Fundamentals of Biostatistics：生物统计学基础.ppt

Basic Statistical Concepts Fundamentals of Biostatistics Lecture 2 Clinical Trials Validity/Reliability Assessing Evidence Randomized Clinical Trials (RCT) Randomization first used by RA Fisher in agriculture expts in 1920s First clinical trial using randomization in 1931 by Anderson on use of sanocrysin on TB patients. Also, first trial using blinding. Placebo first used in RCT in 1938 in cold vaccine trial. Randomized Clinical Trials (RCT) Fundamental Point: A properly planned clinical trial is a powerful expt’l technique for assessing effectiveness of a drug or intervention. Defn: A clinical trial is a prospective study comparing the effect of intervention(s) against a control in humans. Randomized Clinical Trials (RCT) Phases of clinical trials: Phase I: Determine tolerance of a compound in humans (i.e., how large a dose can be given until unacceptable toxicity?). Phase II: Evaluation of biologic activity or effect, and estimate rate of adverse events. Phase III: Definitive comparative trial. Designed to determine effectiveness and its role in clinical practice. Randomized Clinical Trials (RCT) Terminology Efficacy: How well an intervention works in an ideal setting. Effectiveness: How well an intervention works in actual practice. Phase IV: Evaluation of long-term saftey of an intervention believed to be effective in phase III trials. Randomized Clinical Trials (RCT) Why are clinical trials needed? Because evaluating the effectiveness of a treatment using uncontrolled observations is very difficult, since other factors affecting treatment outcome may not be balanced in treatment groups. Randomized Clinical Trials (RCT) Advantages of RCT: Groups more comparable b/c confounding variables are balanced Ability to detect small effects Most stat tests based on assumption of random allocation of pts to trt groups (validity) Randomized Clinical Trials (RCT) Disadvantages of RCT: Expensive and time consuming Subject pool may not be represent