Statistical Essentials in the Design and Analysis of Clinical Trials
Usman Baber
Search for more papers by this authorMauro Chiarito
Search for more papers by this authorStuart J. Pocock
Search for more papers by this authorUsman Baber
Search for more papers by this authorMauro Chiarito
Search for more papers by this authorStuart J. Pocock
Search for more papers by this authorGeorge D. Dangas MD, MACC, MSCAI, FAHA, FESC
Professor of Medicine (Cardiology) & Surgery (Vascular) Professor of Cardiology Adjunct Professor of Internal Medicine
Icahn School of Medicine at Mount Sinai, Mount Sinai Hospital, New York, NY, USA
National Kapodistrian University of Athens, Greece
Medical University of Vienna, Austria
Search for more papers by this authorCarlo Di Mario MD, PhD, FRCP, FACC, FSCAI, FESC
Professor of Cardiology Director of the Structural Interventional Cardiology Division Honorary Consultant
University of Florence
University Hospital Careggi, Florence, Italy
Cardiologist Royal Brompton Hospital, London, UK
Search for more papers by this authorHolger Thiele MD
Professor of Cardiology at University of Leipzig
Heart Center Leipzig at University of Leipzig, Leipzig, Germany
Search for more papers by this authorPeter Barlis MBBS, MPH, PHD, FACC, FESC, FRACP
Professor of Cardiology Interventional Cardiologist
University of Melbourne, Melbourne, Victoria, Australia
St Vincent's & Northern Hospitals Victoria, Australia
Search for more papers by this authorSummary
The rapidly changing landscape of cardiology has been driven by an increasing wealth of clinical data within the scientific literature. Clinical decisions are therefore strongly influenced by the appropriate implementation of evidence-based medicine, requiring the clinician to have an understanding of clinical trial design, study methodology and commonly utilized biostatistical approaches. The chapter begins with succinct descriptions of fundamental statistical principles. Significance testing, the estimation of the magnitude of effect, and the interpretation of p-values are discussed, before the discussion of advanced techniques, such as the analysis of time to event data. This is followed by brief explanations of the basic principles of clinical trial design and planning, addressing issues of bias, sample size and power, and commonly used trial designs, concluding with a brief explanation of the principle of Bayesian statistic. Each topic is coupled with examples from published clinical trials.
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