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

• Date:
  Wednesday, 29 March 2017, All day
• Location:
  European Medicines Agency, Amsterdam, the Netherlands

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.

Documents

Agenda - FP7 Small-population research methods projects and regulatory application workshop

DraftReference Number: EMA/849195/2016

Presentation - 1.1 The Asterix project: overview (Kit Roes)

Presentation - 1.2 Overview on Integrated DEsign and AnaLysis (IDeAl ) of small population group trials (Ralf-Dieter Hilgers)

Presentation - 1.3 InSPiRe: innovative methodology for small-populations research - An overview of research (Nigel Stallard)

Summary:

FP7 small-population research methods projects and regulatory application workshop

Presentation - 1.4 Regulatory pathways for supporting novel methodologies and opportunities for interactions with regulators (Ine Skottheim Rusten, Anja Schiel)

Presentation - 1.5 Patient perspective (Christine Laver)

Presentation - 2.1 Borrowing information at the planning stage (Kit Roes)

Presentation - 2.2 Hierarchical models: a framework for evidence synthesis (Tim Friede)

Presentation - 2.3 Some considerations about combining / borrowing evidence in clinical trials for rare disease (Armin Koch, Kristina Weber)

Presentation - 2.4 Combining evidence : purpose is everything (Stephen Senn)

Summary:

FP7 small-population research methods projects and regulatory application workshop

Presentation - 2.5 Discussion on evidence synthesis (Sà©golà¨ne Aymà©)

Presentation - 3.1 Extrapolation and bridging of adult information in early-phase dose-finding pediatric studies (Sarah Zohar)

Presentation - 3.2 Adaptive levels of evidence: an extrapolation framework to specify requirements for drug development in children (Franz Konig)

Presentation - 3.3 Equivalence of dose-response curves (Holger Dette)

Presentation - 3.4 Discussion on extrapolation (Heinz Schmidli)

Presentation - 4.1 A value-of-information approach to sample size determination in confirmatory clinical trials in small populations (Nigel Stallard)

Summary:

FP7 Small-population research methods projects and regulatory application workshop

Presentation - 4.2 Evidence and decisions (Carl-Fredrik Burman)

Presentation - 4.3 Does the level of evidence depend on randomization? (Ralf-Dieter Hilgers, Nicole Heussen, Diane Uschner)

Presentation - 4.4 A decision theoretic approach to optimize clinical trial designs for targeted therapies (Martin Posch)

Presentation - 5.1 Surrogate marker evaluation when data are small, large, or very large (Geert Molenberghs)

Presentation - 5.2 Goal attainment scaling: individualized instrument with potential for outcome measurement in rare diseases (Hanneke van der Lee, Charlotte Gaasterland, Susanne Urach)

Presentation - 5.3 Leveraging multiple endpoints in small clinical trials (Robin Ristl)

Presentation - 6.1 Historical controls: think cluster not parallel (Stephen Senn, Olivier Collignon, Anna Schritz)

Summary:

FP7 Small-population research methods projects and regulatory application workshop

Presentation - 6.2 Optimal use of sequential trial designs in small populations (Stavros Nikolakopoulos, Susanne Urach)

Presentation - 6.3 Incorporating pharmacokinetic information in phase I studies in small populations (Moreno Ursino)

Presentation - 6.4 Improved methodology for use of non-linear mixed effect models (NLMEM) in decision making (Mats O. Karlsson, Andrew Hooker)

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)

Presentation - 6.6 Innovative designs pharmacometrics and optimal designs (Joe Standing)

Presentation - 7 Summary of sessions and way forward (Frank Pà©tavy)

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