Assessing the Measure of a New Drug: Is Survival the Only Thing That Matters?

摘要: Clinical research to improve breast cancer treatment has produced multiple, meaningful advances in the last decade. New antiestrogen and chemotherapeutic agents—novel therapies directed towards HER2—such as trastuzumab lapatinib, antiangiogenic agents, such bevacizumab, have all been added clinician’s armamentarium treat metastatic disease. 1 Several of these now demonstrated efficacy adjuvant setting, which raises important question: What exactly improved agents introduced into setting? Given current limited possibility cure for patients, there are two clinically goals treatment: make patient live longer delay or relieve symptoms. The first end points, codified overall survival (OS), is simple measure, unambiguous, unquestionable clinical relevance. However, symptom relief palliation (or quality life), despite recent progress, notoriously difficult measure. 2 Ideally, a newly antineoplastic agent will demonstrate significant prolongation setting randomized controlled trial. use primary point trial several major drawbacks. First, because mortality occurs after relatively long time most statistically differences OS require large numbers patients years reliably detect. For example, recently reported women with were randomly assigned receive either bevacizumab no addition first-line paclitaxel, median population was not reached until nearly 8 opened, full initial results (based on an progression-free [PFS]) made public. 3 Second, many new control arm allowed cross over investigational off-study disease progression. This strategy dilutes effects OS, since both arms at some point. Although prohibition cross-over scientifically appealing, ethical considerations often preclude it, particularly if outcomes earlier points found be promising drug approved later line settings. Finally, agent, may more detect than past due beneficial other available cancer.