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Activity Number:
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470
- Beyond Precision Medicine: Making It Personal with N-of-1 and Single Case Methods for Medicine, Rare Diseases, Digital Health, Behavior, and Wearables
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Type:
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Topic Contributed
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Date/Time:
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Wednesday, August 10, 2022 : 2:00 PM to 3:50 PM
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Sponsor:
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Biometrics Section
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Abstract #320890
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Title:
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Efficient Sequential N-of-1 Trials and Observational Studies
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Author(s):
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Nicholas Schork*
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Companies:
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The Translational Genomics Research Institute
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Keywords:
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N-of-1 trial;
sequential tests;
serial correlation
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Abstract:
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Single subject, or ‘N-of-1,’ studies are receiving a great deal of attention from both theoretical and applied researchers. This is consistent with the growing acceptance of ‘personalized’ approaches to health care and the need to prove that personalized interventions tailored to an individual’s likely unique physiological profile and other characteristics work as they should. In fact, the preferred way of referring to N-of-1 studies in contemporary settings is as ‘personalized studies.’ Designing efficient personalized studies and analyzing data from them in ways that ensure statistically valid inferences are not trivial, however. I point out that more efficient sequential designs for personalized and aggregated personalized studies can be developed using, among other techniques, sequential probability ratio tests (SPRTs), and I explore their properties via simulation studies. I also focus on future directions for sequential personalized studies, including those that make use of online False Discovery Rate (FDR) strategies.
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Authors who are presenting talks have a * after their name.
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