Use of health-related quality of life measures by industry and regulatory agencies in evaluating oncology therapies

Abstract

Introduction More than 50 years ago, clinical researchers in oncology recognized that the outcomes of chemotherapy extended beyond survival and other clinical endpoints. The earliest effort to systematically measure the impact of cancer therapy on patient functional status was by Karnofsky et al. During the past 30 years, significant advances have been made in understanding the impact of cancer and oncology treatments on patient health-related quality of life (HRQOL) and other patient-reported outcomes.– HRQOL measures extend patient outcomes assessment beyond survival, toxicity, and clinical efficacy and reflect the patient's perspective on the impact of disease and its treatment on functioning and well-being. Frequently, an important objective for evaluating HRQOL outcomes is demonstrating the value of new oncology treatments relative to other competing treatments. The pharmaceutical industry's intent in funding clinical trials with HRQOL measures is to achieve labeling and/or promotional claims for marketing to physicians, pharmacists, health care decision makers, and patients., Although HRQOL outcomes have been used in evaluating oncology treatments, the use of these endpoints in drug development and the regulatory approval process is a relatively new phenomenon. In the past, the Food and Drug Administration (FDA) has encouraged the measurement of survival, symptom, and HRQOL endpoints in evaluating oncology treatments. During the past 17 years, no new cancer chemotherapy has been approved based primarily on HRQOL endpoints., More recent statements by FDA officials continue to express skepticism about the value of HRQOL in evaluating oncology treatments.