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Activity Number: 470 - Beyond Precision Medicine: Making It Personal with N-of-1 and Single Case Methods for Medicine, Rare Diseases, Digital Health, Behavior, and Wearables
Type: Topic Contributed
Date/Time: Wednesday, August 10, 2022 : 2:00 PM to 3:50 PM
Sponsor: Biometrics Section
Abstract #322843
Title: Experiences with Large Series of Individual Trials for Personalized Health
Author(s): Christopher Schmid*
Companies: Brown University
Keywords: N-of-1 trials; Personalized health; Treatment effect heterogeneity; Mobile devices; Patient-reported outcomes; Multilevel models

The most common personalized experimental design used in the health sciences is the N-of-1 trial, a multi-crossover withdrawal/reversal design in which participants receive a set of two or more treatments multiple times in a randomized order. In addition to measuring individual treatment efficacy, N-of-1 trials may be combined in a multilevel structure to assess average treatment effects in populations and treatment effect heterogeneity in subgroups. Mobile device applications provide a means to implement such trials on a large scale. We discuss some N-of-1 studies in which we have combined mobile device applications with server-driven statistical analytics to return results to individuals. We discuss issues concerning implementation, interpretation and decision making that these studies have raised. These include defining treatments and sequences of treatments, synthesizing treatment networks, incorporating patient-specific prior information, automating the choice of appropriate statistical models and assessment of model assumptions, and automating graphical displays and text to facilitate appropriate interpretation by non-technical users.

Authors who are presenting talks have a * after their name.

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