Patient-reported outcomes in oncology, beyond randomized controlled trials

Abstract

The goal of the treatment of a disease has moved from treating organs and diseases through symptoms, biological parameters and imaging towards treating a human being as a whole. The treatments should deliver benefits that patients can personally perceive. However, the patient’s perspective does not always match the one of those surrounding them. Illustratively, patients’ symptom assessments are more predictable for daily health status, whereas clinicians’ symptom measurements are more related to clinical outcomes. The term, patient-reported outcomes (PROs), includes any data that are reported directly by the patient without an intermediary, such as a family member or a healthcare professional. The use of PROs in oncology trials is increasing and the U.S. Food and Drug Administration has published guidelines on the review and evaluation of PROs. However, while PROs are increasingly used in clinical trials, they are rarely used in daily clinical practice. Further, healthcare payers are concerned with issues related to relevance, quality, and interpretability of these outcomes.