N-of-1 trials: Implications for clinical practice and personalized clinical trials

Abstract

Abstract Traditionally, most studies in autoimmune research are based on randomized control trials (RCT) or cohort studies. However, these designs are limited in rare conditions, such as many autoimmune diseases, due to the large number of patients required to provide adequate power. There is growing evidence that single case experimental designs, such as N-of-1 trials, might provide valuable insights on individual effects that can be used to optimize treatments for individual patients. In addition, results from a series of N-of-1 trials can be combined and analyzed to estimate population effects. Evidence from single N-of-1 trials may also inform future RCT designs. In this chapter we discuss several aspects of N-of-1 trials methodology including statistical, ethical and regulatory aspects.