Methodology of clinical trials. Methodologic foundations of planning, execution and evaluation. (Q2509386)

This compendium about clinical trials is the second and enhanced edition of the firstly in 2002 appeared book, see the review 1010.62097. It gives an introduction into the methodology of clinical trials for researchers and students which is adequate for self-study and also for readers with a non-statistical background. The book deals with the different aspects of clinical trials: statistical analysis, planning and conduct of clinical trials, quality requirements, and presents also examples of special designs for clinical studies. The volume is subdivided into 22 chapters which refer among each other but can also be read separately. In the context of statistical analysis tools for different types of data (continuous, rates, survival data) are presented and equivalence studies, meta-analyses and the intention-to-treat principle are introduced. The part about planning and conduction of clinical trials explains sample size calculations (for dichotomous, normally distributed and event data), the principle of randomization and blinding, interim analyses and statistical monitoring of clinical trials as well as data management (e.g., handling patient data, examples for a query form and case report form (CRF), legal guidelines). In the context of quality control in planning and analysis during the clinical development process, important guidelines are introduced and discussed in detail like the declaration of Helsinki, the ICH E9 as well as ICH E3, E6 and E10 and some EMEA/CPMP guidelines. As special designs four examples are presented: Phase I and II studies, cross-over designs, the design and analysis of diagnosis studies (including choice of cut-points, the notions of prevalence, sensitivity and specificity, analysis with ROC curves) and prognosis studies. Special problems like subgroup analysis and multiple testing are addressed in separate chapters. The new edition contains also an own chapter about competing risks for a special case study of patients with type II diabetes. The appendix gives a comprehensible introduction into the elementary statistical notions for the reader without mathematical and statistical background. The potency of this multi-author volume lies in its practical orientation: Each topic is motivated or illustrated by a case study drawn of the authors' wide operating experience as clinical researchers. The reader can also find numerous references at the end of each chapter as well as hints to helpful websites and useful software.