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.

section 1|1 pages

The Need for Benefit–Risk Assessment and Future Directions

chapter 1|9 pages

The Need for and Future Directions of Benefit–Risk Evaluations

ByNeil McAuslane, Lawrence Liberti, Stuart Walker

section 2|1 pages

Overview of Benefit–Risk Assessment and Regulatory Environment

section 3|1 pages

Considerations of Benefit–Risk Assessment Development in Products’ Life Cycle Management

chapter 4|13 pages

Understanding and Evaluating Uncertainties in the Assessment of Benefit–Risk in Pharmaceutical Drug Development

ByQi Jiang, Haijun Ma, Christy Chuang-Stein, Scott Evans, Weili He, George Quartey, John Scott, Shihua Wen, Ramin B. Arani

chapter 5|20 pages

Quantifying Patient Preferences for Regulatory Benefit–Risk Assessments

ByF. Reed Johnson, Mo Zhou

chapter 6|11 pages

Choice of Metrics and Other Considerations for Benefit–Risk Analysis in Subgroups

BySteven Snapinn, Qi Jiang

chapter 7|26 pages

Sources of Data to Enable Benefit–Risk Assessment

ByChristy Chuang-Stein, George Quartey, Weili He, Qi Jiang, Haijun Ma, Jonathan Norton, John Scott, Jesse A. Berlin

section 4|1 pages

Benefit–Risk Assessment Methods and Visual Tools

chapter 8|30 pages

Overview of Benefit–Risk Evaluation Methods: A Spectrum from Qualitative to Quantitative

ByGeorge Quartey, Chunlei Ke, Christy Chuang-Stein, Weili He, Qi Jiang, Kao-Tai Tsai, Guochen Song, John Scott

chapter 10|35 pages

Visualization of Benefit–Risk Assessment in Medical Products with Real Examples

ByShihua Wen, Weili He, Scott Evans, Haijun Ma, Christy Chuang-Stein, Qi Jiang, Xuefeng Li, George Quartey, Arani B. Ramin

section 5|1 pages

Benefit–Risk Assessment Case Studies and Lessons Learned

chapter 11|15 pages

Practical Considerations for Benefit–Risk Assessment and Implementation: Vorapaxar TRA-2°P TIMI 50 Case Study

ByWeili He, Daniel Bloomfield, Yabing Mai, Scott Evans

chapter 13|17 pages

Benefit–Risk Assessment via Case Studies

ByWeili He, Qi Jiang, George Quartey