft recommendations for application will be discussed by international representatives of patient organisations, pharmaceutical industry, academic statisticians and clinical trial methodologists, clinical researchers, pharmaceutical industry and regulators.

All documents

Name Language First published Last updated
Agenda - FP7 Small-population research methods projects and regulatory application workshop (English only) 2017-02-06 2017-03-28
Agenda - FP7 Small-population research methods projects and regulatory application workshop (English only) 2017-02-06 2017-03-28
Presentation - 1.1 The Asterix project: overview (Kit Roes) (English only) 2017-04-20  
Presentation - 1.1 The Asterix project: overview (Kit Roes) (English only) 2017-04-20  
Presentation - 1.2 Overview on Integrated DEsign and AnaLysis (IDeAl ) of small population group trials (Ralf-Dieter Hilgers) (English only) 2017-04-20  
Presentation - 1.2 Overview on Integrated DEsign and AnaLysis (IDeAl ) of small population group trials (Ralf-Dieter Hilgers) (English only) 2017-04-20  
Presentation - 1.3 InSPiRe: innovative methodology for small-populations research - An overview of research (Nigel Stallard) (English only) 2017-04-20  
Presentation - 1.3 InSPiRe: innovative methodology for small-populations research - An overview of research (Nigel Stallard) (English only) 2017-04-20  
Presentation - 1.4 Regulatory pathways for supporting novel methodologies and opportunities for interactions with regulators (Ine Skottheim Rusten, Anja Schiel) (English only) 2017-04-20  
Presentation - 1.4 Regulatory pathways for supporting novel methodologies and opportunities for interactions with regulators (Ine Skottheim Rusten, Anja Schiel) (English only) 2017-04-20  
Presentation - 1.5 Patient perspective (Christine Laver) (English only) 2017-04-20  
Presentation - 1.5 Patient perspective (Christine Laver) (English only) 2017-04-20  
Presentation - 2.1 Borrowing information at the planning stage (Kit Roes) (English only) 2017-04-20  
Presentation - 2.1 Borrowing information at the planning stage (Kit Roes) (English only) 2017-04-20  
Presentation - 2.2 Hierarchical models: a framework for evidence synthesis (Tim Friede) (English only) 2017-04-20  
Presentation - 2.2 Hierarchical models: a framework for evidence synthesis (Tim Friede) (English only) 2017-04-20  
Presentation - 2.3 Some considerations about combining / borrowing evidence in clinical trials for rare disease (Armin Koch, Kristina Weber) (English only) 2017-04-20  
Presentation - 2.3 Some considerations about combining / borrowing evidence in clinical trials for rare disease (Armin Koch, Kristina Weber) (English only) 2017-04-20  
Presentation - 2.4 Combining evidence : purpose is everything (Stephen Senn) (English only) 2017-04-20  
Presentation - 2.4 Combining evidence : purpose is everything (Stephen Senn) (English only) 2017-04-20  
Presentation - 2.5 Discussion on evidence synthesis (Ségolène Aymé) (English only) 2017-04-20  
Presentation - 2.5 Discussion on evidence synthesis (Ségolène Aymé) (English only) 2017-04-20  
Presentation - 3.1 Extrapolation and bridging of adult information in early-phase dose-finding pediatric studies (Sarah Zohar) (English only) 2017-04-20  
Presentation - 3.1 Extrapolation and bridging of adult information in early-phase dose-finding pediatric studies (Sarah Zohar) (English only) 2017-04-20  
Presentation - 3.2 Adaptive levels of evidence: an extrapolation framework to specify requirements for drug development in children (Franz Konig) (English only) 2017-04-20  
Presentation - 3.2 Adaptive levels of evidence: an extrapolation framework to specify requirements for drug development in children (Franz Konig) (English only) 2017-04-20  
Presentation - 3.3 Equivalence of dose-response curves (Holger Dette) (English only) 2017-04-20  
Presentation - 3.3 Equivalence of dose-response curves (Holger Dette) (English only) 2017-04-20  
Presentation - 3.4 Discussion on extrapolation (Heinz Schmidli) (English only) 2017-04-20  
Presentation - 3.4 Discussion on extrapolation (Heinz Schmidli) (English only) 2017-04-20  
Presentation - 4.1 A value-of-information approach to sample size determination in confirmatory clinical trials in small populations (Nigel Stallard) (English only) 2017-04-20  
Presentation - 4.1 A value-of-information approach to sample size determination in confirmatory clinical trials in small populations (Nigel Stallard) (English only) 2017-04-20  
Presentation - 4.2 Evidence and decisions (Carl-Fredrik Burman) (English only) 2017-04-20  
Presentation - 4.2 Evidence and decisions (Carl-Fredrik Burman) (English only) 2017-04-20  
Presentation - 4.3 Does the level of evidence depend on randomization? (Ralf-Dieter Hilgers, Nicole Heussen, Diane Uschner) (English only) 2017-04-20  
Presentation - 4.3 Does the level of evidence depend on randomization? (Ralf-Dieter Hilgers, Nicole Heussen, Diane Uschner) (English only) 2017-04-20  
Presentation - 4.4 A decision theoretic approach to optimize clinical trial designs for targeted therapies (Martin Posch) (English only) 2017-04-20  
Presentation - 4.4 A decision theoretic approach to optimize clinical trial designs for targeted therapies (Martin Posch) (English only) 2017-04-20  
Presentation - 5.1 Surrogate marker evaluation when data are small, large, or very large (Geert Molenberghs) (English only) 2017-04-20  
Presentation - 5.1 Surrogate marker evaluation when data are small, large, or very large (Geert Molenberghs) (English only) 2017-04-20  
Presentation - 5.2 Goal attainment scaling: individualized instrument with potential for outcome measurement in rare diseases (Hanneke van der Lee, Charlotte Gaasterland, Susanne Urach) (English only) 2017-04-20  
Presentation - 5.2 Goal attainment scaling: individualized instrument with potential for outcome measurement in rare diseases (Hanneke van der Lee, Charlotte Gaasterland, Susanne Urach) (English only) 2017-04-20  
Presentation - 5.3 Leveraging multiple endpoints in small clinical trials (Robin Ristl) (English only) 2017-04-20  
Presentation - 5.3 Leveraging multiple endpoints in small clinical trials (Robin Ristl) (English only) 2017-04-20  
Presentation - 6.1 Historical controls: think cluster not parallel (Stephen Senn, Olivier Collignon, Anna Schritz) (English only) 2017-04-20  
Presentation - 6.1 Historical controls: think cluster not parallel (Stephen Senn, Olivier Collignon, Anna Schritz) (English only) 2017-04-20  
Presentation - 6.2 Optimal use of sequential trial designs in small populations (Stavros Nikolakopoulos, Susanne Urach) (English only) 2017-04-20  
Presentation - 6.2 Optimal use of sequential trial designs in small populations (Stavros Nikolakopoulos, Susanne Urach) (English only) 2017-04-20  
Presentation - 6.3 Incorporating pharmacokinetic information in phase I studies in small populations (Moreno Ursino) (English only) 2017-04-20  
Presentation - 6.3 Incorporating pharmacokinetic information in phase I studies in small populations (Moreno Ursino) (English only) 2017-04-20  
Presentation - 6.4 Improved methodology for use of non-linear mixed effect models (NLMEM) in decision making (Mats O. Karlsson, Andrew Hooker) (English only) 2017-04-20  
Presentation - 6.4 Improved methodology for use of non-linear mixed effect models (NLMEM) in decision making (Mats O. Karlsson, Andrew Hooker) (English only) 2017-04-20  
Presentation - 6.5 Optimal design for trials with discrete longitudinal studies with uncertainty on model and parameters (France Mentré, Florence Loingeville, Marie Karelle Rivière, Thu Thuy Nguyen) (English only) 2017-04-20  
Presentation - 6.5 Optimal design for trials with discrete longitudinal studies with uncertainty on model and parameters (France Mentré, Florence Loingeville, Marie Karelle Rivière, Thu Thuy Nguyen) (English only) 2017-04-20  
Presentation - 6.6 Innovative designs pharmacometrics and optimal designs (Joe Standing) (English only) 2017-04-20  
Presentation - 6.6 Innovative designs pharmacometrics and optimal designs (Joe Standing) (English only) 2017-04-20  
Presentation - 6.6 Innovative designs pharmacometrics and optimal designs (Joe Standing) (English only) 2017-04-20  
Presentation - 6.6 Innovative designs pharmacometrics and optimal designs (Joe Standing) (English only) 2017-04-20  
Presentation - 7 Summary of sessions and way forward (Frank Pétavy) (English only) 2017-04-20  
Presentation - 7 Summary of sessions and way forward (Frank Pétavy) (English only) 2017-04-20  

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