There has been considerable interest among pharmaceutical and other medical product developers in adaptive clinical trials, in which knowledge learned during the course of a trial affects ongoing conduct or analysis of the trial. Following the release of the FDA draft Guidance document on adaptive design clinical trials in early 2010, expectations of an increase in regulatory submissions involving adaptive design features, particularly for confirmatory trials, were high. There are indeed concerns regarding the statistical issues and operational challenges in conducting adaptive design clinical trials. We will share our experiences in the reviews of adaptive design proposals, including surveys performed regarding regulatory submissions of adaptive design proposals as well as case studies which have been reviewed. We will also provide general recommendations for developing proposals for such trials. Our motivation in instructing this short course is to encourage the best study design proposals to be submitted to FDA. Sometimes these can be adaptive and sometimes a simpler design is most efficient.
Shiowjen Lee, Ph.D.
Shiowjen Lee, Ph.D. is currently Team Leader in Center for Biologics Evaluation and Research (CBER), US FDA. In her current role, Dr. Lee oversees regulatory submissions supporting the biologic products regulated by Office of Cellular, Tissue and Gene Therapy (OCTGT). She has been with FDA for more than 15 years. Prior to the current role, she has worked at Center for Drug Evaluation and Research (CDER) and Center for Devices and Radiological Health (CDRH) as a Mathematical Statistician in reviews of regulatory submissions. In her work, she has been reviewing a wide range of regulatory applications including cancer vaccines, anti-inflammatory products, products (including biologic and devices) to treat cardiovascular disease, rare disease and many others. She has expertise in both statistics and clinical trial design and has broad experiences in products regulated at different Centers in FDA. Dr. Lee’s work in FDA has also included developing regulatory guidance and policies regarding specific indications, statistical issues and regulatory submissions.
Dr. Lee has been a recipient for numerous awards at Center and FDA levels in FDA including Outstanding Service award, Team Excellence award, Center Director’s Award for Improving Communication, Managerial Excellence award, and many others. She has a great ability collaborating and communicating statistics to review teams and non-statisticians. Last year, she was a Co-Chair of 2014 FDA/Industry Statistics Workshop that was held at Washington DC.
Min (Annie) Lin, Ph.D.
Dr. Lin joined the Division of Biostatistics at Center for Biologics Evaluation and Research (CBER), US FDA in 2011 as a Mathematical Statistician with special focus on Adaptive Designs. In her current role, Dr. Lin performs critical statistical reviews of regulatory pre-marketing submissions to the Office of Cellular, Tissue and Gene Therapy (OCTGT) and the Office of Blood Research and Review (OBRR), and provides statistical trainings to non-statisticians within CBER. She supervises the FDA interns in conducting biostatistical methodology researches in the area of adaptive designs that are related to CBER regulated biologics products and medical devices. Dr. Lin also serves as a specialist in developing FDA Draft Guidance on Adaptive Designs for Medical Device Clinical Studies and CBER division guidelines.
Prior to joining the FDA, Dr. Lin was an Assistant Professor in the Department of Biostatistics and Bioinformatics at Duke University School of Medicine. She has served as statistical investigator/co-investigator in various pharmaceutical-funded and government-funded studies for a wide range of therapeutic areas.
