Date: October 2024
Location: On-demand webinar
N-of-1 trial designs are a method of studying treatment effectiveness by focusing on individual patients rather than groups. In rare disease research, where patient populations are small and heterogenous, N-of-1 trials offer an opportunity to determine optimal intervention for an individual patient using objective, data-driven criteria. By tailoring treatments to the unique characteristics of each patient, these trials can provide valuable insights into the efficacy of interventions and can lead to more personalised approaches to managing and treating rare diseases. Such trials can leverage study design and statistical techniques associated with standard population-based clinical trials, including randomisation, washout and crossover periods, as well as placebo controls. Additionally, N-of-1 trials help account for the variability often seen in rare disease, ultimately improving patient outcomes and enhancing our understanding of disease pathophysiology.
Despite their appeal and wide use in educational settings, N-of-1 trials have been used sparingly in clinical research.
In this webinar, attendees will learn about:
- The history, design, and use-case of N-of-1 trials emphasise their innovative utility in rare disease clinical research.
- How N-of-1 trials deserve serious consideration among the health research and clinical care communities given the contemporary focus and relevance of personalised medicine.
Moderators:
Neena Nizar
Jana Benesh
Panelists:
Dr. Tim Yu
Julia Viterello
Luke Rosen