International Journal of Technology Assessment in Health Care


EMEA and the evaluation of health-related quality of life data in the drug regulatory process

Sotiria Papanicolaou a1, David Sykes a1 and Elias Mossialos a2
a1 Eli Lilly and Company Limited
a2 LSE Health and Social Care


Objectives: This study reviewed the European guidelines of the Committee for Proprietary Medicinal Products (CPMP) on how health-related quality of life (HRQOL) research should be conducted in clinical trials. Published product-level information was also reviewed to investigate the actual role of HRQOL data in the European regulatory process.

Methods: All disease-specific notes for guidance and concept papers on clinical investigations, development and evaluation of human medicinal products, as well as the European Public Assessment Reports (EPAR) of all approved drugs published on the European Agency for the Evaluation of Medicinal Products (EMEA) Web site were evaluated for their HRQOL recommendations.

Results: Only twenty of the fifty CPMP guidance notes for clinical investigation of pharmaceutical products in specific disease areas included a reference to HRQOL. Most of the recommendations were generic and vague, and the terminology used was inconsistent across documents. The EPAR provided nonspecific information about HRQOL and contradictory conclusions on the effect of a drug on HRQOL sometimes occurred in different documents. The criteria used by the CPMP to assess the HRQOL data could not be identified due to an ad hoc approach to the inclusion of data in the EPAR.

Conclusions: A more systematic approach is needed on the way health outcomes data are considered, reviewed, and interpreted by the regulatory authorities. For this to be achieved, CPMP should develop general guidelines on the importance of HRQOL and how research should be conducted if data are to be included in the registration process.

Key Words: Clinical trials; Drug regulation; Health status; Health-related quality of life instruments; Quality of life.