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.
- 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
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
"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