Bringing new medicines to women with epithelial ovarian cancer: what is the unmet medical need?

BACKGROUND Therapy for advanced epithelial ovarian cancer (OC) includes first line platinum/taxane-containing chemotherapy and re-treatment with platinum-containing regimens for disease recurrence in patients likely to respond again. Single-agent, non-platinum, cytotoxic agents are commonly used to treat patients resistant to platinum retreatment, but these agents are associated with dose-limiting toxicities and response rates below 20%. MAIN BODY Recent advances have led to novel targeted treatments for recurrent OC that offer opportunities to improve response rates and prolong progression-free intervals. However, they also add complexity to the process of selecting treatment for individual patients at different stages of the disease process. Advanced and recurrent OC is rarely cured. Multiple lines of platinum combinations, and nonplatinum chemotherapeutics eventually fail to achieve clinical benefit, thus other active and tolerable systemic therapies are needed. Consequently, the US Food and Drug Administration has created a mechanism for "accelerated approval" of new medicines in situations of high unmet medical need. CONCLUSION We review the clinical implications of recent key clinical studies in these settings and outline the path forward for study design and approval of novel therapeutics to treat recurrent OC.