Download or read book Statistical Validation of Surrogate Endpoints Using Equivalence Testing written by Sanatan Saraf and published by . This book was released on 2013 with total page 152 pages. Available in PDF, EPUB and Kindle. Book excerpt: In randomized clinical trials, the evaluation of potential surrogate endpoints is very important, since a successful surrogate endpoint will reduce follow-up trial time and/or will reduce the number of patients needed to establish a certain treatment effect. We investigate the statistical validation of different types of surrogate endpoints: continuous and binary.

Download or read book The Evaluation of Surrogate Endpoints written by Tomasz Burzykowski and published by Springer Science & Business Media. This book was released on 2005-11-24 with total page 417 pages. Available in PDF, EPUB and Kindle. Book excerpt: Covers the latest research on a sensitive and controversial topic in a professional and well researched manner. Provides practical outlook as well as model guidelines and software tools that should be of interest to people who use the software tools described and those who do not. Related title by Co-author Geert Molenbergh has sold more than 3500 copies world wide. Provides dual viewpoints: from scientists in the industry as well as regulatory authorities.

Download or read book Validation of Surrogate Endpoints by Bayesian Equivalence Testing written by Mathangi Gopalakrishnan and published by . This book was released on 2013 with total page 180 pages. Available in PDF, EPUB and Kindle. Book excerpt: Surrogate endpoints are often used in clinical trials to serve as a substitute for a hard to achieve clinically meaningful endpoint. The surrogate endpoint is expected to predict the effect of the treatment on the true endpoint.

Download or read book Statistical Evaluation of Surrogate Markers in Randomized Clinical Trials written by Xiaopeng Miao and published by . This book was released on 2012 with total page 258 pages. Available in PDF, EPUB and Kindle. Book excerpt: Abstract: In many randomized clinical trials, the primary endpoints are clinical measurements of disease process. For example, the survival time is the gold standard clinical endpoint in most cancer trials. It usually requires an extremely long follow-up period and a considerable sample size to assess the treatment effect on such clinical endpoints. Therefore surrogate markers that can predict the treatment effects on the clinical endpoints would be extremely useful in accelerating the drug development process and depicting the mechanisms of drug action. Although candidate surrogate markers are generally proposed based on biological considerations, their validation largely depends on statistical methods. There are two major statistical frameworks of evaluating candidate surrogate markers in a single-trial setting: one is called the statistical surrogate (Prentice, 1989) and the other is referred to as the principal surrogate (Frangakis and Rubin, 2002). Both frameworks define surrogacy based on the treatment effect on the clinical endpoint that is mediated through the surrogate marker. For the evaluation of statistical surrogates, most existing methods are based on parametric regression analyses, which might provide spurious inference in the presence of model misspecification. For the evaluation of principal surrogates, the applications of existing methods are restricted to some simplified contexts (e.g, HIV vaccine trials) or limited types of clinical endpoints (e.g., binary). In this dissertation, we develop two novel approaches for the evaluation of surrogate markers in randomized clinical trials. In the framework of statistical surrogacy, we develop a nonparametric testing procedure based on measure of divergence and random permutation. The proposed method is robust to model misspecification and influential points, and is applicable to a variety of settings. In the framework of principal surrogacy, we propose a multiple imputation approach based on the incorporation of baseline predictors. The proposed approach can accommodate different types of clinical endpoints, and can be used to evaluate principal surrogates in a general setting where most existing methods are not applicable. Extensive simulation studies are conducted to examine the performance of the proposed methods. The usefulness of the methods is further illustrated by real examples in clinical trials.

Download or read book Statistical Methods for Identifying Surrogate Endpoints in Vaccine Trials written by Julian Wolfson and published by . This book was released on 2009 with total page 152 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease written by Institute of Medicine and published by National Academies Press. This book was released on 2010-06-25 with total page 335 pages. Available in PDF, EPUB and Kindle. Book excerpt: Many people naturally assume that the claims made for foods and nutritional supplements have the same degree of scientific grounding as those for medication, but that is not always the case. The IOM recommends that the FDA adopt a consistent scientific framework for biomarker evaluation in order to achieve a rigorous and transparent process.

Download or read book Pediatric Drug Development written by Andrew E. Mulberg and published by John Wiley & Sons. This book was released on 2013-05-20 with total page 782 pages. Available in PDF, EPUB and Kindle. Book excerpt: Most medicines have never been adequately tested for safety and efficacy in pediatric populations and preterm, infants and children are particularly vulnerable to adverse drug reactions. Pediatric Drug Development: Concepts and Applications, Second Edition, addresses the unique challenges in conducting effective drug research and development in pediatric populations. This new edition covers the legal and ethical issues of consent and assent, the additional legal and safety protections for children, and the appropriate methods of surveillance and assessment for children of varying ages and maturity, particularly for patient reported outcomes. It includes new developments in biomarkers and surrogate endpoints, developmental pharmacology and other novel aspects of global pediatric drug development. It also encompasses the new regulatory initiatives across EU, US and ROW designed to encourage improved access to safe and effective medicines for children globally. From an international team of expert contributors Pediatric Drug Development: Concepts and Applications is the practical guide to all aspects of the research and development of safe and effective medicines for children.

Download or read book Introduction to Statistical Methods for Clinical Trials written by Thomas D. Cook and published by CRC Press. This book was released on 2007-11-19 with total page 465 pages. Available in PDF, EPUB and Kindle. Book excerpt: Clinical trials have become essential research tools for evaluating the benefits and risks of new interventions for the treatment and prevention of diseases, from cardiovascular disease to cancer to AIDS. Based on the authors’ collective experiences in this field, Introduction to Statistical Methods for Clinical Trials presents various statistical topics relevant to the design, monitoring, and analysis of a clinical trial. After reviewing the history, ethics, protocol, and regulatory issues of clinical trials, the book provides guidelines for formulating primary and secondary questions and translating clinical questions into statistical ones. It examines designs used in clinical trials, presents methods for determining sample size, and introduces constrained randomization procedures. The authors also discuss how various types of data must be collected to answer key questions in a trial. In addition, they explore common analysis methods, describe statistical methods that determine what an emerging trend represents, and present issues that arise in the analysis of data. The book concludes with suggestions for reporting trial results that are consistent with universal guidelines recommended by medical journals. Developed from a course taught at the University of Wisconsin for the past 25 years, this textbook provides a solid understanding of the statistical approaches used in the design, conduct, and analysis of clinical trials.

Download or read book Strategy and Statistics in Clinical Trials written by Joseph Tal and published by Academic Press. This book was released on 2011-06-26 with total page 278 pages. Available in PDF, EPUB and Kindle. Book excerpt: Strategy and Statistics in Clinical Trials deals with the research processes and the role of statistics in these processes. The book offers real-life case studies and provides a practical, how to guide to biomedical R&D. It describes the statistical building blocks and concepts of clinical trials and promotes effective cooperation between statisticians and important other parties. The discussion is organized around 15 chapters. After providing an overview of clinical development and statistics, the book explores questions when planning clinical trials, along with the attributes of medical products. It then explains how to set research objectives and goes on to consider statistical thinking, estimation, testing procedures, and statistical significance, explanation and prediction. The rest of the book focuses on exploratory and confirmatory clinical trials; hypothesis testing and multiplicity; elements of clinical trial design; choosing trial endpoints; and determination of sample size. This book is for all individuals engaged in clinical research who are interested in a better understanding of statistics, including professional clinical researchers, professors, physicians, and researchers in laboratory. It will also be of interest to corporate and government laboratories, clinical research nurses, members of the allied health professions, and post-doctoral and graduate students. - Enables non-statisticians to better understand research processes and statistics' role in these processes - Offers real-life case studies and provides a practical, "how to" guide to biomedical R&D - Delineates the statistical building blocks and concepts of clinical trials - Promotes effective cooperation between statisticians and important other parties

Download or read book Small Clinical Trials written by Institute of Medicine and published by National Academies Press. This book was released on 2001-01-01 with total page 221 pages. Available in PDF, EPUB and Kindle. Book excerpt: Clinical trials are used to elucidate the most appropriate preventive, diagnostic, or treatment options for individuals with a given medical condition. Perhaps the most essential feature of a clinical trial is that it aims to use results based on a limited sample of research participants to see if the intervention is safe and effective or if it is comparable to a comparison treatment. Sample size is a crucial component of any clinical trial. A trial with a small number of research participants is more prone to variability and carries a considerable risk of failing to demonstrate the effectiveness of a given intervention when one really is present. This may occur in phase I (safety and pharmacologic profiles), II (pilot efficacy evaluation), and III (extensive assessment of safety and efficacy) trials. Although phase I and II studies may have smaller sample sizes, they usually have adequate statistical power, which is the committee's definition of a "large" trial. Sometimes a trial with eight participants may have adequate statistical power, statistical power being the probability of rejecting the null hypothesis when the hypothesis is false. Small Clinical Trials assesses the current methodologies and the appropriate situations for the conduct of clinical trials with small sample sizes. This report assesses the published literature on various strategies such as (1) meta-analysis to combine disparate information from several studies including Bayesian techniques as in the confidence profile method and (2) other alternatives such as assessing therapeutic results in a single treated population (e.g., astronauts) by sequentially measuring whether the intervention is falling above or below a preestablished probability outcome range and meeting predesigned specifications as opposed to incremental improvement.

Download or read book Oxford Textbook of Oncology written by David J. Kerr and published by Oxford University Press. This book was released on 2016 with total page 1041 pages. Available in PDF, EPUB and Kindle. Book excerpt: This textbook provides current information on best practice in multidisciplinary cancer care. Divided into six sections, the contributors look at the aetiology of cancer, patient care, population health and thethe management of specific types of disease. Written and edited by internationally recognised leaders in the field, the new edition of the Oxford Textbook of Oncology has been fully revised and updated, taking into consideration the advancements in each of the major therapeutic areas, and representing the multidisciplinary management of cancer. Structured in six sections, the book provides an accessible scientific basis to the key topics of oncology, examining how cancer cells grow and function, as well as discussing the aetiology of cancer, and the general principles governing modern approaches to oncology treatment. The book examines the challenges presented by the treatment of cancer on a larger scale within population groups, and the importance of recognising and supporting the needs of individual patients, both during and after treatment. A series of disease-oriented, case-based chapters, ranging from acute leukaemia to colon cancer, highlight the various approaches available for managing the cancer patient, including the translational application of cancer science in order to personalise treatment. The advice imparted in these cases has relevance worldwide, and reflects a modern approach to cancer care. The Oxford Textbook of Oncology provides a comprehensive account of the multiple aspects of best practice in the discipline, making it an indispensable resource for oncologists of all grades and subspecialty interests. Review: Each chapter is nicely illustrated with schemes, cartoons and images. The text, although written by top oncologists, is readily understandable for a non-expert. Thus, the textbook will no doubt be appreciated by a broader audience. * Recent Patents on Anti-Cancer Drug Discovery Vol. 11 Issue No. 4, Alexander Shtil * I recommend this book highly to all oncology and oncologists in training as a thorough, informative, and readable reference. Every large intuitional library and every oncology library should have it. * NEJM, 2002 * This comprehensive textbook of oncology is the first new major textbook on cancer to appear in a decade and is designed for a broad audience of clinicians, oncologists in training, and academics. The coverage is comprehensive...The overall appearance of the book is outstanding. It is a welcome combination of epidemiology, aspects of basic science, pharmacology and radiation therapy that trainees will fine a nice change...should enjoy a wide readership...because of its appealing design and comprehensive approach to oncology. It is the most user-friendly comprehensive text currently available. The pathology, basic science, epidemiology, and radiation therapy sections are all presented with extreme clarity. * Doody's Journal , 2002 * A landmark reference...It sets new standards for publishing in oncology offering a ground-breaking innovative approach to the filed combined with the quality, accuracy , and intellectual rigour you have come to expect from the world's most prestigious reference publisher. * Biomedicine and Pharmacotherapy, 2002 * Under new editorship, the second edition is far more than an updated version of the first...the prose in the Oxford Textbook is exemplary...this textbook is unique among its peers in giving the sense that the authors are addressing the reader personally...an exception level of quality...Respect for the evidence-based medicine is apparent throughout the text...illustrative and anatomical drawing...of remarkable high quality...excellent discussion of doctor-patient communication in relation OT genetic counselling, psychological issues, and terminal cancers. * JAMA, Volume 287, Issue 24, 2002 * The Oxford Textbook of Oncology covers virtually the entire spectrum of malignant diseases in adults and children. It meets very high editorial and production standards: the organization, illustrations, and eye-pleasing typography are outstanding... I have high praise for this textbook. * NEJM, Volume 347, Number 2, 2002 * Review from previous edition The Oxford Textbook of Oncology is a classic and fresh approach to the field. It is a must for all libraries and all those who like to have a single up-to-date reference book that contains sufficient detail for the clinician in all subspecialties: surgery and chapters are sufficiently details to provide a reference for trainees in the field. * Oncology, Volume 63, 2002 * The Oxford Textbook of Oncology is what it is meant to be: a textbook with comprehensive information of the actual status of oncology... an indispensable and attractive source of information. * Professor Jaak Ph. Janssens, European Journal of Cancer Prevention * This volume provides a comprehensive account of the multiple aspects of best practice in the discipline, making it an indispensable resource for oncologists of all grades and subspecialty interests. * Anticancer Research Vol. 36 (2016) * An outstanding gift to the international scientific community... The new textbook is an excellent demonstration of this multifaceted and astonishingly variable problem, as well as of the latest achievements in its understanding and practical management. * Alexander A. Shtil, Recent Patents on Anticancer Drug Discovery * I would recommend anyone considering buying an oncology textbook, and particularly those who work in oncology support services, to consider this textbook as it is well set out, easy to read, easy to comprehend, and covers all of the important aspects of modern day oncology. * Dr Andrew Davies, Consultant in Palliative Medicine, Royal Surrey County Hospital; Review for Supportive Care in Cancer *

Download or read book Applied Surrogate Endpoint Evaluation Methods with SAS and R written by Ariel Alonso and published by CRC Press. This book was released on 2016-11-30 with total page 396 pages. Available in PDF, EPUB and Kindle. Book excerpt: An important factor that affects the duration, complexity and cost of a clinical trial is the endpoint used to study the treatment’s efficacy. When a true endpoint is difficult to use because of such factors as long follow-up times or prohibitive cost, it is sometimes possible to use a surrogate endpoint that can be measured in a more convenient or cost-effective way. This book focuses on the use of surrogate endpoint evaluation methods in practice, using SAS and R.

Download or read book Principles and Practice of Clinical Trials written by Steven Piantadosi and published by Springer Nature. This book was released on 2022-07-19 with total page 2573 pages. Available in PDF, EPUB and Kindle. Book excerpt: This is a comprehensive major reference work for our SpringerReference program covering clinical trials. Although the core of the Work will focus on the design, analysis, and interpretation of scientific data from clinical trials, a broad spectrum of clinical trial application areas will be covered in detail. This is an important time to develop such a Work, as drug safety and efficacy emphasizes the Clinical Trials process. Because of an immense and growing international disease burden, pharmaceutical and biotechnology companies continue to develop new drugs. Clinical trials have also become extremely globalized in the past 15 years, with over 225,000 international trials ongoing at this point in time. Principles in Practice of Clinical Trials is truly an interdisciplinary that will be divided into the following areas: 1) Clinical Trials Basic Perspectives 2) Regulation and Oversight 3) Basic Trial Designs 4) Advanced Trial Designs 5) Analysis 6) Trial Publication 7) Topics Related Specific Populations and Legal Aspects of Clinical Trials The Work is designed to be comprised of 175 chapters and approximately 2500 pages. The Work will be oriented like many of our SpringerReference Handbooks, presenting detailed and comprehensive expository chapters on broad subjects. The Editors are major figures in the field of clinical trials, and both have written textbooks on the topic. There will also be a slate of 7-8 renowned associate editors that will edit individual sections of the Reference.

Download or read book Biomarkers for Traumatic Brain Injury written by Svetlana Dambinova and published by Royal Society of Chemistry. This book was released on 2012 with total page 247 pages. Available in PDF, EPUB and Kindle. Book excerpt: Due to injuries sustained in sports and in combat, interest in traumatic brain injury (TBI) has never been greater. This book will fulfill a gap in understanding of what is occurring in the brain following injury that can subsequently be detected in biological fluids and imaging.

Download or read book Controversial Statistical Issues in Clinical Trials written by Shein-Chung Chow and published by CRC Press. This book was released on 2011-06-15 with total page 614 pages. Available in PDF, EPUB and Kindle. Book excerpt: In clinical trial practice, controversial statistical issues inevitably occur regardless of the compliance with good statistical practice and good clinical practice. But by identifying the causes of the issues and correcting them, the study objectives of clinical trials can be better achieved. Controversial Statistical Issues in Clinical Trials covers commonly encountered controversial statistical issues in clinical trials and, whenever possible, makes recommendations to resolve these problems. The book focuses on issues occurring at various stages of clinical research and development, including early-phase clinical development (such as bioavailability/bioequivalence), bench-to-bedside translational research, and late-phase clinical development. Numerous examples illustrate the impact of these issues on the evaluation of the safety and efficacy of the test treatment under investigation. The author also offers recommendations regarding possible resolutions of the problems. Written by one of the preeminent experts in the field, this book provides a useful desk reference and state-of-the art examination of problematic issues in clinical trials for scientists in the pharmaceutical industry, medical/statistical reviewers in government regulatory agencies, and researchers and students in academia.

Download or read book Encyclopedia of Biopharmaceutical Statistics Four Volume Set written by Shein-Chung Chow and published by CRC Press. This book was released on 2018-09-03 with total page 2434 pages. Available in PDF, EPUB and Kindle. Book excerpt: Since the publication of the first edition in 2000, there has been an explosive growth of literature in biopharmaceutical research and development of new medicines. This encyclopedia (1) provides a comprehensive and unified presentation of designs and analyses used at different stages of the drug development process, (2) gives a well-balanced summary of current regulatory requirements, and (3) describes recently developed statistical methods in the pharmaceutical sciences. Features of the Fourth Edition: 1. 78 new and revised entries have been added for a total of 308 chapters and a fourth volume has been added to encompass the increased number of chapters. 2. Revised and updated entries reflect changes and recent developments in regulatory requirements for the drug review/approval process and statistical designs and methodologies. 3. Additional topics include multiple-stage adaptive trial design in clinical research, translational medicine, design and analysis of biosimilar drug development, big data analytics, and real world evidence for clinical research and development. 4. A table of contents organized by stages of biopharmaceutical development provides easy access to relevant topics. About the Editor: Shein-Chung Chow, Ph.D. is currently an Associate Director, Office of Biostatistics, U.S. Food and Drug Administration (FDA). Dr. Chow is an Adjunct Professor at Duke University School of Medicine, as well as Adjunct Professor at Duke-NUS, Singapore and North Carolina State University. Dr. Chow is the Editor-in-Chief of the Journal of Biopharmaceutical Statistics and the Chapman & Hall/CRC Biostatistics Book Series and the author of 28 books and over 300 methodology papers. He was elected Fellow of the American Statistical Association in 1995.

Download or read book Adaptive Design Theory and Implementation Using SAS and R Second Edition written by Mark Chang and published by CRC Press. This book was released on 2014-12-01 with total page 709 pages. Available in PDF, EPUB and Kindle. Book excerpt: Get Up to Speed on Many Types of Adaptive Designs Since the publication of the first edition, there have been remarkable advances in the methodology and application of adaptive trials. Incorporating many of these new developments, Adaptive Design Theory and Implementation Using SAS and R, Second Edition offers a detailed framework to understand the use of various adaptive design methods in clinical trials. New to the Second Edition Twelve new chapters covering blinded and semi-blinded sample size reestimation design, pick-the-winners design, biomarker-informed adaptive design, Bayesian designs, adaptive multiregional trial design, SAS and R for group sequential design, and much more More analytical methods for K-stage adaptive designs, multiple-endpoint adaptive design, survival modeling, and adaptive treatment switching New material on sequential parallel designs with rerandomization and the skeleton approach in adaptive dose-escalation trials Twenty new SAS macros and R functions Enhanced end-of-chapter problems that give readers hands-on practice addressing issues encountered in designing real-life adaptive trials Covering even more adaptive designs, this book provides biostatisticians, clinical scientists, and regulatory reviewers with up-to-date details on this innovative area in pharmaceutical research and development. Practitioners will be able to improve the efficiency of their trial design, thereby reducing the time and cost of drug development.