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This page is maintained by the Multiplicity Advisory Board (Alex Dmitrienko).
Annotated bibliography project
The annotated bibliography lists publications (papers, book chapters and sometimes presentations) that deal with the analysis of multiple outcomes in pharmaceutical applications. Each publication is accompanied by a brief summary that describes the proposed statistical methods and their applications to pharmaceutical research. The purpose of the annotated bibliography is to help researchers quickly review publications in the area, understand the main themes/approaches and assess their applicability to a particular problem encountered in pharmaceutical drug development.
If you would like to contribute to the annotated bibliography project, we encourage you to post relevant information in this discussion thread in the BioPharmNet forum.
Multiple tests based on marginal p-values rely on elementary probability inequalities and thus do not depend on the joint distribution of test statistics. These multiple testing procedures are intuitive, easy to apply and widely used in pharmaceutical applications.
Parametric multiple tests make explicit assumptions about the joint distribution of the individual test statistics (for example, the test statistics follow a multivariate normal or t distribution). These tests are more powerful than multiple tests based on marginal p-values.
Multi-stage testing procedures play an important role in clinical trials with multiple families of tests (often termed gatekeepers) that are carried out in a sequential manner.
Tests for the global null hypothesis of no treatment effect are used in clinical trials with multiple (co-primary) outcome variables when clinical trial researchers are interested in studying the effects of an investigational therapy on all of the variables.