May 7, 2022
9:00 PM – 1:00 PM CT

Innovative Designs for Early Phase Oncology Trials

Yuan Ji, The University of Chicago Pritzker School of Medicine, Professor of Biostatistics

Sue-Jane Wang, US FDA

In this half-day short course, we will introduce, describe, and demonstrate innovative designs for early-phase oncology trials. The classroom teaching will be delivered by the two instructors through projected slides. There will be two sessions with a Q&A portion in each session. One session will be taught by Dr. Sue-Jane Wang. She will recap types of methodological advances in light of recent regulatory experiences with use of early phase innovative designs. The other session will be taught by Dr. Yuan Ji, who will present novel statistical designs for phase 1a dose escalation, phase 1b expansion cohorts, drug combination dose finding, designs accommodating delayed toxicity like in immune oncology, and designs for early-phase basket trials. Most designs introduced in the short course will use Bayesian modeling and adaptive decision rules. A brief introduction of Bayesian statistics will also be provided. Lastly, some software packages will be briefly illustrated.

Outline of Topics:
Session 1 (2 hours, Ji): Methodologies and applications (I)
  1. Brief introduction of Bayesian modeling and inference
  2. Designs for phase 1a dose escalation studies
  3. Designs for phase 1b cohort expansion studies
Q&A and break (30 minutes)

Session 2 (1 hour, Wang): Regulatory consideration and rationales
  1. Regulatory guidance overview related to early phase dose finding/cohort expansion/adaptive design/master protocol/new drug combinations
  2. Key methodologies recap and some regulatory experiences in early phase design strategies
Session 3 (1 hour, Ji): Methodologies and applications (II)
  1. Designs for dose escalation studies with late-onset toxicity-outcome
  2. Designs for combination dose-escalation studies


Yuan Ji

The University of Chicago Pritzker School of Medicine, Professor of Biostatistics


Sue-Jane Wang


sue-jane_wang Sue-Jane Wang is Biomarker Lead of FDA/CDER Biomarker Qualification Program, Office of Biostatistics (OB) in the Office of Translational Sciences, CDER, US FDA. She is also a Deputy Director from one of the OB divisions. Dr. Wang has joined FDA for more than 20 years. Currently, she facilitates the Biometrics Division that provides regulatory statistical services to neurology, psychiatry, anesthesiology/addition/pain medicines, and medical imaging drug product developments. In her roles, Dr. Wang has been contributing to FDA guidance developments as an Office Lead or a guidance working group member including, e.g., pre-market evaluation on clinical pharmacogenomics, adaptive design, enrichment strategies, drug development tools, co-development of an In Vitro companion diagnostic device with a therapeutic product, multiple endpoints, analytical validation of biomarker for qualification, evidential framework for biomarker qualification, and surrogate endpoints guidance. She is a member of FDA-NIH Biomarker Working Group. Dr. Wang has been active in complex trial design, pharmacogenomics, biomarker, diagnostic imaging, theranostics research and professional editorship. She has been awarded for her professional recognitions including, e.g., a Fellow of the American Statistical Association, an FDA Level Scientific Achievement (Individual) Award on Excellence in Analytical Science.