For decades, oncology research has primarily relied on survival-based endpoints, such as progression-free survival and overall survival, to evaluate treatment efficacy. However, recent studies and international guidelines underscore the importance of incorporating patient-reported outcomes through patient-reported outcomes measures (PROMs). PROMs provide a more comprehensive view of treatment effectiveness, integrating the concepts of ‘living longer’ and ‘living better.’ Health-related quality of life (HRQoL) improvements have an intrinsic value for the patient, with importance in the overall definition of treatment value. These findings have sparked discussions regarding the relationship between HRQoL and traditional survival endpoints, influencing both oncology clinical trials and their interpretation for decision-making processes in practice. To effectively integrate PROMs into research, the choice of study design, appropriate PROMs questionnaires, and timing of administration are critical. The clinician's ability to interpret HRQoL data with awareness is equally important to ensure good clinical decision making. A pivotal concept in this context is the minimum clinically important difference (MCID), which is essential to inform the interpretation of treatment effect size in terms of clinically relevant HRQoL changes. Incorporating PROMs fosters a patient-centered approach to cancer care, aligning treatment goals with individual preferences and values. By balancing survival outcomes with quality of life, and through empathetic communication, healthcare providers can deliver treatments that are not only effective but also resonate with patients' experiences and priorities.

Association Between Health-Related Quality of Life Measures and Survival Endpoints in Oncology Clinical Trials and in Clinical Decision Making: A Narrative Review

Valsecchi, Anna Amela
;
Di Maio, Massimo
2025-01-01

Abstract

For decades, oncology research has primarily relied on survival-based endpoints, such as progression-free survival and overall survival, to evaluate treatment efficacy. However, recent studies and international guidelines underscore the importance of incorporating patient-reported outcomes through patient-reported outcomes measures (PROMs). PROMs provide a more comprehensive view of treatment effectiveness, integrating the concepts of ‘living longer’ and ‘living better.’ Health-related quality of life (HRQoL) improvements have an intrinsic value for the patient, with importance in the overall definition of treatment value. These findings have sparked discussions regarding the relationship between HRQoL and traditional survival endpoints, influencing both oncology clinical trials and their interpretation for decision-making processes in practice. To effectively integrate PROMs into research, the choice of study design, appropriate PROMs questionnaires, and timing of administration are critical. The clinician's ability to interpret HRQoL data with awareness is equally important to ensure good clinical decision making. A pivotal concept in this context is the minimum clinically important difference (MCID), which is essential to inform the interpretation of treatment effect size in terms of clinically relevant HRQoL changes. Incorporating PROMs fosters a patient-centered approach to cancer care, aligning treatment goals with individual preferences and values. By balancing survival outcomes with quality of life, and through empathetic communication, healthcare providers can deliver treatments that are not only effective but also resonate with patients' experiences and priorities.
2025
39
3
171
182
Health-Related Quality; Life Measures; Survival Endpoints; Oncology Clinical Trials; Clinical Decision Making
Valsecchi, Anna Amela; Di Maio, Massimo
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2318/2075070
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