The promise of precision therapeutics has not been fully realized, and a current focus of our research aims to address one key reason. The current method for bringing a clinical therapy to the public is to conduct a definitive, between-patient, Phase III randomized controlled trial (RCT). This between-patient RCT provides evidence that, all other things equal, the therapy tested in one group of patients is better than the placebo or alternative therapy tested in another group of patients. Yet, between-patient RCT results do not actually offer information on which therapy is optimal for each individual patient. Rather, they provide an estimate of the average therapy effect, with some proportion of patients receiving little benefit or even harm.
We strongly challenge the assumption that the best therapy for an individual patient can be ascertained without RCT data for that individual patient. Over the past several years, the group has been laying the foundation necessary for conducting N-of-1 randomized controlled trials (RCT). N-of-1 RCTs are multiple crossover trials conducted within individual patients. Instead of comparing two groups of patients on different treatments, they focus on the individual patient by randomizing time periods in which that patient receives different treatments. Thus, N-of-1 RCTs can provide precise data on the effect of a treatment for the individual patient, so they can work with their clinician to decide upon optimal treatment.
This new approach will optimize therapy for individual patients, and will also yield effectiveness data at the group level, as pooling results across similarly conducted N-of-1 trials allows for inferences about the effectiveness of an intervention across a population. What must be developed is a clinician and patient-friendly, efficient, innovative platform for single-patient trials, with remote, real-time, automated data collection and point-of care statistical analyses and a registry for pooling data from N-of-1 trials. If successful, this N-of-1 approach could revolutionize the way we conceptualize, study, and determine the optimal therapy for each individual patient we see.