摘要在循证医学范畴内，随机对照临床试验（RCT）被认为是评价抗VEGF药物治疗眼底病效果的金标准。当以视力提升作为主要疗效评价指标时疗效水平很难有明显突破，使得非劣效性设计的RCT研究被广泛应用。在应用非劣效性研究设计时应重点关注：在验证新疗法疗效“不受损”的同时能否给患者带来其他方面的获益；界值的确定需要建立在临床意义及统计学意义综合考虑的基础上；传统的显著性检验方法并不适用于非劣效检验；非劣效性结论达成可继续追求优效性。本文将针对上述关注的热点问题进行深入阐述，为临床研究者在实际工作中科学开展研究设计、客观解读研究结果、正确指导临床实践提供帮助。 （中华眼科杂志，2020，56：895-898）

abstractsIn evidence-based medicine, randomized controlled clinical trials are considered as the gold standard to evaluate the curative effect of the anti-vascular endothelial growth factor drugs on fundus diseases. The non-inferiority design is widely used due to the existence of the "ceiling effect" if vision improvement is used as the primary outcome. It is critical to make several issues clear before non-inferiority studies are performed. For example, patients can benefit from the new therapy other than efficacy; the determination of the non-inferiority margin should be based on the comprehensive consideration of clinical and statistical meanings; the traditional significance test method is not suitable for non-inferiority tests; the superiority test could be further performed if the non-inferiority conclusion has been reached. This article aims to elaborate these hot issues and to provide guidance for clinical researchers to carry out research designs and interpret findings objectively and scientifically. (Chin J Ophthalmol, 2020, 56: 895-898)