SHORT COURSE 2 (Dec 8-Dec 9)

TITLE: Multi-regional Clinical Development and Safety/Benefit-Risk evaluation 
INSTRUCTOR: Bruce Binkowitz, Aloka Chakravarty, Arcutis Biotherapeutics Inc, Gang Li, Eisai Inc, Jim Buchanan, Covilance LLC, and Judy Li, BMS
MODERATOR: Ivan S. F. Chan and William Wang


This two-day short course will cover the topics of safety/ benefit risk assessment and multi-regional clinical development.  The short course will be based on two recently published books:  (1) The book: “Multi-Regional Clinical Development After E17”  expands on the ICH E17 guidance, with more insight from the experts who were involved with creating ICH E17.   It further  brings examples from real MRCT trials and discusses development of methodology to improve the design and analysis of MRCTs; (2) The book: “Quantitative Drug Safety and Benefit Risk Evaluation – Practical and Cross-Disciplinary Approaches” provides a comprehensive coverage of safety monitoring methodologies, recognizing emerging global trends. Pharmacovigilance during clinical development has traditionally focused on the handling of individual adverse event reports; however, recently there has been a shift towards emphasizing aggregate analysis on an ongoing basis to better understand the scope of product risks.

Even though these two books focus on different perspectives of drug development,  in this two-day short course we will connect them by the same clinical development case studies.  In particular,  we plan to use a cardiovascular drug development program to illustrate the concepts.  A workshop style discussion will also be arranged on Day 2 on these case studies.

This is a short course for both statisticians and non-statisticians.  Cross disciplinary audience  participation will be highly encouraged..

Outline of the Short Course:
Day 1 Morning: MRCT and ICH E17   
⦁ ICH E17:  Seven (7) principles of Good MRCT Design and Explanation in Each of Them
⦁ Statistical Methodologies for Sample Size and Consistency

⦁ Case Studies in  Therapeutic Areas such as CV and Oncology

Day 1 Afternoon: Safety Monitoring and Evaluation   
⦁ Regulatory Landscape in Safety Monitoring
⦁ Aggregate Safety Assessment Planning,  Safety Signaling and Evaluation, including the use of open-source Interactive Safety Graphics
⦁ Safety Monitoring Methodologies in RCT and RWE
Day 2 Morning:  Case Based Examination on MRCT Consistency and Benefit-Risk Evaluation
⦁ Introduction of the Case Studies in Type II Diabetes
⦁ Statistical Methodologies for Pooling and Consistency
⦁ Benefit-risk Evaluation and Visualization
Day 2 Afternoon:  Workshop Presentation and Panel Discussion 
⦁ Break up for attendees into  subgroup with pre-arranged discussion topics
⦁ Presentations by each of the  working groups
⦁ Panel discussion

Instructors’ Biography:

Dr. Bruce Binkowitz is the Vice President of Biometrics at Arcutis Biotherapeutics Inc. Bruce has more than 30 years of pharmaceutical industry experience across many therapeutic areas as well as from early phase through phase IV clinical trials. He has experience in study design, conduct, analysis, and interpretation of results for clinical trials, as well as many interactions with health authorities worldwide. Dr. Binkowitz is active in the Statistical Community. Bruce is currently in his second term as co-leader of the cross-industry Multi-Regional Clinical Trial Consistency Expert Group. Dr. Binkowitz was elected a fellow of the American Statistical Association in 2015, and was awarded the 2022 Distinguished Alumni Award by the Rutgers School of Public Health.



Dr. Gang Li is Senior Director, Real World Evidence (RWE) and Medical Value, the Neurology Business Group of Eisai Inc. He received his PhD in Mathematical Statistics from the State University of New York at Binghamton. He co-authored over 60 publications on statistical methodologies, and research on psychiatry, obesity, and diabetes. He served as the Executive Director of the International Statistical Association (2017–2019). Dr. Li is a Fellow of the American Statistical Association.





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 entry was posted in . Bookmark the permalink.