ACDRS 2021 Session 4: Statistics: Design, Analysis and Interpretation of Clinical Trials

Monday, September 27, 2021 - 10:00 am to Friday, October 1, 2021 - 3:00 pm

This is Session 4 of 6. Must register for the entire course and not per Session.

Session Chairperson: Stephen Ruberg, PhD, President, Analytix Thinking


Bruce S. Binkowitz, PhD

Vice President, Biometrics, Arcutis

Gregory Campbell, PhD

President, GCStat Consulting LLC; former Director, Biostatistics Division, Center for Devices and Radiological Health, US Food and Drug Administration (FDA)

Daniel Meyer, PhD

Head of Statistics, Rare Disease Research Unit, Pfizer
Richard Payne, PhD  Research Scientist, Eli Lilly and Company
William R. Prucka, PhD Principal Research Scientist, AdvAn Science Driven Adaptive Program, Eli Lilly and Company
Frank W. Rockhold, PhD Professor of Biostatistics and Bioinformatics, Duke Clinical Research Institute, Duke University School of Medicine

Stephen J. Ruberg, PhD

President, Analytix Thinking LLC

Kert Viele, PhD

Senior Statistical Scientist, Berry Consultants

Additional faculty - case study proctors

Jesse Anderson, MPH Team Lead, Clinical Services, Office of Computational Science, US FDA
Erica Berzin, MS Regulatory Affairs Director, NDA Partners
Dean Bottino, PhD Senior Scientific Director, Clinical Translational Modeling & Simulation Leader, Takeda Oncology
Salvador Garcia, MD Senior Medical Director ANZ & NAPAC, Eli Lilly and Company
Charlie T. Gombar, PhD ACDRS Director
Diane K. Jorkasky, MD Consultant to Pharma
Karryn Lepley, MBA PMP Associate Director, Portfolio Management, AbbVie
Karolynn Leon, MA Executive Director, Global Program Management, Novartis
Dustin Little, MD Global Clinical Lead, Renal, AstraZeneca
Carl Peck, MD Adjunct Professor, UCSF; former Director, Center for Evaluation and Research, US FDA
Melissa Reyes, MD MPH DTMH Medical Officer, Division of Pharmacovigilance, Office of Surveillance and Epidemiology, US FDA
Becca Saenz, MD PhD Medical Director, Respiratory and Allergic Group, Genentech
Nilam Shah, PharmD RPh Senior Director, Clinical Research and Development, Cara Therapeutics
Alice Wang, MD Director, Translational Development, Clinical Pharmacology, Bristol Myers Squibb
Daniel Weiss, MD Vice President, Early Clinical Development, Schrodinger 


  • Role of confirmatory phase in drug development
  • Statistical principles for clinical trials
  • Choice of control groups in clinical trials
  • Study objectives, hypothesis testing, sample size, and probability of study success
  • Multiplicity in clinical trials
  • Missing data and estimands
  • Interim analysis
  • Bayesian statistics
  • The design, analysis, and interpretation of a clinical endpoint trial
  • Modeling and simulation
  • Adaptive design in clinical trials and optimization
  • Design and analysis of adaptive clinical trials
  • Enrichment designs
  • Perspectives for developing a targeted medicine
  • Co-development of a diagnostic and targeted medicine
  • Statistical issues related to design, analysis, and reporting of gene therapy clinical trials
  • Multi-regional clinical trials
  • Safety assessment and surveillance
  • Digital health innovations