Chair: Freda Cooner  (Eli Lilly)

Instructors:
Gang Li, PhD (Eisai)
Judy X. Li, PhD (BMS)
Bruce Binkowitz, PhD (Arcutis Biotherapeutics)

Course Description:

This short course will cover specific topics under the broad umbrella of multi-regional clinical trial [MRCT] development.  Both theory and case studies will be discussed.  “General Principles for Planning and Design of Multi-Regional Clinical Trials” is guidance document ICH E17 and is a very useful set of regulatory thoughts and guidances on the planning and design of MRCTs, and is readily available on the International Council for Harmonization [ICH] website.  The purpose of that guidance is to describe general principles for the planning and design of MRCTs with the aim of increasing the acceptability of MRCTs in global regulatory submissions.

The materials for this course will be taken from ICH E17 as well as two recently published books:  (1) “Multi-Regional Clinical Development After E17”, which expands on the ICH E17 guidance, providing more insight from the experts who were involved with creating ICH E17.   This book further brings examples from real MRCT trials and discusses development of methodology to improve the design and analysis of MRCTs;  (2) “Quantitative Drug Safety and Benefit Risk Evaluation – Practical and Cross-Disciplinary Approaches”, which provides a comprehensive coverage of safety monitoring methodologies, recognizing emerging global trends. With these two books as background as well as ICH E17, this short course will cover:

  • As suggested by ICH E17, a holistic approach to assessing consistency of treatment effects across regions
  • A case study of the concept of consistency under a non-inferiority study design
  • Safety in MRCTs – estimands and general methodology
  • Statistical Considerations in MRCT

Goals: Attendees will learn a broad perspective to assess consistency of regional treatment effects from both a methodology and more holistic point of view, as well as develop a deeper understanding of safety analyses and general statistical consideration in MRCTs.


Instructors:

Gang Li, PhD
Senior Director, Real World Evidence and Medical Value
Alzheimer’s Disease & Brain Health (ADBH) of Eisai Inc.

Gang LiDr. Li is responsible for developing and maintaining excellence in Epidemiology/RWE capabilities, optimizing individual and team performance, ensuring appropriate deployment to projects and other functional activities that are necessary to generate the medical evidence required by regulator, payers, and physicians to facilitate patient access and appropriate use of Eisai medicines.

Dr. Li has over 20 years of experience in the pharmaceutical industry. He co-authored over 60 publications on statistical methodologies, and research on psychiatry, obesity, and diabetes. He was an editor of the book Simultaneous Global New Drug Development – Multiregional Clinical Trials after ICH E17. Dr. Li is a Fellow of the American Statistical Association. He served as the Executive Director of the International Statistical Association (2017–2019). He received his PhD in Mathematical Statistics from the State University of New York at Binghamton.



Judy X. Li, PhD
Senior Director and Head of Lymphoma Cluster Biostatistics
Bristol Myers Squibb

Judy X. Li

Dr. Judy X. Li is the Senior Director, and Head of Lymphoma Cluster Biostatistics at Bristol Myers Squibb. Before joining the industry, she worked at the US Food and Drug Administration (FDA) as a master reviewer and supervisory mathematical statistician in multiple centers. She has broad drug development experience with regulatory agency, pharmaceutical and biotech industry, across therapeutic areas including hematology, oncology, immunology, and neurology, as well as special clinical settings for multiregional clinical trials, rare disease, and pediatric trials.

She is an editor of the book Quantitative Methodologies and Process for Safety Monitoring and Ongoing Benefit Risk Evaluation, an active member of the American Statistical Association (ASA) Biopharmaceutical Section Executive Committee, the co-chair of 2019 ASA Biopharmaceutical Section Regulatory Industry Statistics Workshop, and the founding co-chair of the ASA/DIA Safety Scientific Working Group. Dr. Li received her PhD in statistics from the University of California, Riverside, and was the recipient of the FDA Office of the Chief Scientist Intramural Research Grants Award for two consecutive years.



Bruce Binkowitz, PhD

Vice President
Biometrics at Arcutis Biotherapeutics, Inc.

Dr. Binkowitz has over 30 years of drug development experience. Prior to joining Arcutis, Dr. Binkowitz was the VP of Biometrics at Shionogi, Inc., after spending over 30 years at Merck and Co. He has worked across all phases of drug development and across many therapeutic areas including dermatology, oncology, respiratory, metabolism/diabetes, cardiovascular, and ophthalmology. Dr. Binkowitz has presented at major statistics conferences and has publications spanning more than 30 years, including his contributions as a thought leader on the topic of multiregional clinical trials, which culminated in his being co-editor of the textbook “Simultaneous Global New Drug Development”.

Dr. Binkowitz is a fellow of the American Statistical Association (ASA), was the 2020 Chair of the Biopharmaceutical Section of the American Statistical Association and was awarded the 2022 Distinguished Alumni Award from the Rutgers School of Public Health.  Dr. Binkowitz has a PhD from the Rutgers School of Public Health, a Master’s degree in Statistics from the University of Florida, and a Bachelor’s degree in Mathematics/Statistics from Rutgers College.