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.

Averaged versus individualized: pragmatic N-of-1 design as a method to investigate individual treatment response / Serpico, Davide; Maziarz, Mariusz. - In: EUROPEAN JOURNAL FOR PHILOSOPHY OF SCIENCE. - ISSN 1879-4912. - 2023, 13:4(2023), p. 59. [10.1007/s13194-023-00559-0]

Averaged versus individualized: pragmatic N-of-1 design as a method to investigate individual treatment response

Serpico, Davide
Co-primo
;
2023-01-01

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.
2023
4
Serpico, Davide; Maziarz, Mariusz
Averaged versus individualized: pragmatic N-of-1 design as a method to investigate individual treatment response / Serpico, Davide; Maziarz, Mariusz. - In: EUROPEAN JOURNAL FOR PHILOSOPHY OF SCIENCE. - ISSN 1879-4912. - 2023, 13:4(2023), p. 59. [10.1007/s13194-023-00559-0]
File in questo prodotto:
File Dimensione Formato  
Serpico & Maziarz 2023 - EJPS.pdf

accesso aperto

Tipologia: Versione editoriale (Publisher’s layout)
Licenza: Creative commons
Dimensione 840.69 kB
Formato Adobe PDF
840.69 kB Adobe PDF Visualizza/Apri

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11572/399229
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus 0
  • ???jsp.display-item.citation.isi??? 0
social impact