Using Single Subject (N-of-1) Designs to Answer Patient-Identified Research Questions
We will employ a common research method known as N-of-1experiments, which are clinical trials in a single patient, to answer patient-identified research questions from two very different patient-powered research networks (PPRNs). The ImproveCareNow (ICN) network focuse on children with inflammatory bowel disease (IBD), and the Health eHeart (HeH) network focuses on adults with cardiovascular disease. Our work will significantly impact the health of individuals and populations and advance truly personalized and patient-centered outcomes research.
IBD affects as many as 1.4 million children and adults in the United States and has significant lifelong effects on both physical and psychosocial well-being. Preliminary data suggest that the specific carbohydrate diet (SCD) may improve clinical symptoms and reduce inflammation. However, no controlled, large-scale, multi-center studies provide evidence of SCD’s effectiveness compared with unrestricted diets or liberalized versions of the diet. Determining the effectiveness of the SCD will address an important gap in care.
Paroxysmal atrial fibrillation (AF) is a common arrhythmia that impairs the lives of otherwise healthy, high-functioning individuals. While studies have identified risk factors for new-onset AF, triggers for discrete episodes have been largely ignored. Triggers vary from patient to patient and may include alcohol, sleeplessness, stress, caffeine, and certain types of exercise. If each patient’s triggers could be identified, they might be avoided, providing a ready strategy to effectively prevent AF.
N-of-1 trials are ideal for addressing these two important research questions. By focusing on the effect of treatment for a single individual, such studies can identify the best treatment for an individual patient, who can then to stop ineffective treatments, thereby reducing harm and waste. N-of-1 studies are particularly useful when disease pathology and response to treatment varies markedly across individuals, as is the case for diet in IBD and triggers in AF.
The specific aims of this study are
- Use a series of N-of-1 trials to determine the effectiveness of the SCD in reducing symptoms in patients with IBD as compared to a liberalized SCD or unrestricted diet
- Test the comparative effectiveness of using N-of-1 designs versus data tracking alone to help patients identify and remove AF triggers and reduce AF severity
- Assess the feasibility, acceptability, and impact of using N-of-1 research approaches across diverse populations for different clinical questions