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Activity Number: 483 - Multiplicity
Type: Contributed
Date/Time: Wednesday, August 1, 2018 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract #330264 Presentation
Title: Considerations on MCPMod-Type Methods for Proof of Concept in Multi-Armed Studies
Author(s): Tobias Mielke*
Companies: Janssen-Cilag GmbH
Keywords: Proof of concept; Model-based methods; MCPMod; Multiple testing; Dose finding

Prior to larger Phase 2b dose-finding or confirmatory Phase 3 studies, initial signs of efficacy are established in small proof of concept (PoC) studies. Multiple dose groups are frequently not considered in these studies, as the sample size shall be kept at a minimum due to the uncertainty on the efficacy of the drug. The inclusion of multiple dose groups reduces the power for the PoC, while adding valuable insight on the dose-response relation. Model-based methods support efficient sharing of data between treatment groups, what could also reduce the loss of efficiency in multi-armed PoC studies. The commonly used MCPMod approach consists of a model-based trend test to establish any drug-related effect, followed by a dose-response modelling step. In particular the model-based trend test is sometimes considered to serve as a PoC criterion in multi-armed studies. However, generally not only the existence of any drug related effects is of interest, but also the size of effects. Different methods on how to extend the MCPMod framework to test efficiently for targeted effects will be introduced and discussed in the presentation.

Authors who are presenting talks have a * after their name.

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