Patient Reported Outcomes in oncology, what beyond Randomized Controlled Trials?

Patient Reported Outcomes in oncology, what beyond Randomized Controlled Trials?

2017 Regulatory and Economic Aspects in Oncology

Toumi, M. | Array | Array | Array | Array |

The goal of 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. A term patient-reported outcome 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 PRO in oncology trials is increasing and the U.S. Food and Drug Administration published a guidance on the review and evaluation of patient-reported outcomes. However, while patient-reported outcomes 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.