SHORT COURSE 2 (Dec 10-Dec 11)

In new drug development, the goal is to generate the strongest possible evidence—most often through rigorously designed clinical trials such as randomized controlled trials (RCTs)—to support regulatory approval. Alongside these trials, external data sources—such as real-world data (RWD) from insurance claims, electronic health records, and longitudinal observational studies—and real-world evidence (RWE) generated from RWD are increasingly used to support the design, conduct, and evidence synthesis of clinical trials.

This short course aims to provide systematic and integrated approaches for use of RWD and RWE throughout the clinical development of medicinal products, effectively bridging the methodologies of traditional RCTs and modern RWE studies. While grounded in the general statistical concerns of clinical trials within regulatory frameworks, the course incorporates innovative concepts—such as single-arm trials, externally controlled trials, rare disease drug development, and treatment switching—and modern tools—including digital health technologies, artificial intelligence, and machine learning—to address emerging trends in the design and conduct of clinical trial methodologies.

Outline of the short course:

Day 1 Morning:

• Introduction
• A brief history and critical components of clinical trials
• Clinical development process of a new drug

Day 1 Afternoon:

• Design considerations for Phase III confirmatory trials
• Regulatory submission and approval
• An overview of the use of RWD & RWE in regulatory setting
• Single-arm trials

Day 2 Morning:

• Externally controlled trials
• Master protocols
• Drug development for rare diseases
• Time to event analysis with treatment switching

Day 2 Afternoon:

• Precision medicine
• Sensitivity analysis in clinical trials
• Estimands in RWE studies
• Artificial intelligence in clinical studies

Instructors’ Biography:

Jie Chen, PhD, is the Chairman & Chief Scientific Officer in Taimei Intelligence Biopharma R&D, Inc. Dr. Chen has over 30 years of experience in biopharmaceutical research, development, and life-cycle management. He has contributed to the design, conduct, analysis, and regulatory submission of more than 50 new drug clinical trials, leading to regulatory approvals of over 10 drugs across diverse therapeutic areas. His primary research interests focus on adaptive trial design, multiple comparisons, Bayesian methods, drug safety evaluation, and the application of real-world evidence (RWE), artificial intelligence/machine learning, and causal inference to support medical product development and regulatory decision-making. He has co‑authored or edited three books: Medical Product Safety Evaluation—Biological Models and Statistical Methods (Chapman & Hall/CRC, 2019), Real-World Studies—From Real-World Data to Real-World Evidence (China Medical Technology Publishing House, 2025), and Clinical Trials in the Era of Real-World Evidence (Wiley, 2026). Dr. Chen has published more than 60 peer-reviewed papers. He is a Fellow of the American Statistical Association.

Naitee Ting is a Fellow of American Statistical Association (ASA).  Naitee is the recipient of the prestigious Mentoring Award of ASA in 2025.  He is currently Vice President of Veramed.  Naitee is also an Adjunct Professor of Department of Statistics at University of Connecticut, Adjunct Professor of Department of Biostatistics at Columbia University.  He joined Veramed in 2025.  Before Veramed, Naitee has been with Boehringer Ingelheim Pharmaceuticals, Inc. (BI) for 15 years, and he was working at Pfizer Inc. for 22 years (1987-2009).  Naitee received his Ph.D. in 1987 from Colorado State University (major in Statistics).  He has an M.S. degree from Mississippi State University (1979, Statistics) and a B.S. degree from College of Chinese Culture (1976, Forestry) at Taipei, Taiwan.

Naitee published articles in Technometrics, Statistics in Medicine, Drug Information Journal (Therapeutic Innovation and Regulatory Science), Journal of Statistical Planning and Inference, Journal of Biopharmaceutical Statistics, Biometrical Journal, Statistics and Probability Letters, Statistics in Biosciences, Statistics in Biopharmaceutical Research, and Journal of Statistical Computation and Simulation.  His book “Dose Finding in Drug Development” was published in 2006 by Springer, and is considered as the leading reference in the field of dose response clinical trials.  The book “Fundamental Concepts for New Clinical Trialists”, co-authored with Scott Evans, was published by CRC in 2015.  Another book “Phase II Clinical Development of New Drugs”, co-authored with Chen, Ho, and Cappelleri  was published in 2017 (Springer).  Naitee has been an active member of both the ASA and the International Chinese Statistical Association (ICSA).

Feinan Lu is Director of Biometrics at IMPACT Therapeutics. She has over 10 years of experience in biopharmaceutical research and drug development. She has extensive expertise in clinical trial design, statistical analysis, and global regulatory submissions across major health authorities, including the NMPA, FDA, and EMA, with experience spanning more than 10 pivotal clinical trials.

Her primary research interests focus on oncology clinical trial design, including adaptive designs, single-arm trials, and centralized statistical monitoring. Feinan has been an invited speaker at industry conferences and has co-authored multiple publications on oncology trial design and single-arm studies. She is also a co-author of the book Clinical Trials in the Era of Real-World Evidence (Wiley, 2026).