临床试验的设计和解释

你将学到什么

课程概况

为了评价那些新的干预措施或者新的治疗效果，人们设计了临床试验。  接受评价的可以是药物，设备（如助听器），手术，行为干预（如戒烟计划），社区健康计划（如癌症筛查计划）或医疗保障体系（如医院的特殊监护病房）。  我们研究临床试验是因为给病人选择治疗项目的是研究者，而非病人自己或他们的医生。  随机临床试验的结果通常最能说明治疗是否有效，因为这些试验具有一些特征，它们能确保对治疗效果和风险的评价客观公正。  食品及药物管理局规定，药物和生物制剂（如疫苗）必须要在临床试验中被证明确有成效后才可投入市场。

本门课程将解释设计随机临床试验的基本原则以及如何报告试验结果。  在课程的前半部分中，教师将向学生介绍临床试验里用到的术语以及试验常用的设计模式，如平行和交叉设计。  我们还会解释进行临床试验的一些方法，像是随机和盲法治疗。 在课程的后半部分中， 我们将介绍如何分析和解读临床试验。 最后，我们将讨论在人身上做试验主要会涉及到哪些伦理道德方面的问题。

Clinical trials are experiments designed to evaluate new interventions to prevent or treat disease in humans. The interventions evaluated can be drugs, devices (e.g., hearing aid), surgeries, behavioral interventions (e.g., smoking cessation program), community health programs (e.g. cancer screening programs) or health delivery systems (e.g., special care units for hospital admissions). We consider clinical trials experiments because the investigators rather than the patients or their doctors select the treatment the patients receive. Results from randomized clinical trials are usually considered the highest level of evidence for determining whether a treatment is effective because trials incorporates features to ensure that evaluation of the benefits and risks of treatments are objective and unbiased. The FDA requires that drugs or biologics (e.g., vaccines) are shown to be effective in clinical trials before they can be sold in the US.

The course will explain the basic principles for design of randomized clinical trials and how they should be reported. In the first part of the course, students will be introduced to terminology used in clinical trials and the several common designs used for clinical trials, such as parallel and cross-over designs. We will also explain some of the mechanics of clinical trials, like randomization and blinding of treatment. In the second half of the course, we will explain how clinical trials are analyzed and interpreted. Finally, we will review the essential ethical consideration involved in conducting experiments on people.

课程大纲

周1
完成时间为 2 小时
Types of Trial Designs
This week, we explore different types of trial designs, including parallel, crossover, group allocation, factorial, large simple, equivalency,
non-inferiority, and adaptive designs.
5 个视频 （总计 56 分钟）, 3 个阅读材料, 1 个测验

周2
完成时间为 2 小时
Randomization and Masking
This week we discuss two key design features of randomized clinical trials used to protect against bias, randomization and masking.
3 个视频 （总计 67 分钟）, 1 个阅读材料, 1 个测验

周3
完成时间为 2 小时
Outcomes and Analysis
This week focuses on a key design issue - selecting the primary outcome. We will also cover the gold standard for analysis of clinical trials, which
is including all the participants in the analysis regardless of their actual treatment.
4 个视频 （总计 51 分钟）, 1 个阅读材料, 1 个测验

周4
完成时间为 2 小时
Ethics
This week focuses on a key issue in the field of clinical trials, the ethics of experimentation in humans.
2 个视频 （总计 17 分钟）, 6 个阅读材料, 1 个测验

周5
完成时间为 2 小时
Reporting Results
This week, we focus on reporting results of clinical trials in publications. We cover the Consolidated Standards of Reporting Trials (CONSORT)
guidelines.
3 个视频 （总计 31 分钟）, 3 个阅读材料, 1 个测验

周6
完成时间为 1 小时
Randomized Clinical Trials
This week, we focus on whether RCTs are still the gold standard for evaluating evidence.

预备知识

培养学生对健康科学的兴趣。  了解流行病学的基本知识，如偏差和统计数据的分类，平均值，标准差和假设验证等，将会对这门课有所帮助。

参考资料

劳伦斯·弗里德曼，柯特·弗伯格和大卫·德梅兹所编写的《临床试验基础》，是一本非常好的参考书籍，不过不做强制要求。我们将选择发布一些与课程内容有关的阅读材料。