1st Edition

Advanced Statistics in Regulatory Critical Clinical Initiatives

Edited By Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow Copyright 2022
    317 Pages 30 B/W Illustrations
    by Chapman & Hall

    317 Pages 30 B/W Illustrations
    by Chapman & Hall

    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.

    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

    Biography

    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.

    "This is a must have book for any statistician working in the pharmaceutical industry that wants to have an introductory overview of the most recent and challenging issues and ongoing discussions in the field of statistical regulatory science."

    David Manteigas, Cmed Clinical Services, Portugal, ISCB, February 2023