Symposium on N-of-1 Trials

Hosted by:

Icahn School of Medicine at Mount Sinai
N1 App Research Team

Summary:

This virtual symposium brings together international scholars, practitioners and clinicians to discuss the growing field of n-of-1 trials or single-case experimental designs (SCED).

Introduction to N-of-1 trials

A/Prof Jason Bobe introduces the virtual symposium on N-of-1 trials with one origin story of their roots in the 18th century, then reviews trends in digital health and medicine that are transforming the field. Finally, he introduces the speakers and topics for this symposium. The symposium is hosted by the Icahn School of Medicine and the N1 app research team.

Dr. Kravitz discusses small data and n-of-1 studies. Both are potential solutions that aim to address the problem of the heterogeneity of treatment effects, where some patients derive more or less benefit than the average treatment effect as assessed through randomized controlled trials. He discusses the history, set-up and utility of n-of-trials for determining individual treatment effects in order to optimize the selection of treatments.

N-of-1 Design and Analysis Methods

Dr. Moeyaert discusses common methods for the interpretation of n-of-1 studies, also known as single case experimental designs (SCED). She begins with an overview of key design features of these studies. Next, she introduces an example of a sleep study one might conduct using SCED: does an intervention (sleeping pill) reduce the number of night time wake-ups compared to not taking the intervention? In this example, a blocked randomized alternating treatment design is used to investigate the effectiveness of the intervention. Then, she uses synthetic data to walk-through common analysis methods.

Dr. Percha discusses simulation as a useful method for n-of-1 practitioners to design their studies. Simulating an n-of-1 study forces one to think through each study parameter in advance (measurement, observation, treatment, etc) that may affect the study outcome. Simulation can also help to identify potential issues with power in advance. Unlike conventional clinical trials where power calculations estimate the number of patients needed to see an effect size, in n-of-1 studies, there is only 1 participant. Power calculations are based on the number of samples (e.g. observations of blood pressure) required to see a treatment effect. These and other parameters can be used in simulations to help optimize a well-powered study design. She walks through two examples of simulations she performed on data from previously published n-of-1 trials to demonstrate how these methods work and the kinds of things you might learn. Finally, she discusses how you might use her open-source simulation tool for designing your own n-of-1 trials.

Field Building

Dr. Nikles PhD MBBS is interviewed about her 20+ years of experience running an academic center focused on n-of-1 trials and single case designs (SCDs). She reflects on her early career experiences when few people were aware of n-of-1 trials, then describes her current work on sleep trials for children on ADHD medications, and shares her vision for the future of the field. Finally, she discusses resources for people seeking to get involved, including The International Collaborative Network (ICN) for N-of-1 Clinical Trials and Single-Case Designs (SCDs).

Dr. McDonald introduces the mission and objectives of the International Collaborative Network for N-of-1 Clinical Trials and Single-Case Designs (ICN).

Patient Community in Focus: Persistent Lyme disease

Dr. Aucott discusses persistent symptoms in Lyme disease. Lyme disease is the most common vector-borne disease in the United States, with an estimated 300,000 cases per year. Around 10-20% of patients have chronic symptoms following treatment, including fatigue, pain, cognitive issues, and sleep problems. With no FDA approved therapies, these symptoms can last month to years and have a severe impact on the quality of life for patients.

Dr. Schweig introduces the basics of sleep physiology, common sleep disturbance issues, and the importance of good sleep to individual well-being. He then reviews functional and integrative methods for coping with sleep disturbance problems associated with Lyme disease.

Dr. Varga introduces several tools used by physicians to assess sleep disorders. He then provides an introduction to the most common sleep disorders and their treatment.

To view the original virtual symposium on N1 App, please click here.