Emulation of Target Trials to Study the Effectiveness and Safety of Medical Interventions

Anders Huitfeldt, Harvard T.H. Chan School of Public Health, Harvard University

Ideally, clinical guidelines would be informed by well-designed randomized experiments. However, it is generally not possible to conduct a randomized trial for every clinically relevant decision. Decision makers therefore often have to rely on observational data. Guidelines that rely on observational data due to the absence of randomized trials benefit when the analysis mimics the analysis of a hypothetical target trial. This can be achieved by explicitly formulating the protocol of the target trial, and thoroughly discussing the feasibility of the conditions that must be met in order to validly emulate the target trial using observational data. In chapter one, we discuss the emulation of trials that compare the effects of different timing strategies, that is, strategies that vary the frequency of delivery of a medical intervention or procedures, and provide an application to...