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Adaptive Design Methods in Clinical Trials, Second Edition

By Shein-Chung Chow, Mark Chang

Chapman and Hall/CRC – 2012 – 374 pages

Series: Chapman & Hall/CRC Biostatistics Series

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    978-1-43-983987-4
    November 30th 2011

Description

With new statistical and scientific issues arising in adaptive clinical trial design, including the U.S. FDA’s recent draft guidance, a new edition of one of the first books on the topic is needed. Adaptive Design Methods in Clinical Trials, Second Edition reflects recent developments and regulatory positions on the use of adaptive designs in clinical trials. It unifies the vast and continuously growing literature and research activities on regulatory requirements, scientific and practical issues, and statistical methodology.

New to the Second Edition

Along with revisions throughout the text, this edition significantly updates the chapters on protocol amendment and clinical trial simulation to incorporate the latest changes. It also includes five entirely new chapters on two-stage adaptive design, biomarker adaptive trials, target clinical trials, sample size and power estimation, and regulatory perspectives.

Following in the tradition of its acclaimed predecessor, this second edition continues to offer an up-to-date resource for clinical scientists and researchers in academia, regulatory agencies, and the pharmaceutical industry. Written in an intuitive style at a basic mathematical and statistical level, the book maintains its practical approach with an emphasis on concepts via numerous examples and illustrations.

Reviews

"In this second edition, the authors update two chapters on protocol amendment and clinical trial simulation to reflect new developments after the first edition and add five new chapters. … It also provides many useful algorithms for various designs and SAS code throughout the book. … this is an excellent book with wonderful resources on adaptive design methods in clinical trials. It will be very useful to graduate students in the areas of clinical development and biostatistics looking for an advanced textbook on this topic. The book is very well written and a joy to read. I think it would be a critical addition to the bookshelf for statisticians involved in adaptive design and analysis in clinical trials."

—Hongfei Guo, Journal of the American Statistical Association, December 2013

"This second edition remains a useful reference source for anyone interested in advancing innovative trial designs and wishing to incorporate adaptations, modifications, and changes to the drug development process. Five new chapters have been added and are all worth reading; bringing the technical material covered up-to-date. For anyone working in, and studying, clinical research the book is worth purchasing and will make a valuable addition to any library. … this revision continues to provide a balanced summary of statistical methods, together with the authors’ perspective on current regulatory practice."

International Statistical Review, 80, 2012

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

Contents

Introduction

What Is Adaptive Design

Regulatory Perspectives

Target Patient Population

Statistical Inference

Practical Issues

Aims and Scope of the Book

Protocol Amendment

Introduction

Moving Target Patient Population

Analysis with Covariate Adjustment

Assessment of Sensitivity Index

Sample Size Adjustment

Concluding Remarks

Adaptive Randomization

Conventional Randomization

Treatment-Adaptive Randomization

Covariate-Adaptive Randomization

Response-Adaptive Randomization

Issues with Adaptive Randomization

Summary

Adaptive Hypotheses

Modifications of Hypotheses

Switch from Superiority to Noninferiority

Concluding Remarks

Adaptive Dose-Escalation Trials

Introduction

CRM in Phase I Oncology Study

Hybrid Frequentist-Bayesian Adaptive Design

Design Selection and Sample Size

Concluding Remarks

Adaptive Group Sequential Design

Sequential Methods

General Approach for Group Sequential Design

Early Stopping Boundaries

Alpha Spending Function

Group Sequential Design Based on Independent P-Values

Calculation of Stopping Boundaries

Group Sequential Trial Monitoring

Conditional Power

Practical Issues

Statistical Tests for Seamless Adaptive Designs

Why a Seamless Design Is Efficient

Step-Wise Test and Adaptive Procedures

Contrast Test and Naive P-Value

Comparisons of Seamless Design

Drop-the-Loser Adaptive Design

Summary

Adaptive Sample Size Adjustment

Sample Size Re-Estimation without Unblinding Data

Cui-Hung-Wang’s Method

Proschan-Hunsberger’s Method

Müller-Schafer Method

Bauer-Köhne Method

Generalization of Independent P-Value Approaches

Inverse-Normal Method

Concluding Remarks

Two-Stage Adaptive Design

Introduction

Practical Issues

Types of Two-Stage Adaptive Designs

Analysis for Seamless Design with Same Study Objectives/Endpoints

Analysis for Seamless Design with Different Endpoints

Analysis for Seamless Design with Different Objectives/Endpoints

Concluding Remarks

Adaptive Treatment Switching

Latent Event Times

Proportional Hazard Model with Latent Hazard Rate

Mixed Exponential Model

Concluding Remarks

Bayesian Approach

Basic Concepts of Bayesian Approach

Multiple-Stage Design for Single-Arm Trial

Bayesian Optimal Adaptive Designs

Concluding Remarks

Biomarker Adaptive Trials

Introduction

Types of Biomarkers and Validation

Design with Classifier Biomarker

Adaptive Design with Prognostic Biomarker

Adaptive Design with Predictive Marker

Concluding Remarks

Appendix

Target Clinical Trials

Introduction

Potential Impact and Significance

Evaluation of Treatment Effect

Other Study Designs and Models

Concluding Remarks

Sample Size and Power Estimation

Framework and Model/Parameter Assumptions

Method Based on the Sum of P-Values

Method Based on Product of P-Values

Method with Inverse-Normal P-Values

Sample Size Re-Estimation

Summary

Clinical Trial Simulation

Introduction

Software Application of ExpDesign Studio

Early Phases Development

Late Phases Development

Concluding Remarks

Regulatory Perspectives — A Review of FDA Draft Guidance

Introduction

The FDA Draft Guidance

Well-Understood Designs

Less Well-Understood Designs

Adaptive Design Implementation

Concluding Remarks

Case Studies

Basic Considerations

Adaptive Group Sequential Design

Adaptive Dose-Escalation Design

Two-Stage Phase II/III Adaptive Design

Bibliography

Subject Index

Author Bio

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.

Name: Adaptive Design Methods in Clinical Trials, Second Edition (Hardback)Chapman and Hall/CRC 
Description: By Shein-Chung Chow, Mark Chang. With new statistical and scientific issues arising in adaptive clinical trial design, including the U.S. FDA’s recent draft guidance, a new edition of one of the first books on the topic is needed. Adaptive Design Methods in Clinical Trials,...
Categories: Statistics for the Biological Sciences, Clinical Implementation