Djulbegovic B
Interdisciplinary Oncology Program, Division of Blood and Bone Marrow Transplantation, H. Lee Moffitt Cancer Center, University of South Florida, 12902 Magnolia Drive, Tampa, FL 33612, USA.
Curr Oncol Rep. 2001 Sep;3(5):389-95. doi: 10.1007/s11912-001-0024-5.
Recognition of the importance of uncertainty in the design of randomized, controlled trials (RCT) has reached the status of a principle. The "uncertainty principle," or less ambiguously, equipoise, holds that a patient should be enrolled in an RCT only if there is substantial uncertainty about which of the trial treatments would benefit the patient most. In fact, the "uncertainty principle" addresses the most important issue of a clinical trial--the choice of an adequate comparative control. Studies in which intervention and control group are believed to be non-equivalent violate the uncertainty principle. Therefore, one would expect that both editors and authors would be particularly careful to include a statement concerning prior beliefs of the investigator(s) about the uncertainty of the treatments that are reported. However, we found no evidence of such a policy in the randomized, controlled trials we examined. We also show that there is a predictable relationship between the uncertainty principle, that is, the moral principle upon which trials are based, and the ultimate outcomes of clinical trials. We postulate that about 50% of innovations are successful, leading to the conclusion that preserving the ethics of clinical research may be the best investment strategy available.
认识到在随机对照试验（RCT）设计中不确定性的重要性已上升为一项原则。“不确定性原则”，或者更明确地说， equipoise（平衡原则），认为只有当对于哪种试验治疗方法对患者最有益存在实质性不确定性时，患者才应被纳入RCT。事实上，“不确定性原则”解决了临床试验中最重要的问题——选择适当的对照。那些干预组和对照组被认为不相等的研究违反了不确定性原则。因此，人们会期望编辑和作者都格外谨慎地纳入一份关于研究者对所报告治疗方法不确定性的先验信念的声明。然而，在我们审查的随机对照试验中，我们没有发现这种政策的证据。我们还表明，不确定性原则（即试验所基于的道德原则）与临床试验的最终结果之间存在可预测的关系。我们推测大约50%的创新是成功的，从而得出结论：维护临床研究的伦理道德可能是现有的最佳投资策略。
