Standardizing patient-reported outcomes across diseases: development of a novel generic patient-reported outcome set

标准化跨疾病的患者报告结局:开发一种新型通用的患者报告结局集

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Abstract

OBJECTIVES: Patient-reported outcomes (PROs) are an essential component in the implementation of value-based health care. Up to now, no consensus exists on the appropriateness of PROs used across diseases, e.g., to allow for comparability or to assess disease impact. The aim of this study was to develop an international, multi-stakeholder consensus on a generic PRO set applicable for different stakeholders and diseases within of the Health Outcomes Observatory (H2O) project funded by the EU Innovative Medicines Initiative. METHODS: To begin, a literature review was conducted to identify the most frequently utilized generic PROs followed by a three-round Delphi consensus procedure. The resulting outcome set was then cross-referenced with disease-specific outcome sets for lung and metastatic breast cancer, diabetes, and inflammatory bowel diseases to identify overlaps and gaps. Lastly, the identified generic outcome domains were mapped to the Max Neef's human needs model to explore the degree to which the generic domains address a general concept of wellbeing. RESULTS: The literature search resulted in 2357 articles from which 190 PROMs and their measured domains were extracted. The Delphi consensus procedure reduced these to 10 core domains (mental, physical and social wellbeing, overall health status, fatigue, pain, sleep quality, sexuality, self-efficacy, treatment satisfaction). In comparison to the human needs model, needs such as identity and leisure were disregarded. CONCLUSIONS: The H2O generic outcome set presents a disease-generic, domain-centered PRO framework building the groundwork for health data spaces and supporting consistency in treatment outcomes across different sites, settings, and patient populations.

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