Statistical Methods in Biomarker and Early Clinical Development

Description

This contributed volume offers a much-needed overview of the statistical methods in early clinical drug and biomarker development. Chapters are written by expert statisticians with extensive experience in the pharmaceutical industry and regulatory agencies. Because of this, the data presented is often accompanied by real world case studies, which will help make examples more tangible for readers. The many applications of statistics in drug development are covered in detail, making this volume a must-have reference. Biomarker development and early clinical development are the two critical areas on which the book focuses. By having the two sections of the book dedicated to each of these topics, readers will have a more complete understanding of how applying statistical methods to early drug development can help identify the right drug for the right patient at the right dose. Also presented are exciting applications of machine learning and statistical modeling, along with innovative methods and state-of-the-art advances, making this a timely and practical resource. This volume is ideal for statisticians, researchers, and professionals interested in pharmaceutical research and development. Readers should be familiar with the fundamentals of statistics and clinical trials. Front Matter ....Pages i-xii Predictive Subgroup/Biomarker Identification and Machine Learning Methods (M. Man, T. S. Nguyen, C. Battioui, G. Mi)....Pages 1-22 Characterize and Dichotomize a Continuous Biomarker (G. Mi, W. Li, T. S. Nguyen)....Pages 23-38 Surrogate Biomarkers (Yongming Qu)....Pages 39-52 Innovative Designs for Biomarker-Guided Trials (Jing Wang, Mark Chang)....Pages 53-65 Statistical Considerations in the Development of Companion Diagnostic Device (Meijuan Li, Rui Tang)....Pages 67-86 Biomarker Assay Development, Qualification, and Validation (Guang Chen, Shuguang Huang, Winnie Weng, Cheng Su)....Pages 87-115 Validation of Genomic-Based Assay (Yoonha Choi, Jing Huang)....Pages 117-136 Clinical Application of Molecular Features in Therapeutic Selection and Drug Development (Ruixiao Lu, Rui Tang, Jing Huang)....Pages 137-166 Big Data, Real-World Data, and Machine Learning (Jing Lu, Yangyang Hao, Jing Huang, Su Yeon Kim)....Pages 167-195 Design and Analysis of Clinical Pharmacology Studies (Winnie Weng, Liang Fang)....Pages 197-220 Statistical Considerations in Proof-of-Concept Studies (Laurence Colin, Brian Smith)....Pages 221-245 Safety in Early Phase Studies (Laurence Colin, Brian Smith)....Pages 247-274 Statistical Evaluation of QT/QTc Interval Prolongation (Liang Fang, Winnie Weng, Lu Wang, Xiaoru Wu)....Pages 275-299 Phase II Dose Finding (Qiqi Deng, Naitee Ting)....Pages 301-320 Pharmacometrics (Alan H. Hartford, Kenneth G. Kowalski)....Pages 321-348