等效性临床试验等效界值确定原则的探讨

Principles to specify equivalence margin in clinical trials

等效界值的确定是等效性试验设计中最为困难的部分,其确定方法必须保证试验措施相对于安慰剂的有效性,并且必须在分析既往研究的基础上结合统计学意义、临床意义、结局指标的选择、疾病具体特征,并考虑毒副作用、风险-效益的分析等事先做出。本文论述了等效性临床试验设计中确定等效界值的一般原则。

This paper deals with the principles and approaches to specify the equivalence margin in equivalenee or non-inferiority trials. It is a difficult work to define equivalenee boundary appropriately in elinieal trials design. The selection of equivalence margin should provide assuranee the effectiveness of experimental treatment relative to placebo firstly. Based on the analysis of previous studies, the statistical reasoning, clinical relevant, the choice of clinical outcome, the characteristics of particular diseases, toxicity, adverse effects, and risk-benefit assessment must be considered for equivalence margin pre-specified reasonably.