WP3 – Extrapolating dose response information to small populations
The primary goal of the WP is a rigorous statistical methodology for extrapolation of dose response information and conclusions available from a given source population to make inference for another target population which is much smaller in size. We will address issues raised in the CHMP Concept Paper on extrapolation of efficacy and safety information in drug development [CHMP 2012] and develop better methodologies for medicinal drug development that avoid unnecessary studies in the target population for ethical reasons, for efficiency, and to allocate resources to areas where studies are the most needed.
Alternatively, in situations where the feasibility of studies is restricted, the new methodology can be applied for rational interpretation of the limited evidence in the target population in the context of data from other sources.
The main objectives in this context are:
- New statistical measures for similarity of dose-response between a source and a target population
- Extrapolation of efficacy and safety information, accounting for model uncertainty using Bayesian methods
- Robustness against incorrect model assumptions
- Minimisation of false claims through optimal experimental design and dissemination