Clinical trial endpoints in ovarian cancer: Report of an FDA/ASCO/AACR Public Workshop

• Published in: Gynecologic oncology Vol. 107; no. 2; pp. 173 - 176
• Main Authors: Bast, Robert C, Thigpen, J. Tate, Arbuck, Susan G, Basen-Engquist, Karen, Burke, Laurie B, Freedman, Ralph, Horning, Sandra J, Ozols, Robert, Rustin, Gordon J, Spriggs, David, Wenzel, Lari B, Pazdur, Richard
• Format: Journal Article Conference Proceeding
• Language: English
• Published: United States Elsevier Inc 01.11.2007
• Subjects: Antineoplastic Agents; Approval; Biomarkers, Tumor - blood; Biomedical Research; CA-125 Antigen - blood; CA125; Cancer; Clinical Trials as Topic; Disease-Free Survival; Drug Approval; Endpoint Determination; Endpoints; FDA; Female; Gynecology; Health Status; Hematology, Oncology and Palliative Medicine; Humans; Medical Oncology; Obstetrics and Gynecology; Ovarian; Ovarian Neoplasms - drug therapy; Patient Satisfaction; Quality of Life; Societies, Medical; Therapy; Treatment Outcome; United States; United States Food and Drug Administration; United States; Endpoints; Therapy; FDA; CA125; Ovarian; Research; Approval; Cancer
• ISSN: 0090-8258; 1095-6859
• DOI: 10.1016/j.ygyno.2007.08.092

Abstract Objective The unique characteristics of cancer, particularly issues involving the use of surrogate endpoints in clinical trials, present special challenges in the development of cancer drugs. In response, the U.S. Food and Drug Administration (FDA) has partnered with the American Society of Clinical Oncology, the American Association for Cancer Research, and the American Society of Hematology to conduct public workshops evaluating potential endpoints for drug approvals for the most common tumor types. Methods A workshop evaluating potential endpoints in ovarian cancer drug research was held in Bethesda, Maryland, in April 2006. Invited experts presented research findings and discussed endpoints in trials of drugs for treatment of Stage III and IV ovarian cancer. Results The panel responded to specific questions from FDA, discussing use of progression-free survival as a surrogate for overall survival and use of CA-125 levels as an indicator of response. Panel members also addressed endpoints in first-line therapy, second-line and subsequent therapy, and maintenance therapy. Conclusion Expert commentary provided by panel members will inform FDA's draft guidance on clinical endpoints for cancer drug approvals and will be discussed at meetings of the FDA's Oncologic Drugs Advisory Committee. FDA intends to develop a set of principles that can be used to define efficacy standards for drugs used to treat ovarian and other cancers.