Using patient-reported outcomes and health-related quality of life data in regulatory decisions on cancer treatment: highlights from an EMA-EORTC workshop
Cet article met en avant l'intérêt de prendre en compte les symptômes déclarés par les patients et les données concernant leur qualité de vie pour orienter les décisions thérapeutiques en oncologie
The benefit-risk assessment of cancer treatments usually focuses on traditional clinical and disease outcomes, such as overall survival, progression-free survival, and tumour response, balanced against clinician-reported adverse events. Patient-reported outcomes (PROs) measuring symptoms, functioning, and other health-related quality of life (HRQOL) impacts can be used to ensure that a drug's effect on symptoms and functioning are quantified and evaluated as part of cancer clinical research and drug development. However, the impact of PROs on regulatory assessment and health technology assessment (HTA) can vary due to issues related to study design, PRO item selection, assessment frequency, study conduct, and handling of data missingness.
The Lancet Oncology , résumé 2025