1st Edition

Quantitative Drug Safety and Benefit Risk Evaluation Practical and Cross-Disciplinary Approaches

Edited By William Wang, Melvin Munsaka, James Buchanan, Judy Li Copyright 2022
    402 Pages 33 B/W Illustrations
    by Chapman & Hall

    402 Pages 33 B/W Illustrations
    by Chapman & Hall

    402 Pages 33 B/W Illustrations
    by Chapman & Hall

    Quantitative Methodologies and Process for Safety Monitoring and Ongoing Benefit Risk Evaluation provides a comprehensive coverage on safety monitoring methodologies, covering both global trends and regional initiatives. Pharmacovigilance has traditionally focused on the handling of individual adverse event reports however recently there had been a shift towards aggregate analysis to better understand the scope of product risks.

    Written to be accessible not only to statisticians but also to safety scientists with a quantitative interest, this book aims to bridge the gap in knowledge between medical and statistical fields creating a truly multi-disciplinary approach that is very much needed for 21st century safety evaluation.

    Section A:  Regulatory Landscape and Interdisciplinary Collaboration in Safety Evaluation 

    ●        Chapter 1: The Emergence of Aggregate Safety Assessment Across the Regulatory Landscape 

    ●        Chapter 2: Aggregate Safety Assessment Planning in Clinical Development

    ●        Chapter 3: Safety Signaling and Causal Evaluation

    ●        Chapter 4: Safety Monitoring through External Committees

    Section B: Statistical Methodologies for Safety Monitoring

    ●        Chapter 5: An Overview on Statistical Methodologies for Safety Monitoring and Benefit-Risk Assessment

    ●        Chapter 6: Quantitative Methods for Blinded Safety Monitoring

    ●        Chapter 7: Bayesian Safety Methodology

    ●        Chapter 8: Likelihood-Based Methods for Safety Monitoring

    ●        Chapter 9: Meta-Analysis for Drug Safety Assessment


    Section C:  Design and Analysis Considerations in RCT and RWE for Safety Decision Making

    ●        Chapter 10: Design consideration for pragmatic trials with insight from cardiovascular outcome trials

    ●        Chapter 11:  Post-market Safety Assessment Using Observational Studies and the FDA Sentinel System

    ●        Chapter 12: Analysis Considerations for Real-World Evidence and Clinical Trials Related to Safety 


    Section D:  Safety/Benefit-Risk Evaluation and Visualization

    ●        Chapter 13: Trends and Recent Progress in Benefit-Risk Assessment Planning for Medical Products and Devices

    ●        Chapter 14: Estimands in Safety and Benefit Risk Evaluation

    ●        Chapter 15: Visual Analytics for Safety and Benefit Risk Evaluation


    Dr. William (Bill) Wang is currently Executive Director in the department of Biostatistics and Research Decision Sciences, Merck Research Laboratories, where he has worked for 20+ years.  Bill has served as the deputy topics-leader for the ICH E17 Working Group  and has co-chaired the ASA Biopharmaceutical Safety Working Group.  He is an elected ASA Fellow.


    Dr. Melvin S. Munsaka is currently Senior Director and Head of Safety Statistics at AbbVie. He has more than 25 years of industry experience. He is a member of the ASA Biopharmaceutical Safety Scientific Working Group, DIA Bayesian Scientific Working Group, and the PHUSE Safety Analytics Working Group


    Dr. James Buchanan, president of Covilance LLC, has 30+ years of drug safety experience in the pharmaceutical industry. He is a co-lead of the ASA Biopharmaceutical Safety Working Group and the Interactive Safety Graphics taskforce that is developing novel, open-source, interactive graphical tools to evaluate drug safety issues.


    Dr. Judy X. Li is currently Senior Director, Biostatistics Lead for San Diego site Bristol Myers Squibb. She also has extensive experience working at the US Food and Drug Administration as a master statistical reviewer and supervisory mathematical statistician. She is the founding co-chair of the ASA Biopharmaceutical Safety Working Group.

    "This is an amazing book for anyone working in the clinical research industry and does not require at all extensive knowledge of statistics or prior exposure to statistics at all. In fact, I would say that the target audience is broader than just statisticians but including instead everyone working in the clinical research industry involved in study planning, analysis and/or reporting."

    - David Manteigas, ISCB News, September 2022.

    "Overall, this book provides thorough background andknowledge about clinical considerations and regulatory policies, as well as a comprehensive review of frequently used safety-specific statistical methods with clear and thoughtful explanations... Therefore, I strongly recommend this book to all who eithe ralready work on clinical studies or who are new to the area and are interested in clinical trial planning and analyses."

    - Huan Wang, The American Statistician, January 2023.