• guidance;
  • outcomes research;
  • quality-of-life research;
  • regulatory

Introduction: Health-related quality-of-life (HRQL) can be defined as the impact of disease and treatment across the physical, psychological, social and somatic domains of functioning and well-being. Health-related quality-of-life measures are included in clinical trials of drug treatment to assess the impact of therapy on the patient's functioning. HRQL guidance could allow for use of this data in drug labeling and promotion.

Objectives: The aim of our study was to provide recommendations with respect to regulatory issues important to the development of guidelines for HRQL research.

Methods: The HRQL workshop was planned jointly by members of the Pharmaceutical Research and Manufacturers of America Health Outcomes Committee and the Division of Drug Marketing, Advertising, and Communications of the Food and Drug Administration. The workshop was limited to six regulatory issues related to HRQL research in clinical trials of pharmaceutical therapies. These six issues were: instrument selection and validation, study design, data analysis, HRQL and safety, clinical meaning, and promotional use. Before the meeting, a consensus was reached that HRQL does not measure, nor should it be used to measure, safety. Therefore, five work groups discussed HRQL issues and made recommendations.

Results: Overall, the workshop recommended that HRQL measures be treated as any other clinical end point. The workshop recognized that research in HRQL methods is ongoing and that any guidance should be flexible to allow for changes in this developing research area.

Conclusions: HRQL provides a patient perspective on the impact of disease and therapy on patients' daily life and functioning. Including HRQL information in promotion could be beneficial to decision making on the use of therapies. HRQL is a measure of effectiveness, not safety, and should be treated as any other clinical end point.