ABSTRACT

The evidence to support new medicines, devices, or other medical interventions is based primarily on randomized clinical trials. Many of these trials involve assessments taken at the start of treatment (baseline), followed by assessments taken repeatedly during the treatment period. In some cases, such as cancer trials, the primary outcome is whether or not some important event occurred during the assessment intervals. These outcomes can be summarized by the time to the event, or as a percentage of patients experiencing the event at or before some landmark time point. Alternatively, the multiple post-baseline assessments can all be used in a longitudinal, repeated measures framework, which can either focus on a landmark time point or consider outcomes across time points. This book focuses on the longitudinal, repeated measures framework.