Keywords: PragmaticTrials; Randomization; Benefit Risk
Pragmatic clinical trials (PCTs) have been traditionally used for studying comparative effectiveness of approved medications. Recently there is an increasing advocacy on using real world evidence in drug development, in particular, in clinical trials for supporting drug approval. The goal is to increase external validity (on a broader patient population) of the benefit risk profile of an investigational drug and provide more effectiveness information for stakeholders such as patients, physicians, payers and policy makers. PCT is a perfect tool to achieve this goal. Although PCTs and randomized controlled trials (RCTs) are perceived in a continuum, some unique characteristics of pragmatic trials may give rise to many statistical problems of significant regulatory or practical concerns. In this talk, we will discuss some distinct aspects of study designs and analyses of PCTs. It is important to understand these challenges. A case study will be discussed in details for relevant illustrations.