The February 16, 2001, meeting represented an important step in harmonizing efforts across various organizations and in opening a dialogue with the FDA around major issues related to methodologic standards for measuring and interpreting PROs in the drug evaluation process.

The recommendations presented for incorporating PROs in CER are intended to provide a guide to researchers, clinicians, and policy makers to ensure that information derived from PROs is applicable and interpretable for a given CER context. In turn, CER will provide information that is necessary for clinicians, patients, and families to make informed care decisions.

The purpose of this paper is to produce a summary of these articles and to supplement these works with additional information that was gleaned from subsequent meetings and discussions of this material. The primary aim of this paper is to present a cogent and concise description for clinicians to facilitate the incorporation of QOL assessments into oncology clinical research and practice. The theoretical discussion is supplemented with an example of how the various ideas can be operationalized in an oncology clinical trial.

Health-related quality of life (HRQL) outcomes evaluation is becoming an important component of clinical trials of new pharmaceuticals and medical devices. HRQL research provides patients, providers, and decision makers with important information on the impact of disease and treatment on physical, psychological, and social functioning and well-being. These outcomes are also useful to the pharmaceutical and device industries as they attempt to understand and communicate product value to physicians, patients, health insurers and others. HRQL labeling and promotional claims in the US are likely to increase over the next few years. The evidentiary requirements to make such a claim should be based on accepted scientific standards of HRQL evaluation and consistent with the regulatory requirements for clinical efficacy. This report outlines the scientific practices that should be considered in the evaluation of evidence for an HRQL claim, including the selection of appropriate domains, evidence to support the reliability and validity of HRQL measurement, considerations in research design and statistical analyses, and the issue of clinical significance. Representatives from the pharmaceutical and device industries, regulatory agencies, and the HRQL scientific community should work together to make certain the use of HRQL in labeling and promotion are based on sound scientific evidence, and that these messages are clearly and accurately reported to the consumers.