Traditionally, in our health system, treatment guidelines and health recommendations have been developed based on results of studies of large groups of individuals. However, the analysis of such large population-based studies only gives insights into factors affecting diseases and treatments on average. Hence, these results do not allow meaningful predictions whether an intervention will help a given single individual. For example, major drugs in therapeutic areas including asthma, depression, diabetes or rheumatoid arthritis have only shown an efficacy in 50%-60% of patients. Similarly, the effect of health interventions and lifestyle changes on chronic disease and health outcomes on an individual level if often unclear.
N-of-1 trials provide a framework to evaluate personalized treatments and derive invididual treatment effects. More specifically, N-of-1 trials are multi-crossover randomized controlled trials in a single participant, i.e. where the participant follows the different interventions of the study in a pre-specified or randomized order, and the outcome (e.g. improvement of lower back pain or rheumatoid arthritis pain) is compared between interventions.
Here, we are building a platform, developing and evaluating necessary tools and methods for the platform, and applying the platform to derive and evaluate individualized treatment effects.