In the process of medicine discovery and development, understanding the dose-response relationship is one of the most important and challenging tasks. It is critical to identify the right range of doses in early stages of medicine development so that Phase III trials can be properly designed to confirm appropriate dose(s) for the patient. Usually in the beginning of Phase II development, there is limited information to help guide trial designs, therefore Phase II clinical trials often consists of objectives for establishing proof of concept (PoC), identifying a set of potentially safe and efficacious doses, and characterizing the dose-response relationship.
Some of the major challenges in designing these Phase II trials include the selection of dose range and frequency, clinical endpoints and/or biomarkers, and the use of control(s). Inappropriate Phase II trial designs may lead to delay of the medicine development program or waste of investment. Specifically, misleading results from poorly designed Phase II trials could force a Phase III program to confirm sub-optimal dose(s), or even stop developing a potentially useful medicine. Therefore, it is critical to consider Phase II trial designs, in the broader context of the entire medicine development plan, to make the best use of all available information, and to engage relevant experts. This presentation will focus on these perspectives.
Naitee Ting is a Fellow of American Statistical Association (ASA). He is currently a Director in the Department of Biostatistics and Data Sciences at Boehringer-Ingelheim Pharmaceuticals Inc. (BI). He joined BI in September of 2009, and before joining BI, he was at Pfizer Inc. for 22 years (1987-2009). Naitee received his Ph.D. in 1987 from Colorado State University (major in Statistics). He has an M.S. degree from Mississippi State University (1979, Statistics) and a B.S. degree from College of Chinese Culture (1976, Forestry) at Taipei, Taiwan.
Naitee published articles in Technometrics, Statistics in Medicine, Drug Information Journal, Journal of Statistical Planning and Inference, Journal of Biopharmaceutical Statistics, Biometrical Journal, Statistics and Probability Letters, and Journal of Statistical Computation and Simulation. His book “Dose Finding in Drug Development” was published in 2006 by Springer, and is considered as the leading reference in the field of dose response clinical trials. The book “Fundamental Concepts for New Clinical Trialists”, co-authored with Scott Evans, was published by CRC in 2015. Another book “Phase II Clinical Development of New Drugs”, co-authored with Chen, Ho, and Cappelleri was published in 2017 (Springer). Naitee is an adjunct professor of Columbia University, University of Connecticut. Naitee has been an active member of both the ASA and the International Chinese Statistical Association (ICSA).
Shuyen Ho is a Statistical Sciences Director at UCB BioSciences in RTP, North Carolina. Shuyen has worked in the pharmaceutical industry for 27 years. Prior to UCB, he was a Biostatistics Director at Parexel, Clinical Statistics Director at GlaxoSmithKline (GSK) and Statistics Group Leader at Merck. He has extensive experience in Phase II & III clinical development and helped developed widely used respiratory medicines such as Claritin, Advair and Flonase-Sensimist. Shuyen received his PhD in Statistics from University of Wisconsin – Madison, and his Bachelor degree from Taiwan.