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Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs by N. Balakrishnan

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Chapter 73

Superiority Trials

David L. Sackett

73.1 Introduction

This entry is intended to explain (or at least demystify) what trialists mean by “superiority” trials. To understand them, they must be considered in light of another other major trial design, the “non-inferiority” trial. This article will start from a clinical perspective and then negotiate the accompanying statistical concepts. To make the latter presentation easier to follow, it will be assumed at the outset that the measure of patient outcomes under discussion combines both benefit and harm, as in a global measure of function or quality of life. In addition, because these ideas can be difficult to grasp the first time they are encountered, this article will describe them in both words and pictures. Moreover, although this discussion will employ P-values for simplicity’s sake, the author prefers the 95% confidence interval as a more informative measure of the size of treatment effects. Finally, although the “control” treatment in the following examples is often referred to as a “placebo,” these principles apply equally well to nondrug trials and to trials in which the control treatment is an alternative active treatment.

73.2 Clinicians Ask One-Sided Questions, and Want Immediate Answers

When busy clinicians encounter a new treatment, they ask themselves 2 questions [1]. First, is it better than (“superior to”) what they are using now? Second, if it is not superior, is it as good as what they are using now (“non-inferior”) ...

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