Patient-reported outcomes: The example of health-related quality of life - A European guidance document for the improved integration of health-related quality of life assessment in the drug regulatory process

The added value for assessing the health-related quality of life (HRQOL) in chronic conditions is now well documented for evaluation of treatment effectiveness in clinical trials and as a criterion for licensing new medications and in policy decisions. However European standards still need to be developed for the measurement and reporting of HRQOL in clinical trials. This is one of the objectives of the European Regulatory Issues on Quality of Life Assessment (ERIQA) Working Group. This document reviews the major issues arising from the selection of an HRQOL instrument; the integration of HRQOL assessment into the research protocol (methodological design, practicalities of HRQOL administration and collection, prevention and handling of missing data); the statistical analysis plan; and the presentation and interpretation of the results. Finally, to gain wider acceptance, whether HRQOL is considered as a primary or secondary endpoint, the scientific principles of clinical trial design should apply to HRQOL.