Advanced Statistics in Regulatory Critical Clinical Initiatives  book cover
1st Edition

Advanced Statistics in Regulatory Critical Clinical Initiatives

ISBN 9780367561789
Published May 26, 2022 by Chapman and Hall/CRC
317 Pages 30 B/W Illustrations

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Book Description

Advanced Statistics in Regulatory Critical Clinical Initiatives is focused on the critical clinical initiatives introduced by the 21st Century Cure Act passed by the United States Congress in December 2016. The book covers everything from the outline of the initiatives to analysis on the effect on biopharmaceutical research and development. Advanced Statistics in Regulatory Critical Clinical Initiatives provides innovative ways to resolve common challenges in statistical research of rare diseases such small sample sizes and provides guidance for combined use of data. With analysis from regulatory and scientific perspectives this book is an ideal companion for researchers in biostatistics, pharmaceutical development, and policy makers in related fields.

Key Features:

  • Provides better understanding of innovative design and analysis of each critical clinical initiatives which may be used in regulatory review/approval of drug development.
  • Makes recommendations to evaluate submissions accurately and reliably.
  • Proposes innovative study designs and statistical methods for oncology and/or rare disease drug development.
  • Provides insight regarding current regulatory guidance on drug development such as gene therapy and rare diseases.

Table of Contents

About the Editors

List of Contributors

Chapter 1 Introduction   

Wei Zhang, Fangrong Yan, Feng Chen, and Shein-Chung Chow

Chapter 2 Complex Innovative Design   

Beibei Guo and Ying Yuan

Chapter 3 Validation Strategy for Biomarker guided Precision/Personalized Medicine

Hou, Y., Li, M.J., and Li, K.

Chapter 4 Model-Informed Drug Development 

Haochen Liu, Fangrong Yan

Chapter 5 Real-world data and real-world evidence     

Xin Sun

Chapter 6 AI/ML in Medical Research and Drug Development 

Amir Nikooienejad and Haoda Fu

Chapter 7 Challenges in Cancer Clinical Trials   

Xia, Fan; Lin, Xiao; Guo, Xiang

Chapter 8 Statistical Methods for Assessment of Biosimilars   

Yafei Zhang, Vivian Gu, Xiuyu Julie Cong, and Shein-Chung Chow

Chapter 9 Statistical Methods for Assessment of Complex Generic Drugs

Peiyong Yu, Xiaoyan Yan and Chen Yao       

Chapter 10 Rare Diseases Drug Development     

Shein-Chung Chow, Shutian Zhang, Wei Zhang

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Wei Zhang is the Vice President, Head of Biometrics at AffaMed Therapeutics, New York.

Fangrong Yan is a Professor of Biostatistics in the School of Science, and Director of Biostatistics and Computational Pharmacy Research Center at China Pharmaceutical University (CPU).

Feng Chen is a Professor of Biostatistics in School of Public Health, Nanjing Medical University (NJMU).

Shein-Chung Chow is currently a Professor at the Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham.