统计在生命科学中的实践与认识(Ⅵ) 临床试验(上)

The Practice and Cognition of Statistics in Life Sciences(Ⅵ) Clinical trial(1)

本文回顾了临床试验发展的简史、随机临床试验的主要特征、临床试验实践中所面临的问题和困难。

Over the past 40 years, despite of the vigorous criticism, the randomized control trial has still become recognized as a scientific gold standard for evaluating the effectiveness of clinical therapy. After reviewing the history of clinical trial, the key components of it are discussed. They are randomized treatment assignment, blinding, adequate sample size, intent-to-treat analysis, and testing of prestated hypotheses on mature data. Several recent developments related to clinical trials: interim monitoring boundaries, large simple trials, multi-center and meta-analysis, quality of life, compliance, and behavioral aspects of clinical trials, are highlighted here. Major obstacles to conducting randomized clinical trials, including knowledge and attitudes of potential participants, reimbursement problems, informed consent limitations, the problems of equipoise, are also discussed.