TITLE: Bayesian Adaptive Statistical Approaches for Accelerating Drug and Medical Device Approvals in Rare Disease
SPEAKERS: Margaret Gamalo, Jinma Ren, Wenjin Wang, Pfizer Inc
MODERATOR: Li-An Xu
Abstract:
This tutorial is divided into three parts, with a duration of three hours. In the first part, we will introduce model-assisted dose-finding design methods used to identify the maximum tolerated dose (MTD), and the SEARS (Seamless Phase I/IIa with Early Analysis for Response and Safety) framework, which helps identify the optimal biological dose (OBD). We will demonstrate how to implement these methods using the BOIN and SEARS R packages. Moving on to the second part, we will discuss two-stage designs for single-arm phase IIa trials, including Simon’s two-stage design for binary endpoints and its extension for time-to-event endpoints. We will also demonstrate how to implement these methods using the clinfun and OneArm2Stage R packages.
In the third and final part of the tutorial, we will introduce platform design methods. We will discuss the concept of multi-arm design and present an optimal platform design. Using the PlatformDesign R package, we will demonstrate how to implement these methods. By the end of this tutorial, you will have gained a comprehensive understanding of model-assisted dose-finding, two-stage designs for single-arm phase IIa trials, and platform design methods. You will also be equipped with the skills to implement these methods using the R packages BOIN, SEARS, clinfun, OneArm2Stage, and PlatformDesign.
Instructors’ Biography:
Dr. Haitao Pan is an Associate Professor of Biostatistics at St. Jude Children’s Research Hospital. His research is primarily focused on developing innovative clinical trial designs, particularly for oncology, from early to late phases.
Since joining St. Jude in 2017, Dr. Pan has served as the Primary Study Statistician for 32 St. Jude investigator-initiated clinical trials and is a member of the Clinical Trials – Scientific Review Committee at St. Jude. He has authored over 50 publications in peer-reviewed journals, spanning across medical and statistical journals, and has published a book through Springer Nature.
Dr. Pan has also developed 16 R software packages, with a specific focus on adaptive clinical trial design and sample-size calculation. Many of these have been used to develop clinical trial protocols at St. Jude. Additionally, Dr. Pan is an adjunct faculty member at Florida State University and The University of Memphis.
Dr. Pan holds two Ph.D. degrees: one in Preventive Medicine from China, and another in Biostatistics from MD. Anderson Cancer Center.