Adaptive Design Methods in Clinical Trials (Chapman & Hall/CRC Biostatistics Series) [Hardcover]

Shein-Chung Chow (Author), Mark Chang (Author)

Book Description

ISBN-10: 1584887761 | ISBN-13: 978-1584887768 | Publication Date: November 16, 2006 | Edition: 1

Although adaptive design methods are flexible and useful in clinical research, little or no regulatory guidelines are available. One of the first books on the topic, Adaptive Design Methods in Clinical Trials presents the principles and methodologies in adaptive design and analysis that pertain to adaptations made to trial or statistical procedures that are based on accrued data of ongoing clinical trials. The book also offers a well-balanced summary of current regulatory perspectives and recently developed statistical methods in this area.

After an introduction to basic concepts and statistical considerations of adaptive design methods, the book questions the impact on target patient populations as the result of protocol amendments and discusses the generalization of statistical inference. The authors also present various adaptive design methods, including where hypotheses are modified during the conduct of clinical trials, for dose selection, and commonly used adaptive group sequential design methods in clinical trials. Following a discussion of blind procedures for sample size re-estimation, the book describes statistical tests for seamless phase II/III adaptive designs and statistical inference for switching adaptively from one treatment to another. The book concludes with computer simulations and various case studies of clinical trials.

By providing theoretical and computer simulation results, method comparisons, and practical guidelines for choosing an optimal design, Adaptive Design Methods in Clinical Trials fills the need for a unified, comprehensive, and updated resource in the clinical research and development of adaptive design and analysis.

Editorial Reviews

Review

Praise for the First Edition
The authors are to be commended for the endeavour to provide a monograph about this rapidly evolving area in- to our knowledge- the first book specifically devoted to adaptive design methods . . . we think it provides a valuable contribution to the area of adaptive design.
—Frank Miller and Stig Johan Wiklund, AstraZeneca, Statistical Medicine, 2008, Vol. 27

In summary, this is an extremely useful book for clinical trials statisticians wishing to stay abreast with the innovative approaches that are being developed amid some controversies regarding their benefits.
—Yuko Palesch, Medical University of South Carolina, Journal of the American Statistical Association, March 2008, Vol. 103, No. 481

…This book is one of the first of its kind to be released solely dealing with adaptive designs in clinical trials. … a useful reference for those who have a mathematical background and wish to understand some of the adaptive design methodologies. …With the ever-increasing need for adaptive trials, we could see this book having a large influence …
—Pharmaceutical Statistics, 2008

…a useful reference source for anyone interested in advancing innovative trial designs and wishing to incorporate adaptations, modifications, and changes to the drug, device, and biological developmental processes.
—C.M. O’Brien, International Statistical Review, Vol. 75, No. 2, 2007

…uses a broad definition of adaptive design methods… .This breadth of coverage is to be commended and makes the book a useful reference and overview for anyone who is starting to explore this area … the book is worth reading.
—Angela Wade, University College London, UK

The book covers a vast area. To my knowledge this book is the first attempt to provide a concise description of various methodologies in a highly dynamic area. The authors deserve to be praised for bringing this huge range of approaches, including very recent trends, together in one book … .
—Tim Friede, University of Warwick, Biometrics, March 2008

--This text refers to an alternate Hardcover edition.

About the Author

Shein-Chung Chow is a professor in the Department of Biostatistics and Bioinformatics at Duke University School of Medicine. Dr. Chow is also an adjunct professor of clinical sciences at Duke–National University of Singapore Graduate Medical School and the editor-in-chief of the Journal of Biopharmaceutical Statistics. He has authored or co-authored numerous papers and books, including the Handbook of Adaptive Designs in Pharmaceutical and Clinical Development and Controversial Statistical Issues in Clinical Trials.

Mark Chang is the executive director of biostatistics and data management at AMAG Pharmaceuticals and an adjunct professor at Boston University. A fellow of the American Statistical Association, Dr. Chang is a co-founder of the International Society for Biopharmaceutical Statistics and serves on the editorial boards of two statistical journals. He has authored many publications, including Adaptive Design Theory and Implementation Using SAS and R and Monte Carlo Simulation for the Pharmaceutical Industry: Concepts, Algorithms, and Case Studies.

--This text refers to an alternate Hardcover edition.

Product Details

• Hardcover: 296 pages
• Publisher: Chapman and Hall/CRC; 1 edition (November 16, 2006)
• Language: English
• ISBN-10: 1584887761
• ISBN-13: 978-1584887768
• Product Dimensions: 9.1 x 6.2 x 0.8 inches
• Shipping Weight: 1 pounds (View shipping rates and policies)
• Average Customer Review: 2.7 out of 5 stars  See all reviews (3 customer reviews)
• Amazon Best Sellers Rank: #1,393,053 in Books (See Top 100 in Books)

More About the Author

Discover books, learn about writers, read author blogs, and more.

Customer Reviews

Most Helpful Customer Reviews

44 of 46 people found the following review helpful:

5.0 out of 5 stars Great contribution to pharmaceutical industry, April 8, 2007

By 

Michael R. Chernick "statman31147" (Holland PA) - See all my reviews
(REAL NAME)   

Amazon Verified Purchase

This review is from: Adaptive Design Methods in Clinical Trials (Chapman & Hall/CRC Biostatistics Series) (Hardcover)

I meet the second author, Mark Chang, at a conference on adaptive designs. I work as a professional statistician in the pharmaceutical industry. For the past several years, at least ten, these ideas have been the topic of research and it is being investigated as a possible way to speed up drug development and its development is being encouraged by the FDA. There has not been a formal statistical text covering the existing theory and its application to clinical trials. Consequently, when we knew this was coming out we preordered it and have been studying it since it came out last November.

The book has lived up to expectations. Adaptive designs are very similar to group sequential designs in that they have planned times to make preliminary assessment of the trial data and then decide whether or not to continue the trial or modify the design. Adaptive designs can be more flexible than their group sequential counterparts. They even can allow changes to the protocol as long as the criteria for making such changes are mapped out in advance of the trial.

These methods have been controversial in the past and simulation studies are often required to determine their properties. But there has been enough development now that some designs are being applied in real trials. In fact we are considering a two stage adaptive design similar to the ones described in this text (except applied to bioequivalence).

Later this year Mark Chang is coming out with an applied text that include SAS macros to aid in the implementation of the methods. A preview of the manuscript was displayed at an adaptive trials conference that I attended recently. I can enthusiastically recommend that one even more than this one! However, any biostatistician working on clinical trials should have this book on his or her bookshelf.

7 of 7 people found the following review helpful:

2.0 out of 5 stars This book is a "beta" version, January 13, 2008

By 

Andrew M. Hartley (North Carolina) - See all my reviews
(REAL NAME)   

This review is from: Adaptive Design Methods in Clinical Trials (Chapman & Hall/CRC Biostatistics Series) (Hardcover)

This book's best features are its bibliography (about 240 entries) and its broad survey and taxonomy of adaptive methods. Its publication represents an important step in popularizing adaptive trials and, thus, streamlining drug/device/biologic development pipelines.

The book is, however, filled with inaccuracies on several levels: incorrect grammar and equation references, undefined symbols, a reference to a non-existent appendix, unclear language (e.g., what is the "statistical strength for rejecting Ho" on Page 150?), mathematical typos [e.g., P(x|y,theta) rather than P(y|theta) in the integrand for the posterior predictive probability distribution P(y|x)], and misapplications of statistical philosophy (e.g., using Neyman-Pearson hypothesis testing for statistical inference, identifying the p-value as a post-hoc type I error rate). In the sample I took of about 1/3 of the pages, about 120 errors occur. The book should be considered only a pre-publication "beta" version. Any second edition should receive much more attention to detail.

A statistician or clinical scientist planning a potentially adaptive trial could use this book to learn about some of the aspects of a trial that can be made adaptive. The book could also help him/her to assess the assumptions and mathematical complexity of methods under consideration. However, when it comes to actually performing an analysis, one would want to use the bibliography to obtain the relevant articles and books, perhaps together with Chang's "Adaptive Design Theory and Implementation Using SAS and R" (Chapman & Hall/CRC Biostatistics).

Overall, this book disappointed me. The authors should have had several more collaborators and copyeditors check their work.

1 of 1 people found the following review helpful:

1.0 out of 5 stars The software that accompanies this book is only intended to be functional for one year., January 13, 2010

By 

Bruce E. Rodda (Spicewood, TX USA) - See all my reviews
(REAL NAME)   

This review is from: Adaptive Design Methods in Clinical Trials (Chapman & Hall/CRC Biostatistics Series) (Hardcover)

I used this book and accompanying software for about a year and was surprised, when opening the software, to be greeted with the message, "Your license will expire within 30 days. Please contact [e-mail] to renew it immediately." Neither the book, nor the accompanying CD mentions any expiration of the software. When I contacted the vendor, they offered a brief license extension, but going forward I would need to renew the license for $495 per year. When I contacted Wiley, their response was apologetic that they hadn't mentioned the limited life of the software, and that they would do nothing until the next reprinting (at an unspecified time in the future). It was unclear what they would do then.

Although there are some warts, I found the book and software to be useful. However, the book is not valuable without the software and if you plan to use the combination for more than a year, be prepared to pay several hundred dollars per annum to renew the software license.