Advanced Statistics in Regulatory Critical Clinical Initiatives

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

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.