statistical aspects of the design and analysis of clinical trials (revised edition) everitt bs,...
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PHARMACEUTICAL STATISTICS
Pharmaceut. Statist. 2005; 4: 225–228
Published online in Wiley InterScience (www.interscience.wiley.com).
Book Reviews
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the PSI.
Statistical Aspects of the Design and Analysis of Clinical Trials
(Revised Edition)
Everitt BS, Pickles A (2004)
ISBN 1860944418; 340 pages; £48.00, $78.00
Imperial College Press; http://www.icpress.co.uk/books/lifesci/
p321.html
This book has an aim somewhat different from others that
provide comprehensive accounts of how clinical trials should be
carried out and organized. As the title suggests, it provides a
concise account of the more statistical aspects of the design and
analysis of clinical trials. The authors have particularly focused
on statistical methods developed over the last decade or so and
are also largely concerned with Phase III trials. As mentioned in
the preface, the authors hope that the book will be useful to
medical statisticians and others faced with the task of designing
and analysing clinical trials.
The book is divided into ten succinct chapters, with an
appendix related to software. The first chapter gives a brief
introduction to clinical trials from a historical and ethical
perspective, as well as introducing the four phases of drug
development. The second chapter discusses allocation to
treatment, sample-size determination and the reporting of
results. Various methods of randomization are illustrated with
examples. Unfortunately, the subsection on determining the size
of a trial is described briefly and without examples. In the third
chapter, the authors describe methods that should be utilized
when monitoring the progress of a clinical trial. Importantly, the
intention-to-treat approach is discussed, as well as approaches
for dealing with missing data and dropouts. The subsection on
interim analyses gives comprehensive references concerning the
overall maintenance of alpha in clinical trials. The following six
chapters introduce analysis methods within clinical trials,
including generalized linear models, analysis of longitudinal
data, survival analysis, Bayesian methods for longitudinal data,
and meta-analysis. In some cases, theoretical descriptions are
given in tables, which help to maintain the overall flow of the
text and are ideally designed for future reference. The appendix
is a welcome part of the book that will allow the user to further
investigate the software mentioned throughout the text.
Each chapter has several subsections of topics and an overall
summary. The relative strengths and weaknesses of methods
introduced are given along with their practical application; but
those seeking either detailed theory or application will need to
refer to other material. Furthermore, the vast majority of the
content focuses on longitudinal analyses and does not delve into
other new areas such as genomics: a chapter on emerging
methodologies in clinical trials could have dealt with this issue. As
a statistician in the pharmaceutical industry, I was also interested
in knowing how acceptable many of these methodologies were to
regulatory authorities. Unfortunately, any detailed experience the
authors have in this area was missing from the book.
As a concise reference, I found the book to be very useful. In
providing a concise description of the statistical aspects of the
design and analysis of clinical trials, free of any major
typographical errors, the authors have succeeded. Those
concerned with the correct design and analysis of clinical trials,
but wishing to avoid either the advanced theoretical aspects or
too much focus on application of methodologies, will find this
book to be very accessible with relatively up-to-date references.
Jatinder Singh
PRA International, Canada
(DOI: 10.1002/pst.165)
Copyright # 2005 John Wiley & Sons, Ltd.Received 15 January 20012005