Handbook Of Methods For Designing Monitoring And Analyzing Dose Finding Trials
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Handbook Of Methods For Designing Monitoring And Analyzing Dose Finding Trials
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Author : John O'Quigley
language : en
Publisher: CRC Press
Release Date : 2017-04-27
Handbook Of Methods For Designing Monitoring And Analyzing Dose Finding Trials written by John O'Quigley and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017-04-27 with Mathematics categories.
Handbook of Methods for Designing, Monitoring, and Analyzing Dose-Finding Trials gives a thorough presentation of state-of-the-art methods for early phase clinical trials. The methodology of clinical trials has advanced greatly over the last 20 years and, arguably, nowhere greater than that of early phase studies. The need to accelerate drug development in a rapidly evolving context of targeted therapies, immunotherapy, combination treatments and complex group structures has provided the stimulus to these advances. Typically, we deal with very small samples, sequential methods that need to be efficient, while, at the same time adhering to ethical principles due to the involvement of human subjects. Statistical inference is difficult since the standard techniques of maximum likelihood do not usually apply as a result of model misspecification and parameter estimates lying on the boundary of the parameter space. Bayesian methods play an important part in overcoming these difficulties, but nonetheless, require special consideration in this particular context. The purpose of this handbook is to provide an expanded summary of the field as it stands and also, through discussion, provide insights into the thinking of leaders in the field as to the potential developments of the years ahead. With this goal in mind we present: An introduction to the field for graduate students and novices A basis for more established researchers from which to build A collection of material for an advanced course in early phase clinical trials A comprehensive guide to available methodology for practicing statisticians on the design and analysis of dose-finding experiments An extensive guide for the multiple comparison and modeling (MCP-Mod) dose-finding approach, adaptive two-stage designs for dose finding, as well as dose–time–response models and multiple testing in the context of confirmatory dose-finding studies. John O’Quigley is a professor of mathematics and research director at the French National Institute for Health and Medical Research based at the Faculty of Mathematics, University Pierre and Marie Curie in Paris, France. He is author of Proportional Hazards Regression and has published extensively in the field of dose finding. Alexia Iasonos is an associate attending biostatistician at the Memorial Sloan Kettering Cancer Center in New York. She has over one hundred publications in the leading statistical and clinical journals on the methodology and design of early phase clinical trials. Dr. Iasonos has wide experience in the actual implementation of model based early phase trials and has given courses in scientific meetings internationally. Björn Bornkamp is a statistical methodologist at Novartis in Basel, Switzerland, researching and implementing dose-finding designs in Phase II clinical trials. He is one of the co-developers of the MCP-Mod methodology for dose finding and main author of the DoseFinding R package. He has published numerous papers on dose finding, nonlinear models and Bayesian statistics, and in 2013 won the Royal Statistical Society award for statistical excellence in the pharmaceutical industry.
Handbook Of Methods For Designing Monitoring And Analyzing Dose Finding Trials
DOWNLOAD
Author : John O'Quigley
language : en
Publisher: CRC Press
Release Date : 2017-04-27
Handbook Of Methods For Designing Monitoring And Analyzing Dose Finding Trials written by John O'Quigley and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017-04-27 with Mathematics categories.
Handbook of Methods for Designing, Monitoring, and Analyzing Dose-Finding Trials gives a thorough presentation of state-of-the-art methods for early phase clinical trials. The methodology of clinical trials has advanced greatly over the last 20 years and, arguably, nowhere greater than that of early phase studies. The need to accelerate drug development in a rapidly evolving context of targeted therapies, immunotherapy, combination treatments and complex group structures has provided the stimulus to these advances. Typically, we deal with very small samples, sequential methods that need to be efficient, while, at the same time adhering to ethical principles due to the involvement of human subjects. Statistical inference is difficult since the standard techniques of maximum likelihood do not usually apply as a result of model misspecification and parameter estimates lying on the boundary of the parameter space. Bayesian methods play an important part in overcoming these difficulties, but nonetheless, require special consideration in this particular context. The purpose of this handbook is to provide an expanded summary of the field as it stands and also, through discussion, provide insights into the thinking of leaders in the field as to the potential developments of the years ahead. With this goal in mind we present: An introduction to the field for graduate students and novices A basis for more established researchers from which to build A collection of material for an advanced course in early phase clinical trials A comprehensive guide to available methodology for practicing statisticians on the design and analysis of dose-finding experiments An extensive guide for the multiple comparison and modeling (MCP-Mod) dose-finding approach, adaptive two-stage designs for dose finding, as well as dose–time–response models and multiple testing in the context of confirmatory dose-finding studies. John O’Quigley is a professor of mathematics and research director at the French National Institute for Health and Medical Research based at the Faculty of Mathematics, University Pierre and Marie Curie in Paris, France. He is author of Proportional Hazards Regression and has published extensively in the field of dose finding. Alexia Iasonos is an associate attending biostatistician at the Memorial Sloan Kettering Cancer Center in New York. She has over one hundred publications in the leading statistical and clinical journals on the methodology and design of early phase clinical trials. Dr. Iasonos has wide experience in the actual implementation of model based early phase trials and has given courses in scientific meetings internationally. Björn Bornkamp is a statistical methodologist at Novartis in Basel, Switzerland, researching and implementing dose-finding designs in Phase II clinical trials. He is one of the co-developers of the MCP-Mod methodology for dose finding and main author of the DoseFinding R package. He has published numerous papers on dose finding, nonlinear models and Bayesian statistics, and in 2013 won the Royal Statistical Society award for statistical excellence in the pharmaceutical industry.
Handbook Of Methods For Designing And Monitoring Dose Finding Trials
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Author : John O'Quigley
language : en
Publisher: Chapman & Hall/CRC
Release Date : 2017
Handbook Of Methods For Designing And Monitoring Dose Finding Trials written by John O'Quigley and has been published by Chapman & Hall/CRC this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017 with Mathematics categories.
This handbook will provide an overview of most up-to-date statistical methods required for design, monitoring and analysis for dose finding clinical trials, focusing both on the clinical aspects as well as statistical considerations. This handbook will not cover statistical methods for Phase II non-dose finding studies or Phase III clinical trials.
Dose Finding Designs For Early Phase Cancer Clinical Trials
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Author : Takashi Daimon
language : en
Publisher: Springer
Release Date : 2019-05-21
Dose Finding Designs For Early Phase Cancer Clinical Trials written by Takashi Daimon and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019-05-21 with Medical categories.
This book provides a comprehensive introduction to statistical methods for designing early phase dose-finding clinical trials. It will serve as a textbook or handbook for graduate students and practitioners in biostatistics and clinical investigators who are involved in designing, conducting, monitoring, and analyzing dose-finding trials. The book will also provide an overview of advanced topics and discussions in this field for the benefit of researchers in biostatistics and statistical science. Beginning with backgrounds and fundamental notions on dose finding in early phase clinical trials, the book then provides traditional and recent dose-finding designs of phase I trials for, e.g., cytotoxic agents in oncology, to evaluate toxicity outcome. Included are rule-based and model-based designs, such as 3 + 3 designs, accelerated titration designs, toxicity probability interval designs, continual reassessment method and related designs, and escalation overdose control designs. This bookalso covers more complex and updated dose-finding designs of phase I-II and I/II trials for cytotoxic agents, and cytostatic agents, focusing on both toxicity and efficacy outcomes, such as designs with covariates and drug combinations, maximum tolerated dose-schedule finding designs, and so on.
Handbook Of Multiple Comparisons
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Author : Xinping Cui
language : en
Publisher: CRC Press
Release Date : 2021-11-17
Handbook Of Multiple Comparisons written by Xinping Cui and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-11-17 with Mathematics categories.
Written by experts that include originators of some key ideas, chapters in the Handbook of Multiple Testing cover multiple comparison problems big and small, with guidance toward error rate control and insights on how principles developed earlier can be applied to current and emerging problems. Some highlights of the coverages are as follows. Error rate control is useful for controlling the incorrect decision rate. Chapter 1 introduces Tukey's original multiple comparison error rates and point to how they have been applied and adapted to modern multiple comparison problems as discussed in the later chapters. Principles endure. While the closed testing principle is more familiar, Chapter 4 shows the partitioning principle can derive confidence sets for multiple tests, which may become important as the profession goes beyond making decisions based on p-values. Multiple comparisons of treatment efficacy often involve multiple doses and endpoints. Chapter 12 on multiple endpoints explains how different choices of endpoint types lead to different multiplicity adjustment strategies, while Chapter 11 on the MCP-Mod approach is particularly useful for dose-finding. To assess efficacy in clinical trials with multiple doses and multiple endpoints, the reader can see the traditional approach in Chapter 2, the Graphical approach in Chapter 5, and the multivariate approach in Chapter 3. Personalized/precision medicine based on targeted therapies, already a reality, naturally leads to analysis of efficacy in subgroups. Chapter 13 draws attention to subtle logical issues in inferences on subgroups and their mixtures, with a principled solution that resolves these issues. This chapter has implication toward meeting the ICHE9R1 Estimands requirement. Besides the mere multiple testing methodology itself, the handbook also covers related topics like the statistical task of model selection in Chapter 7 or the estimation of the proportion of true null hypotheses (or, in other words, the signal prevalence) in Chapter 8. It also contains decision-theoretic considerations regarding the admissibility of multiple tests in Chapter 6. The issue of selected inference is addressed in Chapter 9. Comparison of responses can involve millions of voxels in medical imaging or SNPs in genome-wide association studies (GWAS). Chapter 14 and Chapter 15 provide state of the art methods for large scale simultaneous inference in these settings.
Dose Finding And Beyond In Biopharmaceutical Development
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Author : Jingjing Ye
language : en
Publisher: Springer Nature
Release Date : 2024-10-28
Dose Finding And Beyond In Biopharmaceutical Development written by Jingjing Ye and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2024-10-28 with Medical categories.
This book covers topics in 2 parts: 1) Review of FDA Guidance, 2) Novel Designs and Analyses. While covering basic principles of dose finding, this book details advancements made in drug development. Finding the right dose(s) is one of the most important objectives in new drug development. In Phase I clinical development, one of the objectives is to escalate test doses from low to high. The low doses should be safe, then escalate up to the maximally tolerable dose (MTD). Phase II clinical trials then lower test doses to the minimal efficacious dose (MinED). Dose range of a study drug can be thought of as the doses between MinED and MTD. From this dose range, one or a few doses are selected for Phase III confirmation. In practice, dose finding is a very difficult in every phase of clinical development for new drugs. The editors brought distinguished researchers and practitioners in biopharmaceuticals and universities, to discuss the statistical procedures, useful methods, and their novel applications in dose finding. The chapters in the book present emerging topics in dose-finding and related interdisciplinary areas. This timely book is a valuable resource to stimulate the development of this growing and exciting field in drug development.
Platform Trial Designs In Drug Development
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Author : Zoran Antonijevic
language : en
Publisher: CRC Press
Release Date : 2018-12-07
Platform Trial Designs In Drug Development written by Zoran Antonijevic and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-12-07 with Mathematics categories.
Platform trials test multiple therapies in one indication, one therapy for multiple indications, or both. These novel clinical trial designs can dramatically increase the cost-effectiveness of drug development, leading to life-altering medicines for people suffering from serious illnesses, possibly at lower cost. Currently, the cost of drug development is unsustainable. Furthermore, there are particular problems in rare diseases and small biomarker defined subsets in oncology, where the required sample sizes for traditional clinical trial designs may not be feasible. The editors recruited the key innovators in this domain. The 20 articles discuss trial designs from perspectives as diverse as quantum computing, patient’s rights to information, and international health. The book begins with an overview of platform trials from multiple perspectives. It then describes impacts of platform trials on the pharmaceutical industry’s key stakeholders: patients, regulators, and payers. Next it provides advanced statistical methods that address multiple aspects of platform trials, before concluding with a pharmaceutical executive’s perspective on platform trials. Except for the statistical methods section, only a basic qualitative knowledge of clinical trials is needed to appreciate the important concepts and novel ideas presented.
Handbook Of Statistical Methods For Case Control Studies
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Author : Ørnulf Borgan
language : en
Publisher: CRC Press
Release Date : 2018-06-27
Handbook Of Statistical Methods For Case Control Studies written by Ørnulf Borgan and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-06-27 with Mathematics categories.
Handbook of Statistical Methods for Case-Control Studies is written by leading researchers in the field. It provides an in-depth treatment of up-to-date and currently developing statistical methods for the design and analysis of case-control studies, as well as a review of classical principles and methods. The handbook is designed to serve as a reference text for biostatisticians and quantitatively-oriented epidemiologists who are working on the design and analysis of case-control studies or on related statistical methods research. Though not specifically intended as a textbook, it may also be used as a backup reference text for graduate level courses. Book Sections Classical designs and causal inference, measurement error, power, and small-sample inference Designs that use full-cohort information Time-to-event data Genetic epidemiology About the Editors Ørnulf Borgan is Professor of Statistics, University of Oslo. His book with Andersen, Gill and Keiding on counting processes in survival analysis is a world classic. Norman E. Breslow was, at the time of his death, Professor Emeritus in Biostatistics, University of Washington. For decades, his book with Nick Day has been the authoritative text on case-control methodology. Nilanjan Chatterjee is Bloomberg Distinguished Professor, Johns Hopkins University. He leads a broad research program in statistical methods for modern large scale biomedical studies. Mitchell H. Gail is a Senior Investigator at the National Cancer Institute. His research includes modeling absolute risk of disease, intervention trials, and statistical methods for epidemiology. Alastair Scott was, at the time of his death, Professor Emeritus of Statistics, University of Auckland. He was a major contributor to using survey sampling methods for analyzing case-control data. Chris J. Wild is Professor of Statistics, University of Auckland. His research includes nonlinear regression and methods for fitting models to response-selective data.
Handbook Of Statistical Methods For Randomized Controlled Trials
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Author : KyungMann Kim
language : en
Publisher: CRC Press
Release Date : 2021-08-23
Handbook Of Statistical Methods For Randomized Controlled Trials written by KyungMann Kim and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-08-23 with Mathematics categories.
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.
Handbook Of Infectious Disease Data Analysis
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Author : Leonhard Held
language : en
Publisher: CRC Press
Release Date : 2019-11-07
Handbook Of Infectious Disease Data Analysis written by Leonhard Held and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019-11-07 with Medical categories.
Recent years have seen an explosion in new kinds of data on infectious diseases, including data on social contacts, whole genome sequences of pathogens, biomarkers for susceptibility to infection, serological panel data, and surveillance data. The Handbook of Infectious Disease Data Analysis provides an overview of many key statistical methods that have been developed in response to such new data streams and the associated ability to address key scientific and epidemiological questions. A unique feature of the Handbook is the wide range of topics covered. Key features Contributors include many leading researchers in the field Divided into four main sections: Basic concepts, Analysis of Outbreak Data, Analysis of Seroprevalence Data, Analysis of Surveillance Data Numerous case studies and examples throughout Provides both introductory material and key reference material