Now viewed as its own scientific discipline, clinical trial methodology encompasses the methods required for the protection of participants in a clinical trial and the methods necessary to provide a valid inference about the objective of the trial. Drawing from the authors’ courses on the subject as well as the first author’s more than 30 years working in the pharmaceutical industry, Clinical Trial Methodology emphasizes the importance of statistical thinking in clinical research and presents the methodology as a key component of clinical research.

From ethical issues and sample size considerations to adaptive design procedures and statistical analysis, the book first covers the methodology that spans every clinical trial regardless of the area of application. Crucial to the generic drug industry, bioequivalence clinical trials are then discussed. The authors describe a parallel bioequivalence clinical trial of six formulations incorporating group sequential procedures that permit sample size re-estimation. The final chapters incorporate real-world case studies of clinical trials from the authors’ own experiences. These examples include a landmark Phase III clinical trial involving the treatment of duodenal ulcers and Phase III clinical trials that contributed to the first drug approved for the treatment of Alzheimer’s disease.

Aided by the U.S. FDA, the U.S. National Institutes of Health, the pharmaceutical industry, and academia, the area of clinical trial methodology has evolved over the last six decades into a scientific discipline. This guide explores the processes essential for developing and conducting a quality clinical trial protocol and providing quality data collection, biostatistical analyses, and a clinical study report, all while maintaining the highest standards of ethics and excellence.

Reviews

This comprehensive text introduces the key areas of clinical trial methodology from the perspective of the biostatistician in the pharma industry. … Throughout, the text benefits from a highly structured and logical flow … the arguments made in the book are grounded in many years of practical experience in drug development and at the very least will act as a prompt for in-depth discussion or critical review of one’s own perceptions. … Clinical Trial Methodology will be of substantial value to early career pharmaceutical industry statisticians.

—Christopher J. Weir, Pharmaceutical Statistics, 2012

… informative discussions of mechanisms such as IND and NDA … are unique strengths of this book, distinguishing it from the many other clinical trial texts available. … Case studies … are presented carefully … The authors’ writing style is disciplined, careful, and informative. … this is a helpful and informative book, a nice reference to have for most biostatisticians working on clinical trials.

—Mithat Gönen, Journal of Biopharmaceu...