Statistics for Biology and Health

Practical Considerations for Adaptive Trial Design and Implementation

Editors: He, Weili, Pinheiro, Jose, Kuznetsova, Olga M. (Eds.)

  • Covers in detail design and implementation of the full scope of adaptive clinical trial designs
  • Examples of trial modeling and adaptive simulation are presented throughout
  • Statistical approaches for probability of success and optimal dose selection are provided as well
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eBook $99.00
price for USA (gross)
  • ISBN 978-1-4939-1100-4
  • Digitally watermarked, DRM-free
  • Included format: PDF, EPUB
  • ebooks can be used on all reading devices
  • Immediate eBook download after purchase
Hardcover $129.00
price for USA
  • ISBN 978-1-4939-1099-1
  • Free shipping for individuals worldwide
  • Usually dispatched within 3 to 5 business days.
Softcover $129.00
price for USA
  • ISBN 978-1-4939-5539-8
  • Free shipping for individuals worldwide
  • Usually dispatched within 3 to 5 business days.
About this book

This edited volume is a definitive text on adaptive clinical trial designs from creation and customization to utilization. As this book covers the full spectrum of topics involved in the adaptive designs arena, it will serve as a valuable reference for researchers working in industry, government and academia. The target audience is anyone involved in the planning and execution of clinical trials, in particular, statisticians, clinicians, pharmacometricians, clinical operation specialists, drug supply managers, and infrastructure providers. In spite of the increased efficiency of adaptive trials in saving costs and time, ultimately getting drugs to patients sooner, their adoption in clinical development is still relatively low. One of the chief reasons is the higher complexity of adaptive design trials as compared to traditional trials. Barriers to the use of clinical trials with adaptive features include the concerns about the integrity of study design and conduct, the risk of regulatory non-acceptance, the need for an advanced infrastructure for complex randomization and clinical supply scenarios, change management for process and behavior modifications, extensive resource requirements for the planning and design of adaptive trials and the potential to relegate key decision makings to outside entities. There have been limited publications that address these practical considerations and recommend best practices and solutions. This book fills this publication gap, providing guidance on practical considerations for adaptive trial design and implementation. The book comprises three parts: Part I focuses on practical considerations from a design perspective, whereas Part II delineates practical considerations related to the implementation of adaptive trials. Putting it all together, Part III presents four illustrative case studies ranging from description and discussion of specific adaptive trial design considerations to the logistic and regulatory issues faced in trial implementation.

Bringing together the expertise of leading key opinion leaders from pharmaceutical industry, academia, and regulatory agencies, this book provides a balanced and comprehensive coverage of practical considerations for adaptive trial design and implementation.

About the authors

Weili He is a Director and Senior Principle Scientist of Clinical Biostatistics at Merck & Co., Inc. She has a Ph.D. degree in biostatistics. She has extensive experience in drug development, and has worked in many therapeutic areas over the years. Dr. He has been active both at Merck and in industry for initiatives relating to adaptive trials, with particular focus on adaptive trial design and implementation.  She is a core member of the DIA Adaptive Design Scientific Working Group (ADSWG) and co-chair of the DIA ADSWG KOL Lecture series. Her research and collaboration with colleagues in various disciplines has led to over 30 publications in statistical and medical journals.   

José Pinheiro has a Ph.D. in Statistics from the University of Wisconsin – Madison, having worked at Bell Labs and Novartis Pharmaceuticals, before his current position as Head of Statistical Modeling in the Model-Based Drug Development department at Janssen Research & Development. He has been involved in methodological development in various areas of statistics and drug development, including dose-finding, adaptive designs and mixed-effects models. He is a Fellow of the American Statistical Association, former co-chair of the PhRMA working group on Adaptive Dose-Ranging Studies, former core member of the PhRMA working group on Novel Adaptive Designs and co-developer of the nlme library/package in S-PLUS and R for linear and non-linear mixed-effects models.

Olga Kuznetsova is a Senior Principal Scientist in the Late Development Statistics department of Merck & Co., Inc. She has a PhD in probability theory and mathematical statistics and more than 15 years of experience in clinical trials. In the last decade, her research interests centered around randomization techniques in clinical trials, in particular, addressing the randomization needs of adaptive design trials. Her collaboration with colleagues on randomization issues resulted in more than 20 presentations and publications.

Reviews

From the book reviews:

“The target audience is anyone with an interest in planning and executing clinical trials. This includes statisticians, clinicians, pharmacometricians, clinical operation specialists, drug supply managers, and infrastructure providers working in academic research organizations, government, or industry. … This is a high quality book, written by contributors who are authorities in the field of adaptive trial design. It is an excellent addition to this field and fills a significant void.” (Ashraf Khaled AbuSara, Doody’s Book Reviews, January, 2015)

Table of contents (20 chapters)

  • The Need for and the Future of Adaptive Designs in Clinical Development

    Chuang-Stein, Christy (et al.)

    Pages 3-23

  • Regulatory Guidance Documents on Adaptive Designs: An Industry Perspective

    Pinheiro, José

    Pages 25-41

  • A Commentary on the U.S. FDA Adaptive Design Draft Guidance and EMA Reflection Paper from a Regulatory Perspective and Regulatory Experiences

    Wang, Sue-Jane

    Pages 43-68

  • Considerations and Optimization of Adaptive Trial Design in Clinical Development Programs

    Krams, Michael (et al.)

    Pages 69-90

  • Optimal Cost-Effective Go–No Go Decisions in Clinical Development

    Chen, Cong (et al.)

    Pages 91-113

Buy this book

eBook $99.00
price for USA (gross)
  • ISBN 978-1-4939-1100-4
  • Digitally watermarked, DRM-free
  • Included format: PDF, EPUB
  • ebooks can be used on all reading devices
  • Immediate eBook download after purchase
Hardcover $129.00
price for USA
  • ISBN 978-1-4939-1099-1
  • Free shipping for individuals worldwide
  • Usually dispatched within 3 to 5 business days.
Softcover $129.00
price for USA
  • ISBN 978-1-4939-5539-8
  • Free shipping for individuals worldwide
  • Usually dispatched within 3 to 5 business days.
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Bibliographic Information

Bibliographic Information
Book Title
Practical Considerations for Adaptive Trial Design and Implementation
Editors
  • Weili He
  • Jose Pinheiro
  • Olga M. Kuznetsova
Series Title
Statistics for Biology and Health
Copyright
2014
Publisher
Springer-Verlag New York
Copyright Holder
Springer Science+Business Media New York
eBook ISBN
978-1-4939-1100-4
DOI
10.1007/978-1-4939-1100-4
Hardcover ISBN
978-1-4939-1099-1
Softcover ISBN
978-1-4939-5539-8
Series ISSN
1431-8776
Edition Number
1
Number of Pages
XX, 416
Number of Illustrations and Tables
25 b/w illustrations, 41 illustrations in colour
Topics