Novel Research Designs

Innovative technologies such as mobile health (mHealth) and just-in-time adaptive interventions have created an opportunity to individualize health care. Yet, the evidence to support scaling of these innovative technologies has lagged behind the pace of technology advancement. This chapter examines consider current approaches to clinical evidence generation and how innovative research designs could drive precision medicine forward. While randomized controlled trials (RCTs) are currently considered the gold standard to inform practice, conventional RCTs can be costly and time intensive. This could hinder their usefulness when evaluating rapidly evolving technologies. As a result, a variety of novel study designs have evolved to help further individualize patient care and in some cases allow faster evidence generation. These new methods are expanded on within the chapter and include micro-randomized trials (MRTs), n-of-1, site-less design, and stepped wedge trials. Furthermore, we discuss study methodologies that are used to develop mHealth interventions such as patient centered participatory research and user centered design. Each of these research designs aim to propagate precision medicine by moving classic research techniques from population-averaged effects to those directly relevant to the individual, which is ideally where the future of medicine is headed.