Introduction to Patient Preference Studies

This chapter begins with a brief introduction to PFDD (Patient-Focused Drug Development) and the role a statistician can play in the design and analysis of Patient Preference Studies (PPSs), which are an important part of PFDD. It then gives a fuller definition of PFDD and reviews a selection of regulatory guidances related to PFDD. The chapter then continues by exploring how PPSs fit into the overall drug development lifecycle. Some different types of quantitative PPS are then described, and a case study based on an actual PPS is used to illustrate the statistical analysis, display of results, and interpretation of the data from a particular type of PPS known as a discrete choice experiment (DCE). How to check on the internal and external validity of a DCE is described next, followed by a section going into more detail regarding the optimal design of a DCE and the calculation of sample size. A case study describing another type of PPS, known as a Swing Weighting study, comes next, followed by some closing final remarks.