Key Statistical Concepts in Clinical Trials for Pharma
This Brief discusses key statistical concepts that facilitate the inferential analysis of data collected from a group of individuals participating in a pharmaceutical clinical trial, the estimation of their clinical significance in the general population
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For further volumes: http://www.springer.com/series/10224
J. Rick Turner
Key Statistical Concepts in Clinical Trials for Pharma
123
J. Rick Turner, Ph.D. Cardiac Safety Services, Quintiles Emperor Boulevard 4820 Durham, NC 27703 USA e-mail: [email protected]
ISSN 1864-8118 ISBN 978-1-4614-1661-6 DOI 10.1007/978-1-4614-1662-3
e-ISSN 1864-8126 e-ISBN 978-1-4614-1662-3
Springer New York Dordrecht Heidelberg London Library of Congress Control Number: 2011938148 Ó The Author(s) 2012 All rights reserved. This work may not be translated or copied in whole or in part without the written permission of the publisher (Springer Science+Business Media, LLC, 233 Spring Street, New York, NY 10013, USA), except for brief excerpts in connection with reviews or scholarly analysis. Use in connection with any form of information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed is forbidden. The use in this publication of trade names, trademarks, service marks, and similar terms, even if they are not identified as such, is not to be taken as an expression of opinion as to whether or not they are subject to proprietary rights. Printed on acid-free paper Springer is part of Springer Science+Business Media (www.springer.com)
Preface
The ultimate purpose of the results from a clinical trial is not to tell us precisely what happened in that trial, but, in combination with results from other trials in the drug’s clinical development program, to gain insight into likely drug responses in patients who would be prescribed the drug should it be approved for marketing. The discipline of Statistics enables us to do this. This book discusses key statistical concepts that facilitate the analysis of data collected from a group of individuals participating in a biopharmaceutical clinical trial, the estimation of their clinical significance in the general population of individuals likely to be prescribed the drug if approved, and the related decision making that occurs at both the public health level (by regulatory agencies when deciding whether or not to approve a new drug for marketing) and the individual patient level (by physicians and their patients when deciding whether or not the patient should be prescribed a drug that is on the market). These key concepts include drug safety and efficacy, clinical significance, statistical significance, and benefit-risk estimation. All of these facilitate decision making during drug development, and also during pharmacotherapy once the drug is marketed.
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Contents
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Setting the Scene . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1.1 Introduction . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1.2 The Discipline of Statistics . . . . . . . . . . . . . . . . . . . . . . . . 1.3 Generalizing Information Gained from a Clinical Trial . . . . 1.4 Blood Pressure . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1.4.1 The Physiology and Measurement
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