The European Medicines Agency (EMA) has published new guidance on the use of patient-reported outcome (PRO) measures in oncology studies. The new guidance is an appendix to the guideline on the evaluation of anticancer medicinal products in man, which was last revised in July 2013.
This guidance document issued by the Committee for Medicinal Products for Human Use (CHMP) and its Oncology Working Party (ONCWP) describes the use of PROs and health related quality of life (HRQL) measures in clinical studies. It acknowledges the importance of bringing the perspective of patients on their disease and the treatment they receive to the assessment of benefits and risks of cancer medicines.
The new guidance outlines principles of scientific best practice rather than prescribing a particular approach to the selection and application of PROs and provides guidance on how patient-reported outcome data should be integrated in oncology clinical trials.
The new guidance provides:
- recommendations for the incorporation of PROs and HRQL in the clinical development of anticancer medicines;
- advice on appropriate design, conduct and analysis of PRO studies.
Patient-reported outcomes and quality of life measures
The experience of patients of how a treatment impacts on their well-being and everyday life is an important aspect of the evaluation of the clinical benefits of new medicines.
PROs include any data directly reported by a patient that is based on his or her perception of a disease and its treatment. They provide information on a patient’s quality of life (impact on any aspect of daily life and social functioning), symptoms, treatment adherence or satisfaction with care. Quality of life during disease and treatment is a personal perspective and may vary from person to person but is possible to be captured using well established methods.
PROs and HRQL measures complement the range of traditional indicators of efficacyof an oncology medicine and can provide information regarding both positive and negative patient experiences.