Maurie Markman, MD
From the perspective of a patient and that patient’s family, it is completely understandable that the single most important goal of an antineoplastic strategy is to prolong survival and, if possible, produce a cure. Further, it is reasonable to argue that this basic philosophy of the aim of treatment has been quite nicely translated into the regulatory environment of drug development, where overall survival (OS) has traditionally been considered the definitive as well as gold standard outcome and endpoint in cancer therapeutic clinical trials.
Biologically and clinically active antineoplastic drug regimens employed as a component of initial therapy (adjuvant or neoadjuvant strategies, metastatic disease) or as second-line or later approaches in disease management have favorably affected both quantity and quality of life. In an increasing number of settings, patients with an advanced or metastatic cancer, while still unable to be cured, are objectively able to lead a high-quality life for a number of years rather than months or even less time. As a result, it is not unreasonable to label such long-term conditions as very serious but chronic. But what does this discussion have to do with endpoints in cancer clinical trials and, specifically, in epithelial ovarian cancer? There are 2 quite powerful arguments that can be provided in response to this highly relevant question.
Higher Inputs for Reliable Data
The first argument is related to how subsequent treatments on posttrial outcomes and survival have affected the way clinical trials are conducted in the current oncology arena and will be conducted in the future. Perhaps the most definitive argument against a disturbingly senseless belief that OS must always remain the gold standard in cancer clinical trials comes from a landmark analysis by a leading group of cancer biostatisticians.
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