Benefit-Risk Assessment Methods in Medical Product Development: Bridging Qualitative and Quantitative Assessments

Qi Jiang, Weili He



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.

Features

• Discusses the key elements of a structured B–R evaluation
• Addresses the regulatory environment and policies of B–R evaluations
• Examines different sources of uncertainty and delineates approaches for uncertainty quantification
• Describes how to elicit patients’ views on benefits and risks and extrapolate their views to the general patient population
• Explains how to select the appropriate B–R method for various evaluation settings
• Covers the graphical presentation of a B–R profile, including common visual displays and real examples of their application

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

Glossary

Index