Guides You on the Development and Implementation of B–R Evaluations
Benefit–Risk Assessment Methods in Medical Product Development: Bridging Qualitative and Quantitative Assessments provides general guidance and case studies to aid practitioners in selecting specific benefit–risk (B–R) frameworks and quantitative methods. Leading experts from industry, regulatory agencies, and academia present practical examples, lessons learned, and best practices that illustrate how to conduct structured B–R assessment in clinical development and regulatory submission.
The first section of the book discusses the role of B–R assessments in medicine development and regulation, the need for both a common B–R framework and patient input into B–R decisions, and future directions. The second section focuses on legislative and regulatory policy initiatives as well as decisions made at the U.S. FDA’s Center for Devices and Radiological Health. The third section examines key elements of B–R evaluations in a product’s life cycle, such as uncertainty evaluation and quantification, quantifying patient B–R trade-off preferences, ways to identify subgroups with the best B–R profiles, and data sources used to assist B–R assessment. The fourth section equips practitioners with tools to conduct B–R evaluations, including assessment methodologies, a quantitative joint modeling and joint evaluation framework, and several visualization tools. The final section presents a rich collection of case studies.
With top specialists sharing their in-depth knowledge, thought-provoking considerations, and practical advice, this book offers comprehensive coverage of B–R evaluation methods, tools, and case studies. It gives practitioners a much-needed toolkit to develop and conduct their own B–R evaluations.
Table of Contents
The Need for Benefit–Risk Assessment and Future Directions
The Need for and Future Directions of Benefit–Risk Evaluations
Neil McAuslane, Larry Liberti, and Stuart Walker
Overview of Benefit–Risk Assessment and Regulatory Environment
Regulatory and Legislative Policy and Science Considerations in the Era of Patient- Centeredness, Big Data, and Value
Tarek Hammad and George Neyarapally
Benefit–Risk Determinations at the FDA Center for Devices and Radiological Health
Telba Irony and Martin Ho
Considerations of Benefit–Risk Assessment Development in Products’ Life Cycle Management
Understanding and Evaluating Uncertainties in the Assessment of Benefit–Risk in Pharmaceutical Drug Development
Qi Jiang, Haijun Ma, Christy Chuang-Stein, Scott Evans, Weili He, George Quartey, John Scott, Shihua Wen, and Ramin Arani
Quantifying Patient Preferences for Regulatory Benefit–Risk Assessments
F. Reed Johnson and Mo Zhou
Choice of Metrics and Other Considerations for Benefit–Risk Analysis in Subgroups
Steven Snapinn and Qi Jiang
Sources of Data to Enable Benefit–Risk Assessment
Christy Chuang-Stein, George Quartey, Weili He, Qi Jiang, Haijun Ma, Jonathan Norton, John Scott, and Jesse Berlin
Benefit–Risk Assessment Methods and Visual Tools
Overview of Benefit–Risk Evaluation Methods: A Spectrum from Qualitative to Quantitative
George Quartey, Chunlei Ke, Christy Chuang-Stein, Weili He, Qi Jiang, Kao-Tai Tsai, Guochen Song, and John Scott
Benefit–Risk Evaluation Using a Framework of Joint Modeling and Joint Evaluations of Multiple Efficacy and Safety Endpoints
Weili He and Bo Fu
Visualization of Benefit–Risk Assessment in Medicinal Products with Real Examples
Shihua Wen, Weili He, Scott Evans, Haijun Ma, Christy Chuang-Stein, Qi Jiang, Xuefeng Li, George Quartey, and Ramin Arani
Benefit–Risk Assessment Case Studies and Lessons Learned
Practical Considerations for Benefit–Risk Assessment and Implementation: Vorapaxar TRA-2°P TIMI 50 Case Study
Weili He, Daniel Bloomfield, Yabing Mai, and Scott Evans
A Quantitative Benefit and Risk Assessment to Determine Optimal Retrieval Time for Inferior Vena Cava Filters in Patients without Pulmonary Embolism
Xuefeng Li, Telba Z. Irony, and Jose Pablo Morales
Benefit–Risk Assessment via Case Studies
Weili He, Qi Jiang, and George Quartey
Dr. Qi Jiang is an executive director of Global Biostatistical Science at Amgen. In this role, she is the biostatistical therapeutic area head for oncology and hematology and the lead of the Center of Excellence for Safety and Benefit–Risk. In addition, Dr. Jiang provides oversight to Amgen’s biostatistical efforts in the Asia–Pacific region. Before joining Amgen, she worked at the Harvard School of Public Health, Merck, and Novartis. Dr. Jiang is the co-editor of the Chapman & Hall/CRC book Quantitative Evaluation of Safety in Drug Development: Design, Analysis and Reporting and the author of more than 60 peer-reviewed publications on method development, study design, and data analysis and reporting. She is a fellow of the American Statistical Association, a co-lead of the American Statistical Association Biopharmaceutical Section Safety Working Group, a co-lead of the Quantitative Sciences in the Pharmaceutical Industry (QSPI) Benefit–Risk Working Group, and an associate editor for Statistics in Biopharmaceutical Research.
Dr. Weili He is a director of clinical biostatistics at Merck & Co., Inc. Her research interests include survival and longitudinal data modeling, missing data imputation, cancer Phase I and II designs, repeated categorical data modeling, surrogate marker evaluations, adaptive design methodologies and implementations, and methods for benefit–risk assessment. Dr. He has published extensively in the areas of adaptive designs and benefit–risk evaluations and is the author of more than 50 peer-reviewed publications in statistical and medical journals. She is also a lead editor of the book Practical Considerations for Adaptive Trial Design and Implementation. She has been actively involved in many professional activities and services, including serving as co-chair of the Quantitative Sciences in the Pharmaceutical Industry (QSPI) Benefit–Risk Working Group, co-chair of the DIA Adaptive Design Scientific Working Group (ADSWG) KOL lecture series, associate editor for Statistics in Biopharmaceutical Research, and a referee for other statistical journals.
"This is one of the few books fully dedicated to the benefit–risk (BR) evaluation of pharmaceutical product. . . This book was edited by two leading experts who not only have done extensive research on the topics but also have established and led the society of clinical trial BR working group. The contributing authors in each chapter are experienced researchers and/or thought-leaders in the field. We think this book gives practitioners a much needed job aid to perform their BR evaluation."
~ Journal of Biopharmaceutical Statistics