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Averaged versus Individualized: Pragmatic N-of-1 Design as a Method to Investigate Individual Treatment Response

Serpico, Davide and Maziarz, Mariusz (2023) Averaged versus Individualized: Pragmatic N-of-1 Design as a Method to Investigate Individual Treatment Response. [Preprint]

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Abstract

Heterogeneous treatment effects represent a major issue for medicine as they undermine reliable inference and clinical decision-making. To overcome the issue, the current vision of precision and personalized medicine acknowledges the need to control individual variability in response to treatment. In this paper, we argue that gene-treatment-environment interactions (G × T × E) undermine inferences about individual treatment effects from the results of both genomics-based methodologies—such as genome-wide association studies (GWAS) and genome-wide interaction studies (GWIS)—and randomized controlled trials (RCTs). Then, we argue that N-of-1 trials can be a solution to overcome difficulties in handling individual variability in treatment response. Although this type of trial has been suggested as a promising strategy to assess individual treatment effects, it nonetheless has limitations that limit its use in everyday clinical practice. We analyze the existing variability within the designs of N-of-1 trials in terms of a continuum where each design prioritizes epistemic and pragmatic considerations. We then support wider use of the designs located at the pragmatic end of the explanatory-pragmatic continuum.


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Item Type: Preprint
Creators:
CreatorsEmailORCID
Serpico, Davide0000-0003-3997-3056
Maziarz, Mariuszmariusz.mm@gmail.com0000-0003-1979-0746
Keywords: Gene-environment interactions; Conflicting results; Randomized controlled trials (RCTs); P-medicine; Genome-wide association studies (GWAS); N-of-1 trials
Subjects: Specific Sciences > Biology > Molecular Biology/Genetics
Specific Sciences > Medicine > Clinical Trials
General Issues > Evidence
General Issues > Values In Science
Depositing User: Dr. Davide Serpico
Date Deposited: 17 Dec 2023 23:49
Last Modified: 17 Dec 2023 23:49
Item ID: 22877
DOI or Unique Handle: https://doi.org/10.1007/s13194-023-00559-0
Subjects: Specific Sciences > Biology > Molecular Biology/Genetics
Specific Sciences > Medicine > Clinical Trials
General Issues > Evidence
General Issues > Values In Science
Date: December 2023
URI: https://philsci-archive-dev.library.pitt.edu/id/eprint/22877

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