新视角解读临床试验中的意向性原则

New perspectives on the principle of intention to treat in clinical trials

意向性原则(intention to treat,ITT)是以意向性治疗(即计划的治疗方案)为基础,而不以实际给予的治疗为基础进行评价的原则,在随机对照试验(randomized controlled trial,RCT)中广泛应用。随着临床试验的不断发展和进步,对ITT原则需要重新理解和诠释。在ITT原则的标准定义中,受试者的意向性分组并未特指随机化分组,而是计划的治疗方案,非随机化临床试验中受试者同意入组某一治疗方案同样为一种意向性。在单臂临床试验及非随机化的现实世界研究(real world study,RWS)中,应用ITT原则可评价由患者/医生决定的意向性治疗方案的有效性。在实际应用过程中,应根据研究目的结合ITT原则进行分组策略的综合考虑。

The intention to treat(ITT)principle is widely applied in the randomized controlled trials(RCTs),which is based on the intention to treat a subject(i.e.the planned treatment regimen)rather than the actual treatment given.With the development and progress of clinical trials,ITT principle needs to be reinterpreted.In the standard definition of ITT principle,the intentional grouping of subjects does not specifically refer to randomization,but rather to planned treatment regimen.In non-randomized clinical trials,that subjects agree to be included in a certain treatment regimen is also a type of intention.ITT principles can be applied to evaluate the effectiveness of treatment regimens under patient/doctor determined intentionality in single-arm clinical trials and non-randomized RWS(real world study,RWS).In the practical application,the grouping strategy based on ITT principle should be considered comprehensively according to the research purpose.