Background <p>Routinely collected health data are increasingly used to generate real-world evidence for therapeutic decision-making. Their use, however, depends on the expectations of multiple stakeholders. Clinicians require clinically interpretable analyses, pharmaceutical stakeholders need robust evidence on effectiveness and safety, patient advocacy groups emphasize transparency, privacy, and meaningful outcome measures, and statisticians focus on bias control, reproducibility, and methodological rigor. Without explicit consideration of these perspectives, analyses risk being fragmented, misaligned with end-user needs, or lacking transparency. Aligning these perspectives early in the design of routine data analyses therefore remains a central challenge.</p> Methods <p>We developed a stakeholder-inclusive conceptual framework for modeling routine health data, through expert panel discussions, an interdisciplinary workshop and targeted literature examples. The synthesis focused on four stakeholder perspectives: clinicians, pharmaceutical industry, patient advocates, and statisticians. To illustrate how stakeholder priorities can be translated into analytical strategies, we reviewed selected applications of multistate models (MSMs) in routine health data settings.</p> Results <p>The conceptual framework links stakeholder-specific priorities, methodological requirements and identifies shared needs for analyses that are clinically meaningful, transparent, reproducible, and able to represent patient pathways, intermediate events, treatment trajectories, disease progression, safety outcomes, and patient-reported measures. While the framework is intended to be applicable across various analytical approaches MSMs are used here to illustrate how these diverse requirements can be operationalized in practice. They can capture longitudinal health processes, competing events, recurrent or intermediate states, and state-specific outcomes while retaining an interpretable graphical structure, and the reviewed examples show their applicability across different research questions using routine health data. Beyond specific methodological choices, clinical research relies fundamentally on statistical expertise. The framework also highlights that the statistician’s role varies with the complexity of the research question, ranging from consultation on standard analyses to adaptation or development of advanced methods.</p> Conclusions <p>The stakeholder-inclusive framework provides methodological guidance for designing analyses of routine health data that are clinically meaningful, scientifically rigorous, and socially acceptable. By aligning the research question with the intended perspective from the beginning, it supports more robust and transparent evidence generation, with multistate models serving as a flexible tool to operationalize this integration.</p>

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A stakeholder-inclusive conceptual framework for modeling routinely collected health data for therapeutic decision-making illustrated by means of multistate models

  • Michelle Pfaffenlehner,
  • Andrea Dreßing,
  • Dietrich Knoerzer,
  • Markus Wagner,
  • Peter Heuschmann,
  • André Scherag,
  • Harald Binder,
  • Nadine Binder

摘要

Background

Routinely collected health data are increasingly used to generate real-world evidence for therapeutic decision-making. Their use, however, depends on the expectations of multiple stakeholders. Clinicians require clinically interpretable analyses, pharmaceutical stakeholders need robust evidence on effectiveness and safety, patient advocacy groups emphasize transparency, privacy, and meaningful outcome measures, and statisticians focus on bias control, reproducibility, and methodological rigor. Without explicit consideration of these perspectives, analyses risk being fragmented, misaligned with end-user needs, or lacking transparency. Aligning these perspectives early in the design of routine data analyses therefore remains a central challenge.

Methods

We developed a stakeholder-inclusive conceptual framework for modeling routine health data, through expert panel discussions, an interdisciplinary workshop and targeted literature examples. The synthesis focused on four stakeholder perspectives: clinicians, pharmaceutical industry, patient advocates, and statisticians. To illustrate how stakeholder priorities can be translated into analytical strategies, we reviewed selected applications of multistate models (MSMs) in routine health data settings.

Results

The conceptual framework links stakeholder-specific priorities, methodological requirements and identifies shared needs for analyses that are clinically meaningful, transparent, reproducible, and able to represent patient pathways, intermediate events, treatment trajectories, disease progression, safety outcomes, and patient-reported measures. While the framework is intended to be applicable across various analytical approaches MSMs are used here to illustrate how these diverse requirements can be operationalized in practice. They can capture longitudinal health processes, competing events, recurrent or intermediate states, and state-specific outcomes while retaining an interpretable graphical structure, and the reviewed examples show their applicability across different research questions using routine health data. Beyond specific methodological choices, clinical research relies fundamentally on statistical expertise. The framework also highlights that the statistician’s role varies with the complexity of the research question, ranging from consultation on standard analyses to adaptation or development of advanced methods.

Conclusions

The stakeholder-inclusive framework provides methodological guidance for designing analyses of routine health data that are clinically meaningful, scientifically rigorous, and socially acceptable. By aligning the research question with the intended perspective from the beginning, it supports more robust and transparent evidence generation, with multistate models serving as a flexible tool to operationalize this integration.