Health-related quality of life and other patient-reported outcomes in the European centralized drug regulatory process: a review of guidance documents and performed authorizations of medicinal products 1995 to 2003.

OBJECTIVES: The objective of this study was to review and analyze the use of health-related quality of life (HRQL) and other patient-reported outcome (PRO) evaluations for the approval of new pharmaceutical products by the European Medicines Agency (EMEA).
METHODS: All published EMEA guidance documents and regulatory information for products authorized at the EMEA and appearing in the European Public Assessment Report (EPAR) database between 1995 and 2003 were examined for reference to HRQL and other PROs.
RESULTS: More than half of the guidance documents for clinical investigation of pharmaceutical products in specific disease areas included reference to HRQL or other PROs. Guidance notes for 10 conditions indicated PROs can serve as primary endpoints in clinical trials, among which three included HRQL outcomes. The review of EPAR documentation uncovered HRQL and other PRO data for 34% of the drugs registered during the period of the review, with cancer-related treatments most frequently including PRO data. There was a trend toward increasing HRQL and other PRO claims in regulatory documents of pharmaceutical products in recent years, with the proportion exceeding 30% from 1999 to 2003.
CONCLUSIONS: There is further scope for health outcomes researchers and regulatory decision-makers to contribute to the more efficient utilization of PROs and HRQL outcomes. Health researchers need to better justify the inclusion of these outcomes in clinical trials and highlight the added value of PRO data; while the regulators should develop harmonized procedures and capacities to adequately appraise the submitted information.