Unraveling assumptions about clinical relevance in patient-reported outcome data : A qualitative study on perspectives of different interest groups.

PURPOSE: The interpretation of patient-reported outcomes (PROs) in oncology research lacks a shared understanding of clinical relevance among interest groups. Terms like minimal important differences and clinically meaningful change aim to aid interpretation but remain inconsistently conceptualized. This study explores interest-holders' perspectives to support setting-specific definitions of clinical relevance in PRO research.

METHODS

An online survey was distributed via international networks to multi-professional interest-holders. Responses to an open-ended question regarding participants' understanding of clinical relevance were analyzed using qualitative content analysis to identify recurring themes and patterns within the quotes.

RESULTS

The survey included 92 participants: clinical practitioners (38.5%), academic researchers (47.3%), industry researchers (8.8%), and patients/patient representatives (rep.) (5.5%). Five clusters emerged reflecting facets of clinical relevance: (a) patient value (e.g., impact on well-being), (b) practical implications (e.g., treatment changes), (c) external criteria (e.g., physiological changes), (d) statistical approaches (e.g., 10% difference), and (e) proxy value (e.g., physician's perspective). Practitioners primarily focused on patient value (55.6%), while academic researchers showed a similar distribution but with greater variance across clusters. In contrast, industry researchers more frequently emphasized external criteria (20.0%) and proxy value (20.0%) compared to other groups. Patient value and statistical approaches were not mentioned by the same participants, nor were external criteria and practical implications.

CONCLUSION

Conceptual understanding of clinical relevance varies by professional background, highlighting its multifaceted nature. These findings, identifying distinct conceptual clusters across interest groups, provide a foundation for developing harmonized, context-specific definitions of clinical relevance in PRO research.