Synopses & Reviews
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.
Review
"...A broad range of statistical methodologies is surveyed including restricted chi-square tests, normal theory tests, nonparametric tests, permutation and resampling methods and Bayesian estimation...For anyone who wants to think deeply about the basic issues involving the design and analysis of clinical trials, this book is a must read.."(Zentralblatt für Mathematik)
Synopsis
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.