Fueglistaler Philipp, Adamina Michel, Guller Ulrich
Department of Surgery, Divisions of General Surgery and Surgical Research, University of Basel, Basel, Switzerland.
Ann Surg Oncol. 2007 May;14(5):1532-9. doi: 10.1245/s10434-006-9295-2. Epub 2007 Feb 9.
The classical randomized controlled clinical trial is designed to prove superiority of an investigational therapy over an established therapy or placebo (here referred to as "superiority trial"). Although the randomized controlled superiority trial has its well-grounded role, clinical trials of non-inferiority are equally important in the advance of medical science. Non-inferiority trials test whether a new intervention is as good as a standard treatment with respect to curing the illness (e.g., overall survival) while offering other benefits over the standard therapy, such as lower toxicity, better side-effect profile, improved ease of administration, or reduced costs. The evaluation of non-inferiority is critical in many settings. In surgical oncology, for instance, treatments often combine advantages (e.g., survival benefit) with disadvantages (e.g., high post-operative morbidity due to extensive surgery, considerable toxic effects of an aggressive chemotherapy regimen). The various aspects of different therapeutic strategies may make a treatment decision difficult, requiring a non-inferiority trial to quantify risks and benefits. However, despite their great importance in clinical cancer research, the concept, design, and objectives of non-inferiority trials remain poorly understood in the surgical community. The goal of this review is to discuss the principles, strengths, and challenges of non-inferiority trials and introduce this highly relevant topic to the surgical reader, using examples from the field of surgical oncology.
经典的随机对照临床试验旨在证明一种研究性疗法优于既定疗法或安慰剂（此处称为“优效性试验”）。尽管随机对照优效性试验有其合理的作用，但非劣效性临床试验在医学科学发展中同样重要。非劣效性试验检验一种新干预措施在治愈疾病（如总生存期）方面是否与标准治疗一样好，同时相对于标准疗法具有其他益处，如更低的毒性、更好的副作用特征、更高的给药便利性或更低的成本。在许多情况下，非劣效性评估至关重要。例如，在外科肿瘤学中，治疗往往兼具优点（如生存获益）和缺点（如因广泛手术导致的高术后发病率、积极化疗方案的相当大的毒性作用）。不同治疗策略的各个方面可能使治疗决策变得困难，需要进行非劣效性试验来量化风险和益处。然而，尽管非劣效性试验在临床癌症研究中非常重要，但外科界对其概念、设计和目标仍知之甚少。本综述的目的是讨论非劣效性试验的原则、优势和挑战，并以外科肿瘤学领域的实例向外科读者介绍这一高度相关的主题。
