The Use of Restricted Significance Tests in Clinical Trials (Statistics for Biology and Health)

The reader will soon find that this is more than a "how-to-do-it" book. It describes a philosophical approach to the use of statistics in the analysis of clinical trials. I have come gradually to the position described here, but I have not come that way alone. This approach is heavily influenced by my reading the papers of R.A. Fisher, F.S. Anscombe, F. Mosteller, and J. Neyman. But the most important influences have been those of my medical colleagues, who had important real-life medical questions that needed to be answered. Statistical methods depend on abstract mathematical theorems and often complicated algorithms on the computer. But these are only a means to an end, because in the end the statistical techniques we apply to clinical studies have to provide useful answers. When I was studying martingales and symbolic logic in graduate school, my wife, Fran, had to be left out of the intellectual excitement. But, as she looked on, she kept asking me how is this knowledge useful. That question, what can you do with this? haunted my studies. When I began working in bio­ statistics, she continued asking me where it was all going, and I had to explain what I was doing in terms of the practical problems that were being ad­ dressed. Front Matter....Pages i-x Front Matter....Pages 1-1 The Randomized Controlled Clinical Trial....Pages 3-9 Probability Models and Clinical Medicine....Pages 10-21 Significance Tests Versus Hypothesis Tests....Pages 22-29 Neyman’s Insights....Pages 30-38 Reconciling Fisher and Neyman....Pages 39-46 Front Matter....Pages 47-47 Continous Measures Taken over Time....Pages 49-59 Combining Data Across Measures....Pages 60-77 Counts of Events....Pages 78-88 Permutation Tests and Resampling Techniques....Pages 89-96 Neyman’s Restricted Chi Square Tests....Pages 97-103 Miscellaneous Methods for Increasing Power....Pages 104-114 Bayesian Estimation....Pages 115-125 An Example of the Type of Analysis Suggested in This Book....Pages 126-160 Back Matter....Pages 161-174

This thought-provoking book discusses the use of statistics in randomized clinical trials. Its aim is two-fold: firstly, it presents a clear account of the design and analysis of experiments in this setting which stresses the foundational issues involved. Secondly, the book seeks to develop the specific tools of analysis which can be derived from Neyman's model of restricted tests. The book is based on the author's many years of experience of clinical trials. Throughout, examples are used from a variety of types of study. As a result, all statisticians and research scientists who work on clinical trials will find this presentation clear and accessible, and very relevant to their own research interests.