Registration & coffee

Pre Conference course(s)

1. Meta-Analysis of Few Studies: Tim Friede, Christian Röver, University Medical Center Göttingen, Simon Wandel, Novartis
2. Adaptive Designs for Studies in Small Populations: Martin Posch & Gernot Wassmer, Medical University of Vienna

Applications in Paediatrics (I)

• Case study 1: LEVNEONATE study in neonatal seizures:

Géraldine Favrais (University Hospital of Tours) and Sarah Zohar (INSERM)

Applications in Paediatrics (II)

• Case study 2: The EARLY Protect study in Alport Disease:

Introduction to Alport disease and motivation and rationale for the study: Oliver Gross, Dept Nephrology, Göttingen,

Evidence synthesis for a single randomized controlled trial and observational data in small populations: Steffen Unkel, Göttingen

• Discussion of applications in Paediatrics: Norbert Benda and Frederike Lentz (BfArM)

Registration & Coffee

Research in early phase dose-finding trials in small populations

Session chair: Sarah Zohar, INSERM

• Johanna H. van der Lee, Academisch Medisch Centrum Universiteit van Amsterdam
• Using historical data to inform extrapolation decisions in children: Lisa Hampson, Lancaster University
• Incorporating PK/PD information in early phase dose-finding trials in small populations: Moreno Ursino, INSERM

Decision-theoretic approaches for clinical trials in small populations

Session chair: Nigel Stallard, Warwick Medical School, University of Warwick

• The advantages of Bayesian decision analysis in small populations: Andrew R. Willan, University of Toronto
• Optimising group sequential designs: Christopher Jennison, University of Bath
• Decision-theoretic designs for clinical trials in rare diseases/small populations: Dr Siew Wan Hee, Warwick Medical School, University of Warwick

Evidence: Assessment and synthesis

Session chair: Tim Friede, University Medical Center Göttingen, Germany

• On P-values and Bayes factors for small samples: Leo Held, University of Zurich
• Bayesian approaches to extrapolation in clinical research: Heinz Schmidli, Novartis

Break

Research in confirmatory trials for small populations and personalised medicines

Session chair: Martin Posch, Medical University of Vienna

• A General Design for Confirmatory Basket Trials: Robert Beckman, Georgetown University Medical Center, Washington DC
• Optimizing trial design for targeted therapies: Thomas Ondra, Medical University of Vienna
• Clinical Trial Designs with Threshold Selection for a Continuous Biomarker: Alexandra Graf, Medical University of Vienna

Poster Reception

Conference Dinner

Registration & Coffee

Evidence synthesis of clinical trials in small populations and rare diseases

Session chair: Tim Friede, University Medical Center Göttingen, Germany

• Predictive evidential threshold scaling (PETS): does
  the actual evidence meet confirmatory standards?: Beat Neuenschwander, Novartis (40 mins)
• Bayesian meta-analysis for few studies: Christian Röver, University Medical Center, Göttingen (25 mins)
• A design-by-treatment interaction model for network meta-analysis using integrated nested Laplace approximations: Burak Gunhan, University Medical Center, Göttingen (25 mins)

Ongoing work and future challenges in methodology for small population clinical trials

• Simon Day, Clinical Trials Consulting & Training Ltd
• Ralf-Dieter Hilgers, Aachen University
• Kit Roes, University Medical Center, Utrecht

This project has received funding from the European Union's Seventh Framework Programme for research, technological development and demonstration under grant agreement number FP HEALTH 2013 - 602144.