December 4-8, 2017 at Atlantic City, New Jersey
The 73rd Deming Conference on Applied Statistics
Sponsored by Metropolitan Section, ASQ and Biopharmaceutical Section, ASA

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12/5/2016 (Monday) 1:30 PM - 4:30 PM

Session D: Benefit-risk assessment methods in medical product development: bridging qualitative and quantitative assessments

Dr. Qi Jiang, Amgen
Dr. Weili He, Merck

Moderator: Alfred Balch
Book: Qi Jiang, Weili He. Benefit-Risk Assessment Methods in Medical Product Development: Bridging Qualitative and Quantitative Assessments, by Chapman and Hall/CRC, June 2016:

Evaluation of a new treatment has always required a benefit-risk (B-R) assessment. However, in the past the assessment tended to be informal and was often subjective, involving judgments from separate assessments of efficacy and safety. Notably, a few regulatory submissions in the last few years failed to gain regulatory approval due to benefits that did not outweigh risks. Such failures speak to the importance of structured B-R assessments to improve consistency, transparency, communication, and objectivity. However, B-R assessment is multi-faceted and complex, and the B-R landscape is still evolving. There is increased interest and effort from companies, regulatory agencies, and other governance bodies to further enhance structured B-R assessments. Amid numerous existing frameworks, metrics, estimation techniques, and utility survey techniques in B-R assessment, however, there is still no commonly accepted B-R approach. Guidance on how to select specific B-R frameworks and quantitative methods, along with case studies and best practice sharing, is still lacking. This tutorial will provide general guidance and case studies to aid statisticians in selecting specific benefit–risk frameworks and quantitative methods. In addition, we will 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 part of the tutorial covers the role of B–R assessments in medicine development and regulation, along with key elements of B–R evaluations in a product’s life cycle. The second part presents visual tools for B-R evaluations along with a couple of illustrative case studies for lessons learned and best practice sharing.

Dr. Qi Jiang is an executive director of Global Biostatistical Science at Amgen. In this role, she is biostatistical therapeutic area head for oncology and hematology and is the lead of the Center of Excellence for Safety and Benefit-Risk. In addition, Qi provides oversight to Amgen’s biostatistical efforts in the Asia-Pacific region. Before joining Amgen, Qi worked at the Harvard School of Public Health, Merck, and Novartis. Dr. Jiang is an editor of a book “Quantitative Evaluation of Safety in Drug Development” and also an editor of a book “Benefit-Risk Assessment Methods in Medical Product Development: Bridging Qualitative and Quantitative Assessments”. In addition, Dr. Jiang is 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. Her research interests include survival and longitudinal data modeling, missing data imputation, cancer Phase I & 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 an editor of the book, “Practical Considerations for Adaptive Trial Design and Implementation”, published by Springer in 2014, and the book, “Benefit-Risk Assessment Methods in Medical Product Development: Bridging Qualitative and Quantitative Assessments”, published by CRC Press in 2016. She has also been involved in many professional activities and services, including serving as co-chair of the QSPI Benefit-Risk working, co-chair of the DIA ADSWG KOL lecture series, co-lead of the DIA ADSWG Best Practice Subteam until January 2016, Associate Editor for the journal of Statistics in Biopharmaceutical Research (SBR) and a referee for other statistical journals.

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