Seventh Framework Programme (FP7) small-population research methods projects and regulatory application workshop
The 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.
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Agenda - FP7 Small-population research methods projects and regulatory application workshop (PDF/854.97 KB)
Draft
First published: 06/02/2017
Last updated: 28/03/2017
EMA/849195/2016 -
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Presentation - 1.1 The Asterix project: overview (Kit Roes) (PDF/702.39 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 1.2 Overview on Integrated DEsign and AnaLysis (IDeAl ) of small population group trials (Ralf-Dieter Hilgers) (PDF/1.12 MB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 1.3 InSPiRe: innovative methodology for small-populations research - An overview of research (Nigel Stallard) (PDF/334.04 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 1.4 Regulatory pathways for supporting novel methodologies and opportunities for interactions with regulators (Ine Skottheim Rusten, Anja Schiel) (PDF/358.09 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 1.5 Patient perspective (Christine Laver) (PDF/1.11 MB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 2.1 Borrowing information at the planning stage (Kit Roes) (PDF/281.87 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 2.2 Hierarchical models: a framework for evidence synthesis (Tim Friede) (PDF/465.66 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 2.3 Some considerations about combining / borrowing evidence in clinical trials for rare disease (Armin Koch, Kristina Weber) (PDF/452.67 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 2.4 Combining evidence : purpose is everything (Stephen Senn) (PDF/574.86 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 2.5 Discussion on evidence synthesis (Sà©golà¨ne Aymà©) (PDF/723.16 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 3.1 Extrapolation and bridging of adult information in early-phase dose-finding pediatric studies (Sarah Zohar) (PDF/878.56 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 3.2 Adaptive levels of evidence: an extrapolation framework to specify requirements for drug development in children (Franz Konig) (PDF/592.42 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 3.3 Equivalence of dose-response curves (Holger Dette) (PDF/235.7 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 3.4 Discussion on extrapolation (Heinz Schmidli) (PDF/307.67 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 4.1 A value-of-information approach to sample size determination in confirmatory clinical trials in small populations (Nigel Stallard) (PDF/371.23 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 4.2 Evidence and decisions (Carl-Fredrik Burman) (PDF/136.67 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 4.3 Does the level of evidence depend on randomization? (Ralf-Dieter Hilgers, Nicole Heussen, Diane Uschner) (PDF/391.43 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 4.4 A decision theoretic approach to optimize clinical trial designs for targeted therapies (Martin Posch) (PDF/275.9 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 5.1 Surrogate marker evaluation when data are small, large, or very large (Geert Molenberghs) (PDF/159.73 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 5.2 Goal attainment scaling: individualized instrument with potential for outcome measurement in rare diseases (Hanneke van der Lee, Charlotte Gaasterland, Susanne Urach) (PDF/289.16 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 5.3 Leveraging multiple endpoints in small clinical trials (Robin Ristl) (PDF/154.46 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 6.1 Historical controls: think cluster not parallel (Stephen Senn, Olivier Collignon, Anna Schritz) (PDF/505.52 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 6.2 Optimal use of sequential trial designs in small populations (Stavros Nikolakopoulos, Susanne Urach) (PDF/561.53 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 6.3 Incorporating pharmacokinetic information in phase I studies in small populations (Moreno Ursino) (PDF/23.07 MB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 6.4 Improved methodology for use of non-linear mixed effect models (NLMEM) in decision making (Mats O. Karlsson, Andrew Hooker) (PDF/651.74 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 6.5 Optimal design for trials with discrete longitudinal studies with uncertainty on model and parameters (France Mentrà©, Florence Loingeville, Marie Karelle Rivià¨re, Thu Thuy Nguyen) (PDF/765.34 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 6.6 Innovative designs pharmacometrics and optimal designs (Joe Standing) (PDF/928.5 KB)
First published: 20/04/2017
Last updated: 20/04/2017 -
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Presentation - 7 Summary of sessions and way forward (Frank Pà©tavy) (PDF/85.02 KB)
First published: 20/04/2017
Last updated: 20/04/2017