Noninferiority Trials

Authored by: Kenneth J. Kopecky , Stephanie Green

Handbook of Statistics in Clinical Oncology

Print publication date:  March  2012
Online publication date:  March  2012

Print ISBN: 9781439862001
eBook ISBN: 9781439862018
Adobe ISBN:


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Phase III therapeutic trials in oncology are conducted to compare the effectiveness of treatment regimens. In most settings, an accepted standard therapy exists and the motivation is the hope that a new treatment regimen (E) will prove to be superior to the standard therapy (S) in some respect, for example, survival or response rate. Such trials are called “superiority trials.” Let ρ(X,Y) denote a parameter that characterizes the difference between outcomes with treatments X and Y. Without loss of generality, we assume that ρ(E,S) is parameterized so that ρ(E,S) = 0 if E and S are equally effective and ρ(E,S) > 0 if E is superior to S. For example, for comparisons of response rates, ρ(E,S) might be rate difference P E P S or the log odds ratio ln{PE(1−PS )/[PS ((1−PE )]}, where P X denotes the response rate for arm X = S or E. Similarly ρ(E,S) might be the log hazard ratio for S relative to E in comparing censored outcomes such as overall or progression-free survival, or ρ(E,S) might be the difference in mean values of a continuous outcome variable measured without censoring, such as a quantitative measure of molecular response. In the hypothesis testing context, a superiority trial tests the null hypothesis H 0:ρ(E,S) ≦ 0 against the one-sided alternative H A :ρ(E,S) > 0.

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