Discussion on Extrapolation Heinz Schmidli FP7 Small-population - - PowerPoint PPT Presentation

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Statistical Methodology Clinical Development Discussion on Extrapolation Heinz Schmidli FP7 Small-population research methods projects and regulatory application workshop London 29-30 March 2017 Evidence Synthesis and Extrapolation Source

SLIDE 1

Discussion on Extrapolation

Heinz Schmidli

FP7 Small-population research methods projects and regulatory application workshop London 29-30 March 2017

Statistical Methodology Clinical Development

SLIDE 2

Evidence Synthesis and Extrapolation

Source data

Y1 Y2 YJ ...

θ1

θJ θ2

θ*

Y* Target data ?

Borrowing strength by linking parameters
Robust extrapolation for decision making and design
Bayesian framework natural for inference/prediction

SLIDE 3

Borrow ing strength and Robustness

Borrowing strength by linking parameters

Test and Pool

Yes/No Holger Dette

Hierarchical models

Gradual Sarah Zohar Robustness Possibility that source data may not be relevant Prior probability for scepticism Franz König

FDA (2016) Leveraging existing clinical data for extrapolation to pediatric uses of medical devices. Schmidli H et al. (2014) Biometrics

SLIDE 4

Decision making and Design

Clinical development

Data from various phases

Pre-clinical, phase I, II, III, ...

Data from different populations

Adult/pediatric, disease subtypes, ...

Sequentially available data used for

Stop/go decisions
Design of future trials

"... design is a decision problem. Introduction of some kind of prior information can hardly be avoided.“ DR Cox

Neuenschwander et al. (2016) Statistics in Biopharmaceutical Research