Health-related quality of life and regulatory issues in the US and Canada.

The purpose of this paper is to examine the utilisation of HR-QOL information by US and Canadian federal regulatory bodies, with a focus on pre- and postmarket product review processes. There are a number of challenges facing regulators in using HR-QOL data derived from clinical trials and/or pharmacoeconomic analyses. Some of these challenges are inherent to HR-QOL tools and methodologies, while others relate to their inappropriate application in clinical studies. Regulators also need to take some responsibility for the chasm that exists between the potential and the reality of the contribution of HR-QOL information in decision making. Federal regulators need to develop and promulgate clear guidances that will help improve the quality and validity of studies submitted for review, and greater transparency is needed in terms of how such information is used in the product review process. Advances in technology are sure to increase the volume and complexity of HR-QOL information collected within clinical trials, and regulators need to prepare themselves to address this influx of data. Key actions are proposed to improve both the credibility as well as the uptake of HR-QOL information within the regulatory process so that this important source of information can be used appropriately and to its full potential.