› › Success Probability Estimation With Applications To Clinical Trials

Success Probability Estimation with Applications to Clinical Trials

Author : Daniele De Martini

Publisher : John Wiley & Sons

Page : 187 pages

File Size : 41,23 MB

Release : 2013-05-06

Category : Medical

ISBN : 1118548310

Get eBook

Book Description

Provides an introduction to the various statistical techniques involved in medical research and drug development with a focus on estimating the success probability of an experiment Success Probability Estimation with Applications to Clinical Trials details the use of success probability estimation in both the planning and analyzing of clinical trials and in widely used statistical tests. Devoted to both statisticians and non-statisticians who are involved in clinical trials, Part I of the book presents new concepts related to success probability estimation and their usefulness in clinical trials, and each section begins with a non-technical explanation of the presented concepts. Part II delves deeper into the techniques for success probability estimation and features applications to both reproducibility probability estimation and conservative sample size estimation. Success Probability Estimation with Applications to Clinical Trials: • Addresses the theoretical and practical aspects of the topic and introduces new and promising techniques in the statistical and pharmaceutical industries Features practical solutions for problems that are often encountered in clinical trials Includes success probability estimation for widely used statistical tests, such as parametric and nonparametric models Focuses on experimental planning, specifically the sample size of clinical trials using phase II results and data for planning phase III trials Introduces statistical concepts related to success probability estimation and their usefulness in clinical trials Success Probability Estimation with Applications to Clinical Trials is an ideal reference for statisticians and biostatisticians in the pharmaceutical industry as well as researchers and practitioners in medical centers who are actively involved in health policy, clinical research, and the design and evaluation of clinical trials.



Small Clinical Trials

Author : Institute of Medicine

Publisher : National Academies Press

Page : 221 pages

File Size : 43,28 MB

Release : 2001-01-01

Category : Medical

ISBN : 0309171148

Get eBook

Book Description

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.



Group Sequential and Confirmatory Adaptive Designs in Clinical Trials

Author : Gernot Wassmer

Publisher : Springer

Page : 310 pages

File Size : 24,55 MB

Release : 2016-07-04

Category : Medical

ISBN : 3319325620

Get eBook

Book Description

This book provides an up-to-date review of the general principles of and techniques for confirmatory adaptive designs. Confirmatory adaptive designs are a generalization of group sequential designs. With these designs, interim analyses are performed in order to stop the trial prematurely under control of the Type I error rate. In adaptive designs, it is also permissible to perform a data-driven change of relevant aspects of the study design at interim stages. This includes, for example, a sample-size reassessment, a treatment-arm selection or a selection of a pre-specified sub-population. Essentially, this adaptive methodology was introduced in the 1990s. Since then, it has become popular and the object of intense discussion and still represents a rapidly growing field of statistical research. This book describes adaptive design methodology at an elementary level, while also considering designing and planning issues as well as methods for analyzing an adaptively planned trial. This includes estimation methods and methods for the determination of an overall p-value. Part I of the book provides the group sequential methods that are necessary for understanding and applying the adaptive design methodology supplied in Parts II and III of the book. The book contains many examples that illustrate use of the methods for practical application. The book is primarily written for applied statisticians from academia and industry who are interested in confirmatory adaptive designs. It is assumed that readers are familiar with the basic principles of descriptive statistics, parameter estimation and statistical testing. This book will also be suitable for an advanced statistical course for applied statisticians or clinicians with a sound statistical background.



Randomization in Clinical Trials

Author : William F. Rosenberger

Publisher : John Wiley & Sons

Page : 286 pages

File Size : 33,43 MB

Release : 2004-03-24

Category : Mathematics

ISBN : 0471654078

Get eBook

Book Description

A unique overview that melds the concepts of conditionalprobability and stochastic processes into real-lifeapplications The role of randomization techniques in clinical trials has becomeincreasingly important. This comprehensive guide combines both theapplied aspects of randomization in clinical trials with aprobabilistic treatment of properties of randomization. Taking anunabashedly non-Bayesian and nonparametric approach to inference,the book focuses on the linear rank test under a randomizationmodel, with added discussion on likelihood-based inference as itrelates to sufficiency and ancillarity. Developments in stochasticprocesses and applied probability are also given where appropriate.Intuition is stressed over mathematics, but not without a cleardevelopment of the latter in the context of the former. Providing a consolidated review of the field, the book includesrelevant and practical discussions of: * The benefits of randomization in terms of reduction of bias * Randomization as a basis for inference * Covariate-adaptive and response-adaptive randomization * Current philosophies, controversies, and new developments With ample problem sets, theoretical exercises, and short computersimulations using SAS, Randomization in Clinical Trials: Theory andPractice is equally useful as a standard textbook in biostatisticsgraduate programs as well as a reliable reference forbiostatisticians in practice.



Encyclopedia of Biopharmaceutical Statistics - Four Volume Set

Author : Shein-Chung Chow

Publisher : CRC Press

Page : 4031 pages

File Size : 35,33 MB

Release : 2018-09-03

Category : Medical

ISBN : 135111025X

Get eBook

Book Description

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.



New Developments in Statistical Modeling, Inference and Application

Author : Zhezhen Jin

Publisher : Springer

Page : 218 pages

File Size : 19,35 MB

Release : 2016-10-28

Category : Medical

ISBN : 3319425714

Get eBook

Book Description

The papers in this volume represent the most timely and advanced contributions to the 2014 Joint Applied Statistics Symposium of the International Chinese Statistical Association (ICSA) and the Korean International Statistical Society (KISS), held in Portland, Oregon. The contributions cover new developments in statistical modeling and clinical research: including model development, model checking, and innovative clinical trial design and analysis. Each paper was peer-reviewed by at least two referees and also by an editor. The conference was attended by over 400 participants from academia, industry, and government agencies around the world, including from North America, Asia, and Europe. It offered 3 keynote speeches, 7 short courses, 76 parallel scientific sessions, student paper sessions, and social events.



The Theory of Response-Adaptive Randomization in Clinical Trials

Author : Feifang Hu

Publisher : John Wiley & Sons

Page : 234 pages

File Size : 36,39 MB

Release : 2006-09-29

Category : Mathematics

ISBN : 0470055871

Get eBook

Book Description

Presents a firm mathematical basis for the use of response-adaptive randomization procedures in practice The Theory of Response-Adaptive Randomization in Clinical Trials is the result of the authors' ten-year collaboration as well as their collaborations with other researchers in investigating the important questions regarding response-adaptive randomization in a rigorous mathematical framework. Response-adaptive allocation has a long history in biostatistics literature; however, largely due to the disastrous ECMO trial in the early 1980s, there is a general reluctance to use these procedures. This timely book represents a mathematically rigorous subdiscipline of experimental design involving randomization and answers fundamental questions, including: How does response-adaptive randomization affect power? Can standard inferential tests be applied following response-adaptive randomization? What is the effect of delayed response? Which procedure is most appropriate and how can "most appropriate" be quantified? How can heterogeneity of the patient population be incorporated? Can response-adaptive randomization be performed with more than two treatments or with continuous responses? The answers to these questions communicate a thorough understanding of the asymptotic properties of each procedure discussed, including asymptotic normality, consistency, and asymptotic variance of the induced allocation. Topical coverage includes: The relationship between power and response-adaptive randomization The general result for determining asymptotically best procedures Procedures based on urn models Procedures based on sequential estimation Implications for the practice of clinical trials Useful for graduate students in mathematics, statistics, and biostatistics as well as researchers and industrial and academic biostatisticians, this book offers a rigorous treatment of the subject in order to find the optimal procedure to use in practice.



Practical Applications of Bayesian Reliability

Author : Yan Liu

Publisher : John Wiley & Sons

Page : 322 pages

File Size : 36,49 MB

Release : 2019-03-21

Category : Technology & Engineering

ISBN : 1119288002

Get eBook

Book Description

Demonstrates how to solve reliability problems using practical applications of Bayesian models This self-contained reference provides fundamental knowledge of Bayesian reliability and utilizes numerous examples to show how Bayesian models can solve real life reliability problems. It teaches engineers and scientists exactly what Bayesian analysis is, what its benefits are, and how they can apply the methods to solve their own problems. To help readers get started quickly, the book presents many Bayesian models that use JAGS and which require fewer than 10 lines of command. It also offers a number of short R scripts consisting of simple functions to help them become familiar with R coding. Practical Applications of Bayesian Reliability starts by introducing basic concepts of reliability engineering, including random variables, discrete and continuous probability distributions, hazard function, and censored data. Basic concepts of Bayesian statistics, models, reasons, and theory are presented in the following chapter. Coverage of Bayesian computation, Metropolis-Hastings algorithm, and Gibbs Sampling comes next. The book then goes on to teach the concepts of design capability and design for reliability; introduce Bayesian models for estimating system reliability; discuss Bayesian Hierarchical Models and their applications; present linear and logistic regression models in Bayesian Perspective; and more. Provides a step-by-step approach for developing advanced reliability models to solve complex problems, and does not require in-depth understanding of statistical methodology Educates managers on the potential of Bayesian reliability models and associated impact Introduces commonly used predictive reliability models and advanced Bayesian models based on real life applications Includes practical guidelines to construct Bayesian reliability models along with computer codes for all of the case studies JAGS and R codes are provided on an accompanying website to enable practitioners to easily copy them and tailor them to their own applications Practical Applications of Bayesian Reliability is a helpful book for industry practitioners such as reliability engineers, mechanical engineers, electrical engineers, product engineers, system engineers, and materials scientists whose work includes predicting design or product performance.



Methods and Applications of Statistics in Clinical Trials, Volume 1

Author : Narayanaswamy Balakrishnan

Publisher : John Wiley & Sons

Page : 937 pages

File Size : 47,41 MB

Release : 2014-03-05

Category : Medical

ISBN : 1118595912

Get eBook

Book Description

A complete guide to the key statistical concepts essential for the design and construction of clinical trials As the newest major resource in the field of medical research, Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs presents a timely and authoritative reviewof the central statistical concepts used to build clinical trials that obtain the best results. The referenceunveils modern approaches vital to understanding, creating, and evaluating data obtained throughoutthe various stages of clinical trial design and analysis. Accessible and comprehensive, the first volume in a two-part set includes newly-written articles as well as established literature from the Wiley Encyclopedia of Clinical Trials. Illustrating a variety of statistical concepts and principles such as longitudinal data, missing data, covariates, biased-coin randomization, repeated measurements, and simple randomization, the book also provides in-depth coverage of the various trial designs found within phase I-IV trials. Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs also features: Detailed chapters on the type of trial designs, such as adaptive, crossover, group-randomized, multicenter, non-inferiority, non-randomized, open-labeled, preference, prevention, and superiority trials Over 100 contributions from leading academics, researchers, and practitioners An exploration of ongoing, cutting-edge clinical trials on early cancer and heart disease, mother-to-child human immunodeficiency virus transmission trials, and the AIDS Clinical Trials Group Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs is an excellent reference for researchers, practitioners, and students in the fields of clinicaltrials, pharmaceutics, biostatistics, medical research design, biology, biomedicine, epidemiology,and public health.



Encyclopedia of Health Economics

Author :

Publisher : Newnes

Page : 1663 pages

File Size : 45,53 MB

Release : 2014-02-21

Category : Medical

ISBN : 0123756790

Get eBook

Book Description

The Encyclopedia of Health Economics offers students, researchers and policymakers objective and detailed empirical analysis and clear reviews of current theories and polices. It helps practitioners such as health care managers and planners by providing accessible overviews into the broad field of health economics, including the economics of designing health service finance and delivery and the economics of public and population health. This encyclopedia provides an organized overview of this diverse field, providing one trusted source for up-to-date research and analysis of this highly charged and fast-moving subject area. Features research-driven articles that are objective, better-crafted, and more detailed than is currently available in journals and handbooks Combines insights and scholarship across the breadth of health economics, where theory and empirical work increasingly come from non-economists Provides overviews of key policies, theories and programs in easy-to-understand language