Health-related quality of life (HR-QOL) and regulatory issues. An assessment of the European Agency for the Evaluation of Medicinal Products (EMEA) recommendations on the use of HR-QOL measures in drug approval.

Interest in measuring qualitative aspects of life that are most closely related to health and healthcare has increased in recent years. Methods of describing patients' subjective health status now incorporate standardised measures, and several psychometric measures are available. Despite the thousands of empirical and conceptual papers in the medical and pharmacological literature on health-related quality of life (HR-QOL), the value of such measures in the regulatory process is still being debated. We conducted an assessment to understand and document the position of the European Agency for the Evaluation of Medicinal Products (EMEA) on the use of HR-QOL measures in studies conducted for regulatory purposes. Official documents produced and circulated by the EMEA containing recommendations on trial design, conduct and analysis for sponsors and scientific experts were independently reviewed by authors to document the position of the Agency on the specific topic of HR-QOL. All documents found in the Agency website on 30 September 1999 were identified and then assessed to: (i) identify diseases or drugs for which formal HR-QOL assessment is recommended; (ii) identify measures and methods recommended; and (iii) evaluate the reliability of recommendations across documents. Of the 189 documents retrieved, none focused directly on health-related quality of life. A few explicit recommendations were identified for 13 specific drugs or conditions. These recommendations were mostly general and vague, and used nonstandard terminology. In addition, terminology and recommendations were not consistent across documents and, in at least one case, were in contrast with the US Food and Drug Administration (FDA) guidelines. EMEA guidelines incorporating quality-of-life outcomes are welcomed but it is obvious that more detailed guidance is required. Closer collaboration between the EMEA and the FDA is also recommended. Experts from different disciplines should be involved in the preparation of such documents to assure the necessary technical expertise and the representativeness of the various counterparts.