N-of-1 Design and Its Applications to Personalized Treatment Studies.

N-of-1 trial is a type of clinical trial which has been applied in chronic recurrent conditions that require long-term non-curative treatment. In this type of trials, each patient will be randomly assigned to one of the treatment sequences and repeatedly crossed over two or more treatments of interests. Through this cross-comparing method (cross-over phase), investigator can identify an optimal treatment (medicine or therapy) for the patient and treat the patient with the optimal treatment in an extension phase. This design could efficiently reduce the placebo effect, which is often seen in clinical trials, and maximize the true treatment effect. This type of design has been used in some traditional Chinese medicine (TCM) clinical trials lately. However, it brings some challenges for collecting and analyzing the data. Research on statistical methodology of this type of design is rarely found in the literature. The goal of this research is to discuss the application of the N-of-1 design to personalized treatment studies. We will demonstrate a real study conducted in TCM and present some theoretical and simulation results.