Stat4Onc 2019 Symposium Short Courses


April 25, 2019             Morning courses         8:30 AM to 12:00 noon          with one break

                                     Afternoon courses      1:30 PM to 5:00 PM               with one break


Course 2 (Morning Course)  Review of Early Phase Trial designs with Recent Development for Precision Oncology


Instructor – Professor Ying Kuen K. Cheung, Columbia University






Applications in early phase cancer trials have motivated the development of many statistical designs since the late 1980s, including futility screening, dose finding methods, treatment selection, and early stopping rules.  These methods are often proposed to address the conventional cytotoxic therapeutics for neoplastic diseases and cancer.  In contrast, recent advances in precision medicine have motivated novel trial designs, most notably the idea of master protocol (e.g., basket trial, platform trial, “N-of-1”, etc.), for the evaluation of molecularly targeted cancer therapies.  In this short course, we will have a quick review of trial concepts from these two sets of literature.  We will discuss when and how the designs and the principles developed for cytotoxic agents may be relevant for targeted cancer therapies.  We will also suggest open design questions that call for further research.




Ying Kuen (Ken) Cheung, PhD, is Professor and Interim Chair of Biostatistics in the Mailman School of Public Health at Columbia University.  He has general interests in the development and evaluation of evidence-based treatments, interventions and policies at all phases of translational research.  An overarching goal of his research and professional activities is to advance precision medicine using data science and biostatistical methods.  He led a schoolwide task force on precision medicine that identified priority areas crucial to the advances of the mission of precision public health.  He is also an expert in experimental designs, adaptive designs in clinical trials of treatments for cancer, stroke, and other neurological disorders, SMART designs for behavioral intervention technologies, N-of-1 personalized trials, implementation study designs, and the analysis of high dimensional behavioral data.  He is a Fellow of the American Statistical Association, and a Fellow of the New York Academy of Medicine.  He is also a recipient of IBM Faculty Award on Big Data and Analytics.