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

Authors Martin Schumacher, Gabi Schulgen

Publication date 11 September 2002

Introductory exposition (textbooks, tutorial papers, etc.) pertaining to statistics (62-01) Research exposition (monographs, survey articles) pertaining to statistics (62-02) Applications of statistics to biology and medical sciences; meta analysis (62P10)

Review text

This multi-authored monograph presents on a total of 371 pages a comprehensive introduction into the methodology of clinical trials relevant for academic research and pharmaceutical drug development. The book is organized in 21 individual chapters, subordinated under five headings (Introduction, Analysis, Planning and Conduct, Qualiiy Requirements, Special Designs, Special Problems) and an Appendix. The Introduction is devoted to the basic rationale for the need of controlled and randomized clinical trials. The Analysis part provides the methods of statistical estimation and hypothesis testing in seven chapters organized by the type and the scale of the data (qualitative, quantitative, time to event) and it considers tests for equivalence, meta-analysis and the intent-to-treat analysis. The next core part of the book on the planning and the conduct of clinical trials considers general requirements of sample size calculation, randomization and blinding, interim analysis and also a very informative chapter on data documentation and management. Quality aspects for design, conduct and analysis are described in two chapters which emphasize current regulatory guidelines, e.g., the ICH E9, and the role of standard operating procedures. Four Special Designs are described: Phase I and Phase II studies, cross-over studies, diagnostic studies with special applications to breast cancer, and prognostic studies. The notorious problems with subgroup analyses and with multiple testing are treated in a clear and illustrative way in the last two chapters. Mathematical and statistical foundations are described in the Appendix and, finally, the CONSORT statement is presented as guidance document for the reporting and publication of study results. The book has been designed to be used for self-instruction as well as for the classroom and the individual chapters are written in a selfcontained mode to be read independently from each other. Examples are taken from the practice of the authors and their long lasting collaboration with clinical researchers.

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