Assessing Tumor-Related Signs and Symptoms to Support Cancer Drug Approval

Abstract Cancer causes premature death and significant, often devastating, symptoms. While prolongation of survival is an obvious end point for new cancer drug approval, the US Food and Drug Administration (FDA) has also utilized end points that evaluate patient symptoms. In this article we discuss the end points, evidence, and analyses supporting cancer drug approvals based on evaluations of tumor-related signs and symptoms. With advice from the Oncologic Drug Advisory Committee (ODAC) in the late 1970s and early 1980s, FDA determined that acceptable end points for cancer drug approval were survival or an improvement in the quality of a patient's life, e.g., an improvement in tumor-related symptoms. This article summarizes 15 FDA cancer drug approvals based on patient symptom assessments and/or physical signs (thought to represent symptomatic improvement) as the primary evidence of effectiveness. These include painful bone events (three cases), cosmetic improvement in Kaposi's sarcoma and cutaneous T-cel...