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 | |||||||||
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| 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 |
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| 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 |
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| Date: | December 2023 | |||||||||
| URI: | https://philsci-archive.pitt.edu/id/eprint/22877 |
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