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The Design of Studies for Medical Research

Author: David Machin

Publisher: John Wiley & Sons

Published: 2005-05-13

Total Pages: 286

ISBN-13: 0470012986

The same careful rigour imposed on the design of phase III randomised controlled trials is not always applied to medical research in other areas such as trials conducted at earlier stages of drug development. With the emphasis that is now placed on evidence-based medicine, such care and rigour will inevitably impact on these areas with increasing attention turned to the quality of design. This title describes what principles can be used to structure research effectively allowing for the required degree of accuracy. Written by two best selling authors, this book includes many examples from medical literature and will be of great value to all groups conducting studies at the interface of clinical and laboratory research.

Small Clinical Trials

Author: Institute of Medicine

Publisher: National Academies Press

Published: 2001-01-01

Total Pages: 221

ISBN-13: 0309171148

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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.

Designing Clinical Research

Author: Stephen B. Hulley

Publisher: Lippincott Williams & Wilkins

Published: 2011-11-30

Total Pages: 388

ISBN-13: 1451165854

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Designing Clinical Research sets the standard for providing a practical guide to planning, tabulating, formulating, and implementing clinical research, with an easy-to-read, uncomplicated presentation. This edition incorporates current research methodology—including molecular and genetic clinical research—and offers an updated syllabus for conducting a clinical research workshop. Emphasis is on common sense as the main ingredient of good science. The book explains how to choose well-focused research questions and details the steps through all the elements of study design, data collection, quality assurance, and basic grant-writing. All chapters have been thoroughly revised, updated, and made more user-friendly.

Design and Analysis of Quality of Life Studies in Clinical Trials

Author: Diane L. Fairclough

Publisher: CRC Press

Published: 2002-03-28

Total Pages: 332

ISBN-13: 9781584882633

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More and more frequently, clinical trials include the evaluation of Health-Related Quality of Life (HRQoL), yet many investigators remain unaware of the unique measurement and analysis issues associated with the assessment of HRQoL. At the end of a study, clinicians and statisticians often face challenging and sometimes insurmountable analytic problems. Design and Analysis of Quality of Life Studies in Clinical Trials details these issues and presents a range of solutions. Written from the author's extensive experience in the field, it focuses on the very specific features of QoL data: its longitudinal nature, multidimensionality, and the problem of missing data. The author uses three real clinical trials throughout her discussions to illustrate practical implementation of the strategies and analytic methods presented. As Quality of Life becomes an increasingly important aspect of clinical trials, it becomes essential for clinicians, statisticians, and designers of these studies to understand and meet the challenges this kind of data present. In this book, SAS and S-PLUS programs, checklists, numerous figures, and a clear, concise presentation combine to provide readers with the tools and skills they need to successfully design, conduct, analyze, and report their own studies.

The Prevention and Treatment of Missing Data in Clinical Trials

Author: National Research Council

Publisher: National Academies Press

Published: 2010-12-21

Total Pages: 163

ISBN-13: 030918651X

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Randomized clinical trials are the primary tool for evaluating new medical interventions. Randomization provides for a fair comparison between treatment and control groups, balancing out, on average, distributions of known and unknown factors among the participants. Unfortunately, these studies often lack a substantial percentage of data. This missing data reduces the benefit provided by the randomization and introduces potential biases in the comparison of the treatment groups. Missing data can arise for a variety of reasons, including the inability or unwillingness of participants to meet appointments for evaluation. And in some studies, some or all of data collection ceases when participants discontinue study treatment. Existing guidelines for the design and conduct of clinical trials, and the analysis of the resulting data, provide only limited advice on how to handle missing data. Thus, approaches to the analysis of data with an appreciable amount of missing values tend to be ad hoc and variable. The Prevention and Treatment of Missing Data in Clinical Trials concludes that a more principled approach to design and analysis in the presence of missing data is both needed and possible. Such an approach needs to focus on two critical elements: (1) careful design and conduct to limit the amount and impact of missing data and (2) analysis that makes full use of information on all randomized participants and is based on careful attention to the assumptions about the nature of the missing data underlying estimates of treatment effects. In addition to the highest priority recommendations, the book offers more detailed recommendations on the conduct of clinical trials and techniques for analysis of trial data.

Design and Analysis of Clinical Trials

Author: Shein-Chung Chow

Publisher: Wiley-Interscience

Published: 1998-06-23

Total Pages: 680

ISBN-13:

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A unique, unifying treatment for statistics and science in clinical trials What sets this volume apart from the many books dealing with clinical trials is its integration of statistical and clinical disciplines. Stressing communication between biostatisticians and clinical scientists, this work clearly relates statistical interpretation to clinical issues arising in different stages of pharmaceutical research and development. Plus, the principles presented here are universal enough to be easily adapted in non-biopharmaceutical settings. Design and Analysis of Clinical Trials tackles concepts and methodologies. It not only covers statistical basics such as uncertainty and bias, design considerations such as patient selection, randomization, and the different types of clinical trials but also deals with various methods of data analysis, group sequential procedures for interim analysis, efficacy data evaluation, analysis of safety data, and more. Throughout, the book: * Surveys current and emerging clinical issues and newly developed statistical methods * Presents a critical review of statistical methodologies in various therapeutic areas * Features case studies from actual clinical trials * Minimizes the mathematics involved, making the material widely accessible * Offers each chapter as a self-contained entity * Includes illustrations to highlight the text This monumental reference on all facets of clinical trials is important reading for physicians, clinical and medical researchers, pharmaceutical scientists, clinical programmers, biostatisticians, and anyone involved in this burgeoning area of clinical research. It can also be used as a textbook in graduate-level courses in the field.

Testing Treatments

Author: Imogen Evans

Publisher: Pinter & Martin Publishers

Published: 2011

Total Pages: 187

ISBN-13: 1905177488

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This work provides a thought-provoking account of how medical treatments can be tested with unbiased or 'fair' trials and explains how patients can work with doctors to achieve this vital goal. It spans the gamut of therapy from mastectomy to thalidomide and explores a vast range of case studies.

Clinical Trials

Author: Tom Brody

Publisher: Academic Press

Published: 2016-02-19

Total Pages: 897

ISBN-13: 0128042583

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Clinical Trials, Second Edition, offers those engaged in clinical trial design a valuable and practical guide. This book takes an integrated approach to incorporate biomedical science, laboratory data of human study, endpoint specification, legal and regulatory aspects and much more with the fundamentals of clinical trial design. It provides an overview of the design options along with the specific details of trial design and offers guidance on how to make appropriate choices. Full of numerous examples and now containing actual decisions from FDA reviewers to better inform trial design, the 2nd edition of Clinical Trials is a must-have resource for early and mid-career researchers and clinicians who design and conduct clinical trials. Contains new and fully revised material on key topics such as biostatistics, biomarkers, orphan drugs, biosimilars, drug regulations in Europe, drug safety, regulatory approval and more Extensively covers the "study schema" and related features of study design Incorporates laboratory data from studies on human patients to provide a concrete tool for understanding the concepts in the design and conduct of clinical trials Includes decisions made by FDA reviewers when granting approval of a drug as real world learning examples for readers

Handbook for Clinical Research

Author: Flora Hammond, MD

Publisher: Demos Medical Publishing

Published: 2014-08-26

Total Pages: 348

ISBN-13: 1936287544

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The majority of physicians are poorly knowledgeable about statistics and research design, yet are expected to do clinical research and write articles (if in academia) or, at the very least, to read the literature critically and provide evidence-based care to patients. The basic skills involved are touched on very minimally in residency, but not in enough depth for an untrained investigator to successfully design or conduct a study, or analyze research findings in any meaningful way. This volume is intended as a “quick fix”, allowing readers to look up information rapidly about various design types and statistical methods to see what the pros, cons, and indications for each are. Research implementation, including regulatory issues and grant writing, is also covered. The book is unique in physical medicine and rehabilitation, and with the increased emphasis on outcomes measurement and push toward a national agenda for disability research, will appeal both to investigators planning and executing studies and clinicians looking to better understand how the findings impact their practice. A list of topics with an outline of headings for each of the sections is attached.

Designs for Clinical Trials

Author: David Harrington

Publisher: Springer Science & Business Media

Published: 2011-10-09

Total Pages: 213

ISBN-13: 1461401402

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This book will examine current issues and controversies in the design of clinical trials, including topics in adaptive and sequential designs, the design of correlative genomic studies, the design of studies in which missing data is anticipated. Each chapter will be written by an expert conducting research in the topic of that chapter. As a collection, the chapters would be intended to serve as a guidance for statisticians designing trials.