Key Considerations in Forming Research Questions and Conducting Research in Real-World Setting

In this chapter, we review and identify key elements of forming sound research questions in real-world setting. Such research questions would be answered through real-world studies and the analysis of real-world data. The derived real-world evidence may be used to help support approval of a new indication for a medical product that has already been approved or to help support postapproval study requirements. We also discuss how to revise the research question if it cannot be answered. Furthermore, we summarize real-world study designs into three major categories, from which we could choose an appropriate design to answer the research question if an answerable question is formed. In category one, we design a real-world study to prospectively generate real-world data for research purpose. In category two, based on one or several existing real-world data sources, we design a retrospective real-world study. In category three, we utilize real-world data in the design, conduct, and analysis of clinical trials.