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.
Preston Long, Alize Rogge, Ann-Kristin Porth, Evelyn Gross, Liselotte Fierens, Belle H de Rooij, Nadia Kamminga, Tanja Stamm
Disease Category:
Disease Name:
Age Range: 18 - 100
Sex: Either
Nature of Intervention: Any
- Administrative experts
- Clinical experts
- Consumers (patients)
- Policy makers
- Regulatory agency representatives
- Researchers
- COS for practice
- Consensus meeting
- Delphi process
- Systematic review
- Generic outcome domains identified in the Delphi consensus procedure were cross-mapped to the developed disease-specific H2O PRO outcome sets for lung (13) and metastatic breast cancer (14), diabetes (12) and inflammatory bowel diseases (15). Ranked in order of most shared/overlapping to least, the domains were then compared to two existing and well-established generic outcome frameworks: PROMIS (generic outcomes across diseases) and the EORTC (generic outcome across different cancer entities).
- This mapping exercise used the selected domains from the generic Delphi exercise to explore areas of redundancy and thus assumed importance in the field, as well as gaps, and thus inferred unimportance (11). In doing so, the aim was to highlight the fundamental attributes currently considered key to human well-being as well as to identify which are not represented in the sets reviewed. The Max-Neef model of human needs was selected for this task as it is the only well-validated model which envisions human needs as intersectional and interactive, rather than hierarchical (20).