Analysis Of Data From Randomized Controlled Trials

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Analysis of Data from Randomized Controlled Trials

Jos W.R. Twisk
Analysis of Data from Randomized Controlled Trials Book PDF

Author : Jos W.R. Twisk
Publisher : Springer Nature
Page : 167 pages
File Size : 48,9 Mb
Release : 2021-10-15
Category : Mathematics
ISBN : 9783030818654

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Analysis of Data from Randomized Controlled Trials by Jos W.R. Twisk Pdf

This book provides a practical guide to the analysis of data from randomized controlled trials (RCT). It gives an answer to the question of how to estimate the intervention effect in an appropriate way. This problem is examined for different RCT designs, such as RCTs with one follow-up measurement, RCTs with more than one follow-up measurement, cluster RCTs, cross-over trials, stepped wedge trials, and N-of-1 trials. The statistical methods are explained in a non-mathematical way and are illustrated by extensive examples. All datasets used in the book are available for download, so readers can reanalyse the examples to gain a better understanding of the methods used. Although most examples are taken from epidemiological and clinical studies, this book is also highly recommended for researchers working in other fields.

The Prevention and Treatment of Missing Data in Clinical Trials

National Research Council,Division of Behavioral and Social Sciences and Education,Committee on National Statistics,Panel on Handling Missing Data in Clinical Trials
The Prevention and Treatment of Missing Data in Clinical Trials Book PDF

Author : National Research Council,Division of Behavioral and Social Sciences and Education,Committee on National Statistics,Panel on Handling Missing Data in Clinical Trials
Publisher : National Academies Press
Page : 163 pages
File Size : 54,9 Mb
Release : 2010-12-21
Category : Medical
ISBN : 9780309186513

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The Prevention and Treatment of Missing Data in Clinical Trials by National Research Council,Division of Behavioral and Social Sciences and Education,Committee on National Statistics,Panel on Handling Missing Data in Clinical Trials Pdf

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.

Small Clinical Trials

Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Small-Number-Participant Clinical Research Trials
Small Clinical Trials Book PDF

Author : Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Small-Number-Participant Clinical Research Trials
Publisher : National Academies Press
Page : 222 pages
File Size : 47,5 Mb
Release : 2001-01-01
Category : Medical
ISBN : 0309171148

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Small Clinical Trials by Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Small-Number-Participant Clinical Research Trials Pdf

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.

Introduction to Randomized Controlled Clinical Trials

John N.S. Matthews
Introduction to Randomized Controlled Clinical Trials Book PDF

Author : John N.S. Matthews
Publisher : CRC Press
Page : 302 pages
File Size : 54,5 Mb
Release : 2006-06-26
Category : Mathematics
ISBN : 9781420011302

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Introduction to Randomized Controlled Clinical Trials by John N.S. Matthews Pdf

Evidence from randomized controlled clinical trials is widely accepted as the only sound basis for assessing the efficacy of new medical treatments. Statistical methods play a key role in all stages of these trials, including their justification, design, and analysis. This second edition of Introduction to Randomized Controlled Clinical Trials prov

Sharing Clinical Trial Data

Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Responsible Sharing of Clinical Trial Data
Sharing Clinical Trial Data Book PDF

Author : Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Responsible Sharing of Clinical Trial Data
Publisher : National Academies Press
Page : 304 pages
File Size : 52,9 Mb
Release : 2015-04-20
Category : Medical
ISBN : 9780309316323

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Sharing Clinical Trial Data by Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Responsible Sharing of Clinical Trial Data Pdf

Data sharing can accelerate new discoveries by avoiding duplicative trials, stimulating new ideas for research, and enabling the maximal scientific knowledge and benefits to be gained from the efforts of clinical trial participants and investigators. At the same time, sharing clinical trial data presents risks, burdens, and challenges. These include the need to protect the privacy and honor the consent of clinical trial participants; safeguard the legitimate economic interests of sponsors; and guard against invalid secondary analyses, which could undermine trust in clinical trials or otherwise harm public health. Sharing Clinical Trial Data presents activities and strategies for the responsible sharing of clinical trial data. With the goal of increasing scientific knowledge to lead to better therapies for patients, this book identifies guiding principles and makes recommendations to maximize the benefits and minimize risks. This report offers guidance on the types of clinical trial data available at different points in the process, the points in the process at which each type of data should be shared, methods for sharing data, what groups should have access to data, and future knowledge and infrastructure needs. Responsible sharing of clinical trial data will allow other investigators to replicate published findings and carry out additional analyses, strengthen the evidence base for regulatory and clinical decisions, and increase the scientific knowledge gained from investments by the funders of clinical trials. The recommendations of Sharing Clinical Trial Data will be useful both now and well into the future as improved sharing of data leads to a stronger evidence base for treatment. This book will be of interest to stakeholders across the spectrum of research--from funders, to researchers, to journals, to physicians, and ultimately, to patients.

Binary Data Analysis of Randomized Clinical Trials with Noncompliance

Kung-Jong Lui
Binary Data Analysis of Randomized Clinical Trials with Noncompliance Book PDF

Author : Kung-Jong Lui
Publisher : John Wiley & Sons
Page : 217 pages
File Size : 50,7 Mb
Release : 2011-03-31
Category : Medical
ISBN : 9781119993902

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Binary Data Analysis of Randomized Clinical Trials with Noncompliance by Kung-Jong Lui Pdf

It is quite common in a randomized clinical trial (RCT) to encounter patients who do not comply with their assigned treatment. Since noncompliance often occurs non-randomly, the commonly-used approaches, including both the as-treated (AT) and as-protocol (AP) analysis, and the intent-to-treat (ITT) (or as-randomized) analysis, are all well known to possibly produce a biased inference of the treatment efficacy. This book provides a systematic and organized approach to analyzing data for RCTs with noncompliance under the most frequently-encountered situations. These include parallel sampling, stratified sampling, cluster sampling, parallel sampling with subsequent missing outcomes, and a series of dependent Bernoulli sampling for repeated measurements. The author provides a comprehensive approach by using contingency tables to illustrate the latent probability structure of observed data. Using real-life examples, computer-simulated data and exercises in each chapter, the book illustrates the underlying theory in an accessible, and easy to understand way. Key features: Consort-flow diagrams and numerical examples are used to illustrate the bias of commonly used approaches, such as, AT analysis, AP analysis and ITT analysis for a RCT with noncompliance. Real-life examples are used throughout the book to explain the practical usefulness of test procedures and estimators. Each chapter is self-contained, allowing the book to be used as a reference source. Includes SAS programs which can be easily modified in calculating the required sample size. Biostatisticians, clinicians, researchers and data analysts working in pharmaceutical industries will benefit from this book. This text can also be used as supplemental material for a course focusing on clinical statistics or experimental trials in epidemiology, psychology and sociology.

Validity and Inter-Rater Reliability Testing of Quality Assessment Instruments

U. S. Department of Health and Human Services,Agency for Healthcare Research and Quality
Validity and Inter Rater Reliability Testing of Quality Assessment Instruments Book PDF

Author : U. S. Department of Health and Human Services,Agency for Healthcare Research and Quality
Publisher : CreateSpace
Page : 108 pages
File Size : 44,5 Mb
Release : 2013-04-09
Category : Medical
ISBN : 1484077148

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Validity and Inter-Rater Reliability Testing of Quality Assessment Instruments by U. S. Department of Health and Human Services,Agency for Healthcare Research and Quality Pdf

The internal validity of a study reflects the extent to which the design and conduct of the study have prevented bias(es). One of the key steps in a systematic review is assessment of a study's internal validity, or potential for bias. This assessment serves to: (1) identify the strengths and limitations of the included studies; (2) investigate, and potentially explain heterogeneity in findings across different studies included in a systematic review; and (3) grade the strength of evidence for a given question. The risk of bias assessment directly informs one of four key domains considered when assessing the strength of evidence. With the increase in the number of published systematic reviews and development of systematic review methodology over the past 15 years, close attention has been paid to the methods for assessing internal validity. Until recently this has been referred to as “quality assessment” or “assessment of methodological quality.” In this context “quality” refers to “the confidence that the trial design, conduct, and analysis has minimized or avoided biases in its treatment comparisons.” To facilitate the assessment of methodological quality, a plethora of tools has emerged. Some of these tools were developed for specific study designs (e.g., randomized controlled trials (RCTs), cohort studies, case-control studies), while others were intended to be applied to a range of designs. The tools often incorporate characteristics that may be associated with bias; however, many tools also contain elements related to reporting (e.g., was the study population described) and design (e.g., was a sample size calculation performed) that are not related to bias. The Cochrane Collaboration recently developed a tool to assess the potential risk of bias in RCTs. The Risk of Bias (ROB) tool was developed to address some of the shortcomings of existing quality assessment instruments, including over-reliance on reporting rather than methods. Several systematic reviews have catalogued and critiqued the numerous tools available to assess methodological quality, or risk of bias of primary studies. In summary, few existing tools have undergone extensive inter-rater reliability or validity testing. Moreover, the focus of much of the tool development or testing that has been done has been on criterion or face validity. Therefore it is unknown whether, or to what extent, the summary assessments based on these tools differentiate between studies with biased and unbiased results (i.e., studies that may over- or underestimate treatment effects). There is a clear need for inter-rater reliability testing of different tools in order to enhance consistency in their application and interpretation across different systematic reviews. Further, validity testing is essential to ensure that the tools being used can identify studies with biased results. Finally, there is a need to determine inter-rater reliability and validity in order to support the uptake and use of individual tools that are recommended by the systematic review community, and specifically the ROB tool within the Evidence-based Practice Center (EPC) Program. In this project we focused on two tools that are commonly used in systematic reviews. The Cochrane ROB tool was designed for RCTs and is the instrument recommended by The Cochrane Collaboration for use in systematic reviews of RCTs. The Newcastle-Ottawa Scale is commonly used for nonrandomized studies, specifically cohort and case-control studies.

Clinical Trial Data Analysis Using R

Ding-Geng (Din) Chen,Karl E. Peace
Clinical Trial Data Analysis Using R Book PDF

Author : Ding-Geng (Din) Chen,Karl E. Peace
Publisher : CRC Press
Page : 384 pages
File Size : 52,5 Mb
Release : 2010-12-14
Category : Mathematics
ISBN : 9781439840214

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Clinical Trial Data Analysis Using R by Ding-Geng (Din) Chen,Karl E. Peace Pdf

Too often in biostatistical research and clinical trials, a knowledge gap exists between developed statistical methods and the applications of these methods. Filling this gap, Clinical Trial Data Analysis Using R provides a thorough presentation of biostatistical analyses of clinical trial data and shows step by step how to implement the statistical methods using R. The book’s practical, detailed approach draws on the authors’ 30 years of real-world experience in biostatistical research and clinical development. Each chapter presents examples of clinical trials based on the authors’ actual experiences in clinical drug development. Various biostatistical methods for analyzing the data are then identified. The authors develop analysis code step by step using appropriate R packages and functions. This approach enables readers to gain an understanding of the analysis methods and R implementation so that they can use R to analyze their own clinical trial data. With step-by-step illustrations of R implementations, this book shows how to easily use R to simulate and analyze data from a clinical trial. It describes numerous up-to-date statistical methods and offers sound guidance on the processes involved in clinical trials.

Statistical Monitoring of Clinical Trials

Michael A. Proschan,K. K. Gordon Lan,Janet Turk Wittes
Statistical Monitoring of Clinical Trials Book PDF

Author : Michael A. Proschan,K. K. Gordon Lan,Janet Turk Wittes
Publisher : Springer Science & Business Media
Page : 268 pages
File Size : 42,8 Mb
Release : 2006-12-31
Category : Medical
ISBN : 9780387449708

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Statistical Monitoring of Clinical Trials by Michael A. Proschan,K. K. Gordon Lan,Janet Turk Wittes Pdf

The approach taken in this book is, to studies monitored over time, what the Central Limit Theorem is to studies with only one analysis. Just as the Central Limit Theorem shows that test statistics involving very different types of clinical trial outcomes are asymptotically normal, this book shows that the joint distribution of the test statistics at different analysis times is asymptotically multivariate normal with the correlation structure of Brownian motion ("the B-value") – irrespective of the test statistic. Thus, this book offers statisticians an accessible, incremental approach to understanding Brownian motion as related to clinical trials.

Handbook of Statistics in Clinical Oncology

John Crowley,Antje Hoering,Donna Ankerst
Handbook of Statistics in Clinical Oncology Book PDF

Author : John Crowley,Antje Hoering,Donna Ankerst
Publisher : CRC Press
Page : 642 pages
File Size : 45,7 Mb
Release : 2005-12-01
Category : Mathematics
ISBN : 9781420027761

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Handbook of Statistics in Clinical Oncology by John Crowley,Antje Hoering,Donna Ankerst Pdf

A compendium of cutting-edge statistical approaches to solving problems in clinical oncology, Handbook of Statistics in Clinical Oncology, Second Edition focuses on clinical trials in phases I, II, and III, proteomic and genomic studies, complementary outcomes and exploratory methods. Cancer Forum called the first edition a

Applied Longitudinal Data Analysis for Epidemiology

Jos W. R. Twisk
Applied Longitudinal Data Analysis for Epidemiology Book PDF

Author : Jos W. R. Twisk
Publisher : Cambridge University Press
Page : 337 pages
File Size : 53,9 Mb
Release : 2013-05-09
Category : Medical
ISBN : 9781107030039

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Applied Longitudinal Data Analysis for Epidemiology by Jos W. R. Twisk Pdf

A practical guide to the most important techniques available for longitudinal data analysis, essential for non-statisticians and researchers.

Meta-Analysis of Controlled Clinical Trials

Anne Whitehead
Meta Analysis of Controlled Clinical Trials Book PDF

Author : Anne Whitehead
Publisher : John Wiley & Sons
Page : 352 pages
File Size : 51,6 Mb
Release : 2003-01-17
Category : Mathematics
ISBN : 9780470854198

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Meta-Analysis of Controlled Clinical Trials by Anne Whitehead Pdf

Over the last twenty years there has been a dramatic upsurge in the application of meta-analysis to medical research. This has mainly been due to greater emphasis on evidence-based medicine and the need for reliable summaries of the vast and expanding volume of clinical research. At the same time there have been great strides in the development and refinement of the associated statistical methodology. This book describes the planning, conduct and reporting of a meta-analysis as applied to a series of randomized controlled clinical trials. * The various approaches are presented within a general unified framework. * Meta-analysis techniques are described in detail, from their theoretical development through to practical implementation. * Each topic discussed is supported by detailed worked examples. * A comparison of fixed and random effects approaches is included, as well as a discussion of Bayesian methods and cumulative meta-analysis. * Fully documented programs using standard statistical procedures in SAS are available on the Web. Ideally suited for practising statisticians and statistically-minded medical professionals, the book will also be of use to graduate students of medical statistics. The book is a self-contained and comprehensive account of the subject and an essential purchase for anyone involved in clinical trials.

Field Trials of Health Interventions

Peter G. Smith,R. H. Morrow,Richard H. Morrow,David A. Ross
Field Trials of Health Interventions Book PDF

Author : Peter G. Smith,R. H. Morrow,Richard H. Morrow,David A. Ross
Publisher : Oxford University Press, USA
Page : 479 pages
File Size : 54,9 Mb
Release : 2015
Category : Medical
ISBN : 9780198732860

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Field Trials of Health Interventions by Peter G. Smith,R. H. Morrow,Richard H. Morrow,David A. Ross Pdf

"IEA, International Epidemiological Association, Welcome Trust."

Randomised Clinical Trials

David Machin,Peter M. Fayers,Bee Choo Tai
Randomised Clinical Trials Book PDF

Author : David Machin,Peter M. Fayers,Bee Choo Tai
Publisher : John Wiley & Sons
Page : 86 pages
File Size : 41,6 Mb
Release : 2021-07-26
Category : Medical
ISBN : 9781119524649

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Randomised Clinical Trials by David Machin,Peter M. Fayers,Bee Choo Tai Pdf

Randomised Clinical Trials: Design, Practice and Reporting provides a detailed overview of the methodology for conducting clinical trials, including developing protocols, data capture, randomisation, analysis and reporting. Assuming no prior background, this user-friendly resource describes the statistical, regulatory, and practical components required for conducting randomised clinical trials. Numerous examples and case studies from industry, academia, and the research literature help readers understand each stage of the clinical trial process. This second edition contains extensively revised material throughout, including new chapters covering designs for repeated measures, non-inferiority, cluster and stepped wedge trials. Other new chapters describe data and safety monitoring, biomarker studies, and feasibility studies. Updated and expanded sections discuss situations where multiple organs, different body locations or competing risks are involved, subgroup analysis, and multiple outcomes. Written by an author team with extensive experience in conducting clinical trials, this book: Provides comprehensive coverage of randomised clinical trials, ranging from basic to advanced Features several new chapters, updated case studies and examples, and references to changes in regulations Explains basic randomised trials, including the parallel two-group controlled trial with a single outcome measure Covers paired trial designs and trials with more than two interventions Includes a chapter on miscellaneous topics such as adaptive designs, large simple trials, Bayesian methods for very small trials, alpha-spending functions and the predictive probability test Randomised Clinical Trials is essential reading for clinicians, nurses, data managers, and medical statisticians involved in clinical trials, and for health practitioners responsible for direct patient care in a clinical trial setting.

Handbook of Statistical Methods for Randomized Controlled Trials

KyungMann Kim,Frank Bretz,Ying Kuen K. Cheung,Lisa V. Hampson
Handbook of Statistical Methods for Randomized Controlled Trials Book PDF

Author : KyungMann Kim,Frank Bretz,Ying Kuen K. Cheung,Lisa V. Hampson
Publisher : CRC Press
Page : 655 pages
File Size : 42,5 Mb
Release : 2021-08-23
Category : Mathematics
ISBN : 9781498714648

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Handbook of Statistical Methods for Randomized Controlled Trials by KyungMann Kim,Frank Bretz,Ying Kuen K. Cheung,Lisa V. Hampson Pdf

Statistical concepts provide scientific framework in experimental studies, including randomized controlled trials. In order to design, monitor, analyze and draw conclusions scientifically from such clinical trials, clinical investigators and statisticians should have a firm grasp of the requisite statistical concepts. The Handbook of Statistical Methods for Randomized Controlled Trials presents these statistical concepts in a logical sequence from beginning to end and can be used as a textbook in a course or as a reference on statistical methods for randomized controlled trials. Part I provides a brief historical background on modern randomized controlled trials and introduces statistical concepts central to planning, monitoring and analysis of randomized controlled trials. Part II describes statistical methods for analysis of different types of outcomes and the associated statistical distributions used in testing the statistical hypotheses regarding the clinical questions. Part III describes some of the most used experimental designs for randomized controlled trials including the sample size estimation necessary in planning. Part IV describe statistical methods used in interim analysis for monitoring of efficacy and safety data. Part V describe important issues in statistical analyses such as multiple testing, subgroup analysis, competing risks and joint models for longitudinal markers and clinical outcomes. Part VI addresses selected miscellaneous topics in design and analysis including multiple assignment randomization trials, analysis of safety outcomes, non-inferiority trials, incorporating historical data, and validation of surrogate outcomes.