One important component of phase II is looking at various efficacy outcomes to determine which primary end point you will use for phase III and what specific label claims you will be able to make following an approved NDA. This article looks at four types of efficacy outcomes to consider as you design your phase II studies.
Karen Kesler, Ph.D. is a senior statistical scientist at Rho. Dr. Kesler currently serves as the co-Primary Investigator of the Statistics and Data Management Center for two NIH sponsored coordinating centers, has been deeply involved in researching more efficient Phase II and III trials, and has led many adaptive studies. She has written protocols, statistical analysis plans, and performed the analyses for many adaptive studies, including sample size recalculations, pruning designs, Bayesian dose escalation studies, and adaptive randomizations.
Andrea Mospan, PhD, is a program manager at Rho. She has nearly nine years of experience in the pharmaceutical industry, specializing in clinical aspects of pharmaceutical development. Dr. Mospan has collaborated with scientists and thought leaders in the design, development, implementation and management of programs for several compounds, and she has managed multiple cross-functional clinical study teams. Dr. Mospan’s therapeutic areas of expertise include respiratory and ophthalmology disorders, such as cystic fibrosis, blepharitis, allergic rhinitis, and allergic and bacterial conjunctivitis.