Fundamentals of Clinical Trials

Abstract

The clinical trial is “the most definitive tool for evaluation of the applicability of clinical research.” It represents “a key research activity with the potential to improve the quality of health care and control costs through careful comparison of alternative treatments” [1]. It has been called on many occasions, “the gold standard” against which all other clinical research is measured. Although many clinical trials are of high quality, a careful reader of the medical literature will notice that a large number have deficiencies in design, conduct, analysis, presentation, and/or interpretation of results. Improvements have occurred over the past few decades, but too many trials are still conducted without adequate attention to the fundamental principles. Certainly, numerous studies could have been improved if the authors had had a better understanding of the fundamentals. Since the publication of the first edition of this book in 1981, a large number of other texts on clinical trials have appeared, most of which are indicated here [2–21]. Several of them, however, discuss only specific issues involved in clinical trials. Additionally, many are no longer current. The purpose of this fifth edition is to update areas in which major progress has been made since the publication of the fourth edition. We have revised most chapters considerably. Because it was becom- ing unwieldy, we divided the chapter on monitoring response variables into two chapters, one on monitoring committees and the other on monitoring approaches. We also added a chapter on regulatory issues.