The evidence is not the average: why personalized therapies expose a structural limit in RCT logic.

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Autore principale: Domargård, Anita
Natura: Recurso digital
Lingua:inglese
Pubblicazione: Zenodo 2026
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author Domargård, Anita
author_facet Domargård, Anita
contents <p>This Evidence Note examines a structural limitation in randomized controlled trial (RCT) logic when applied to personalized therapies.</p> <p>While RCTs estimate average treatment effects across populations, many emerging interventions—particularly in oncology—are inherently individualized. In these contexts, the assumption of comparability across patients breaks down. What appears as variability or inconsistency in outcomes may instead reflect structured, state-dependent responses within dynamic biological systems.</p> <p>Drawing on clinical epidemiology, oncology, systems biology, and decision science, this work reframes treatment response as an emergent property of system state and trajectory rather than a fixed effect of an intervention. The note introduces state-dependence as a central principle for interpreting treatment effects, extending beyond traditional discussions of heterogeneity.</p> <p>Within the Universal Resonance Model (URM), disease is understood as a dynamic system in which signal meaning changes over time. From this perspective, the challenge is not a lack of rigor in evidence generation, but a mismatch between static methodologies and evolving biological processes.</p> <p>The implications are both methodological and clinical. Averaging effects across populations may obscure meaningful signals, increase false negatives, and misclassify effective therapies. Clinically, treatment decisions require interpretation of trajectory and timing, not only endpoint outcomes.</p> <p>This note contributes to the ongoing shift from population-based inference toward dynamic, context-sensitive models of clinical evidence.</p>
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spellingShingle The evidence is not the average: why personalized therapies expose a structural limit in RCT logic.
Domargård, Anita
Personalized medicine
Randomized controlled trials
Treatment heterogeneity
State-dependent response
Systems medicine
Dynamic disease;
Clinical decision-making
Treatment timing
Biological variability
Precision oncology
Causal inference
Universal Resonance Model (URM)
Medicine
Rheumatology
Systems Biology
Computer and Information Science
Clinical Medicine
Epidemiology
<p>This Evidence Note examines a structural limitation in randomized controlled trial (RCT) logic when applied to personalized therapies.</p> <p>While RCTs estimate average treatment effects across populations, many emerging interventions—particularly in oncology—are inherently individualized. In these contexts, the assumption of comparability across patients breaks down. What appears as variability or inconsistency in outcomes may instead reflect structured, state-dependent responses within dynamic biological systems.</p> <p>Drawing on clinical epidemiology, oncology, systems biology, and decision science, this work reframes treatment response as an emergent property of system state and trajectory rather than a fixed effect of an intervention. The note introduces state-dependence as a central principle for interpreting treatment effects, extending beyond traditional discussions of heterogeneity.</p> <p>Within the Universal Resonance Model (URM), disease is understood as a dynamic system in which signal meaning changes over time. From this perspective, the challenge is not a lack of rigor in evidence generation, but a mismatch between static methodologies and evolving biological processes.</p> <p>The implications are both methodological and clinical. Averaging effects across populations may obscure meaningful signals, increase false negatives, and misclassify effective therapies. Clinically, treatment decisions require interpretation of trajectory and timing, not only endpoint outcomes.</p> <p>This note contributes to the ongoing shift from population-based inference toward dynamic, context-sensitive models of clinical evidence.</p>
title The evidence is not the average: why personalized therapies expose a structural limit in RCT logic.
topic Personalized medicine
Randomized controlled trials
Treatment heterogeneity
State-dependent response
Systems medicine
Dynamic disease;
Clinical decision-making
Treatment timing
Biological variability
Precision oncology
Causal inference
Universal Resonance Model (URM)
Medicine
Rheumatology
Systems Biology
Computer and Information Science
Clinical Medicine
Epidemiology
url https://doi.org/10.5281/zenodo.19650951