Statistical Tests Based on New Composite Hypotheses in Clinical Trials Reflecting the Relative Clinical Importance of Multiple Endpoints Quantitatively

In clinical trials, several endpoints (EPs) are often evaluated to compare treatments in some therapeutic area. Suppose that there are two EPs in a clinical trial. We propose a new set of composite hypotheses for continuous variables, taking the relative clinical importance of the EPs into account....

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Bibliographic Details
Published in:Biometrical journal 2009-10, Vol.51 (5), p.749-762
Main Authors: Nishikawa, Masako, Tango, Toshiro, Ohtaki, Megu
Format: Article
Language:English
Subjects:
Applications
Asthma - drug therapy
Asthma - physiopathology
Biology, psychology, social sciences
Biometry - methods
Clinical Trials as Topic - statistics & numerical data
Composite endpoint
Endpoint Determination - statistics & numerical data
Exact sciences and technology
Forced Expiratory Volume
General topics
Humans
Insurance, economics, finance
Mathematics
Medical sciences
Models, Statistical
Multiple endpoints
One-sided tests
Probability and statistics
Pulmonary Disease, Chronic Obstructive - drug therapy
Pulmonary Disease, Chronic Obstructive - physiopathology
Randomized Controlled Trials as Topic - statistics & numerical data
Relative clinical weights
Sciences and techniques of general use
Statistics
Superiority
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Summary:In clinical trials, several endpoints (EPs) are often evaluated to compare treatments in some therapeutic area. Suppose that there are two EPs in a clinical trial. We propose a new set of composite hypotheses for continuous variables, taking the relative clinical importance of the EPs into account. The main hypotheses were formulated to show that a treatment is so superior to the control treatment, which is not necessarily a placebo, in one EP, that the possible non‐inferiority of the treatment by at most a certain value in the other EP can be compensated sufficiently, taking the clinical point of view into account. The maximum non‐inferiority margin of one EP might not be a biologically unimportant difference in exchange for much superiority of the other EP. This formulation leads to a new composite EP and a very simple test statistic. The intersection‐union principle was employed to derive the proposed test.
ISSN:0323-3847
1521-4036
DOI:10.1002/bimj.200800190