Confirmatory trials hypothesis testing

The requirement to show statistical significance in therapeutic confirmatory trials places a certain importance on a priori hypothesis testing. However, as we have seen, there is much more to clinical research than p-values. Piantadosi (2005) commented on this issue as follows ... 

The null hypothesis is the crux of hypothesis testing. (It is important to note that the form of the null hypothesis varies in different statistical approaches. As the main type of clinical trial discussed in this book is the therapeutic confirmatory trial, we talk about this first. We then talk briefly about the forms of the null hypothesis that are used in other types of trials in Section 3.10.) As noted earlier, therapeutic confirmatory trials are comparative in nature. We want to evaluate the efficacy of the investi-... 

We discussed hypothesis testing in some detail in Chapter 6. For present purposes, the role of hypothesis testing in confirmatory clinical trials can be restated simply as follows ... 

What is the real purpose of running a clinical trial In a confirmatory trial, the stated purpose of that trial is usually to test the null hypothesis. Clinical trials are often focused on testing the null hypothesis because there is usually an alternative model, such as that the drug does have clinically relevant activity, that can be accepted in place of the null model. Furthermore, testing the null hypothesis is an... 

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