In the process of drug discovery and drug development, understanding the dose-response relationship is one of the most challenging tasks. It is also critical to identify the right range of doses in early stages of clinical development so that Phase III trials can be designed to confirm these doses. Usually at the beginning of Phase II, there is not a lot of available information to help guiding the study design. At this stage, Phase II clinical studies are needed to establish proof of concept (PoC), to identify a set of potentially effective and safe doses, and to estimate dose-response relationships.
Challenges in designing these studies include: selection of the dose frequency and the dose range, choice of clinical endpoints or biomarkers, and use of control(s), among others. Consequences of bad Phase II study designs may lead to the delay of the entire clinical development program or the waste of R&D investment. Misleading results obtained from poor designs could cause a Phase III program to confirm a wrong set of doses, or to stop developing a potentially useful drug. Therefore, it is critical to consider an entire drug development plan, to make best use of all the available information, and to include all relevant experts in designing Phase II dose response clinical trials. This presentation discusses some of these considerations.
Naitee Ting, Ph.D.
Naitee Ting is a Fellow of ASA. He is currently a Sr. Principal Biostatistician in the Biometrics Department of 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).
Naitee published articles in Technometrics, Drug Information Journal, Journal of Statistical Planning and Inference, Journal of Biopharmaceutical Statistics, Biometrical Journal, Statistics and Probability Letters, Statistics in Medicine and Journal of Statistical Computation and Simulation. His book “Dose Finding in Drug Development” was published in March 2006 by Springer. Naitee is an adjunct professor of Columbia University, University of Connecticut and an adjunct professor of University of Rhode Island. Naitee has been an active member of both the American Statistical Association (ASA) and the International Chinese Statistical Association (ICSA).
