A Note on Design and Analysis of Clinical Trials

Most countries have adopted the good clinical practice (GCP) principles as laws and regulations for approving clinical trials which are conducted to test new methods to prevent, detect, and treat disease or drug development. Most regulatory agencies such as the United State Food and Drug Administration (FDA) indicate that only adequate and well-controlled clinical trials can provide substantial evidence of safety and effectiveness of the test treatment under investigation. FDA indicates that “substantial evidence” can only be provided through “adequate and wellcontrolled” clinical investigations which include valid study design and appropriate statistical methods for data analysis. In this article, we are going to highlight the key points in design and analysis of clinical trials. We will also review the current research and point out the issues for future research. Few commonly employed study designs and its concepts in clinical trials will be described. In addition, statistical methods for data analysis on efficacy and safety are reviewed. To maintain the integrity and quality of the intended trial, Good Clinical Practice (GCP) and independent Data Monitoring Committee (DMC) are also discussed. Recent development such as biomarker development for target clinical trial, translational research/medicine, and traditional Chinese medicine are also provided.