Design and Analysis of Clinical Trials: Concepts and Methodologies

The authors have experience conducting clinical trials in the pharmaceutical industry and this shows in their approach. All the issues that arise in an NDA submission to the FDA are covered. In the introduction the authors describe the regulatory process and the role of the FDA. They even provide organizational charts for the FDA Center for Drug Evaluation and Research. I have been working as a senior biostatistician for medical device companies for the last 5 years. Most of the trials I have worked on were regulated by the FDA and a number of issues that have been important to the FDA include (1) multiple comparisons, (2) intention-to-treat versus per protocol analysis, (3) pooling centers, (4) baseline data and demographics, (5) data monitoring and (6) safety. They are all covered in chapters 11 and 12 of this book. Chapter 10 emphasizes sample size determination and interim analyses are covered in chapter 9. Randomization and blinding are covered in chapter 4. These topics are emphasized because of their importance in regulated clinical trials. One does not find them covered very much in other statistics texts on survival analysis or clinical trials. The mathematics level is intermediate. The authors write well and incorporate the important practical interplay between the statistician, the clinician and the physician. They provide many good references. The book is a good reference for anyone interested in clinical trials. Points are illustrated through the use of real trials. Recent advances in Bayesian methods, resampling and meta analysis are not covered but most important topics are covered including group sequential methods.