Download or read book Statistical Methods for Evaluating Safety in Medical Product Development written by A. Lawrence Gould and published by John Wiley & Sons. This book was released on 2015-02-23 with total page 390 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book gives professionals in clinical research valuable information on the challenging issues of the design, execution, and management of clinical trials, and how to resolve these issues effectively. It also provides understanding and practical guidance on the application of contemporary statistical methods to contemporary issues in safety evaluation during medical product development. Each chapter provides sufficient detail to the reader to undertake the design and analysis of experiments at various stages of product development, including comprehensive references to the relevant literature. Provides a guide to statistical methods and application in medical product development Assists readers in undertaking design and analysis of experiments at various stages of product development Features case studies throughout the book, as well as, SAS and R code

Download or read book Medical Product Safety Evaluation written by Jie Chen and published by CRC Press. This book was released on 2018-09-03 with total page 372 pages. Available in PDF, EPUB and Kindle. Book excerpt: Medical Product Safety Evaluation: Biological Models and Statistical Methods presents cutting-edge biological models and statistical methods that are tailored to specific objectives and data types for safety analysis and benefit-risk assessment. Some frequently encountered issues and challenges in the design and analysis of safety studies are discussed with illustrative applications and examples. Medical Product Safety Evaluation: Biological Models and Statistical Methods presents cutting-edge biological models and statistical methods that are tailored to specific objectives and data types for safety analysis and benefit-risk assessment. Some frequently encountered issues and challenges in the design and analysis of safety studies are discussed with illustrative applications and examples. The book is designed not only for biopharmaceutical professionals, such as statisticians, safety specialists, pharmacovigilance experts, and pharmacoepidemiologists, who can use the book as self-learning materials or in short courses or training programs, but also for graduate students in statistics and biomedical data science for a one-semester course. Each chapter provides supplements and problems as more readings and exercises.

Download or read book Medical Product Safety Evaluation written by Jie Chen and published by CRC Press. This book was released on 2018-09-03 with total page 220 pages. Available in PDF, EPUB and Kindle. Book excerpt: Medical Product Safety Evaluation: Biological Models and Statistical Methods presents cutting-edge biological models and statistical methods that are tailored to specific objectives and data types for safety analysis and benefit-risk assessment. Some frequently encountered issues and challenges in the design and analysis of safety studies are discussed with illustrative applications and examples. Medical Product Safety Evaluation: Biological Models and Statistical Methods presents cutting-edge biological models and statistical methods that are tailored to specific objectives and data types for safety analysis and benefit-risk assessment. Some frequently encountered issues and challenges in the design and analysis of safety studies are discussed with illustrative applications and examples. The book is designed not only for biopharmaceutical professionals, such as statisticians, safety specialists, pharmacovigilance experts, and pharmacoepidemiologists, who can use the book as self-learning materials or in short courses or training programs, but also for graduate students in statistics and biomedical data science for a one-semester course. Each chapter provides supplements and problems as more readings and exercises.

Download or read book Statistical Methods in Healthcare written by Frederick W. Faltin and published by John Wiley & Sons. This book was released on 2012-07-24 with total page 533 pages. Available in PDF, EPUB and Kindle. Book excerpt: Statistical Methods in Healthcare In recent years the number of innovative medicinal products and devices submitted and approved by regulatory bodies has declined dramatically. The medical product development process is no longer able to keep pace with increasing technologies, science and innovations and the goal is to develop new scientific and technical tools and to make product development processes more efficient and effective. Statistical Methods in Healthcare focuses on the application of statistical methodologies to evaluate promising alternatives and to optimize the performance and demonstrate the effectiveness of those that warrant pursuit is critical to success. Statistical methods used in planning, delivering and monitoring health care, as well as selected statistical aspects of the development and/or production of pharmaceuticals and medical devices are also addressed. With a focus on finding solutions to these challenges, this book: Provides a comprehensive, in-depth treatment of statistical methods in healthcare, along with a reference source for practitioners and specialists in health care and drug development. Offers a broad coverage of standards and established methods through leading edge techniques. Uses an integrated case study based approach, with focus on applications. Looks at the use of analytical and monitoring schemes to evaluate therapeutic performance. Features the application of modern quality management systems to clinical practice, and to pharmaceutical development and production processes. Addresses the use of modern statistical methods such as Adaptive Design, Seamless Design, Data Mining, Bayesian networks and Bootstrapping that can be applied to support the challenging new vision. Practitioners in healthcare-related professions, ranging from clinical trials to care delivery to medical device design, as well as statistical researchers in the field, will benefit from this book.

Download or read book Biopharmaceutical Applied Statistics Symposium written by Karl E. Peace and published by Springer. This book was released on 2018-08-21 with total page 251 pages. Available in PDF, EPUB and Kindle. Book excerpt: This BASS book Series publishes selected high-quality papers reflecting recent advances in the design and biostatistical analysis of biopharmaceutical experiments – particularly biopharmaceutical clinical trials. The papers were selected from invited presentations at the Biopharmaceutical Applied Statistics Symposium (BASS), which was founded by the first Editor in 1994 and has since become the premier international conference in biopharmaceutical statistics. The primary aims of the BASS are: 1) to raise funding to support graduate students in biostatistics programs, and 2) to provide an opportunity for professionals engaged in pharmaceutical drug research and development to share insights into solving the problems they encounter. The BASS book series is initially divided into three volumes addressing: 1) Design of Clinical Trials; 2) Biostatistical Analysis of Clinical Trials; and 3) Pharmaceutical Applications. This book is the second of the 3-volume book series. The topics covered include: Statistical Approaches to the Meta-analysis of Randomized Clinical Trials, Collaborative Targeted Maximum Likelihood Estimation to Assess Causal Effects in Observational Studies, Generalized Tests in Clinical Trials, Discrete Time-to-event and Score-based Methods with Application to Composite Endpoint for Assessing Evidence of Disease Activity-Free , Imputing Missing Data Using a Surrogate Biomarker: Analyzing the Incidence of Endometrial Hyperplasia, Selected Statistical Issues in Patient-reported Outcomes, Network Meta-analysis, Detecting Safety Signals Among Adverse Events in Clinical Trials, Applied Meta-analysis Using R, Treatment of Missing Data in Comparative Effectiveness Research, Causal Estimands: A Common Language for Missing Data, Bayesian Subgroup Analysis with Examples, Statistical Methods in Diagnostic Devices, A Question-Based Approach to the Analysis of Safety Data, Analysis of Two-stage Adaptive Seamless Trial Design, and Multiplicity Problems in Clinical Trials – A Regulatory Perspective.

Download or read book Statistical Approaches in Oncology Clinical Development written by Satrajit Roychoudhury and published by CRC Press. This book was released on 2018-12-07 with total page 237 pages. Available in PDF, EPUB and Kindle. Book excerpt: Statistical Approaches in Oncology Clinical Development : Current Paradigm and Methodological Advancement presents an overview of statistical considerations in oncology clinical trials, both early and late phase of development. It illustrates how novel statistical methods can enrich the design and analysis of modern oncology trials. The authors include many relevant real life examples from the pharmaceutical industry and academia based on their first-hand experience. Along with relevant references, the book highlights current regulatory views. The book covers all aspects of cancer clinical trial starting from early phase development. The early part of the book covers novel phase I dose escalation design, exposure response analysis, and innovative phase II design. This includes early development strategy for cancer immunotherapy trials. The contributors also emphasized the role of biomarker and modern era of precision medicine. The second part focuses on the late stage development. This includes the application of adaptive design, safety analysis, and quality of life (QoL) data analysis. The final part discusses current regulatory perspective and challenges. Features: Covers a wide spectrum of topics related to real-life statistical challenges in oncology clinical trials. Provides a comprehensive overview of novel statistical methods to improve trial design and statistical analysis. Detailed case studies illustrate the real life applications. Satrajit Roychoudhury is a Senior Director and a member of the Statistical Research and Innovation group in Pfizer Inc. Prior to joining; he was a member of Statistical Methodology and consulting group in Novartis. He has 11 years of extensive experience in working with different phases of clinical trial. His area of research includes early phase oncology trials, survival analysis, model informed drug development, and use of Bayesian methods in clinical trials. He is industry co-chair for the ASA Biopharmaceutical Section Regulatory-Industry Workshop and has provided statistical training in major conferences including the Joint Statistical Meetings, ASA Biopharmaceutical Section Regulatory-Industry Workshop, and ICSA Applied Statistics Symposium. Soumi Lahiri has 12 years of extensive experience in working different therapeutic areas. She is the former Director of Biostatistics in Clinical Oncology, GlaxoSmithKline. She has also worked in the oncology division of Novartis Pharmaceutical Company for two years. She is an active member of the ASA Biopharmaceutical section and former chair of the membership committee.

Download or read book Data and Safety Monitoring Committees in Clinical Trials Second Edition written by Jay Herson and published by CRC Press. This book was released on 2016-12-19 with total page 179 pages. Available in PDF, EPUB and Kindle. Book excerpt: Praise for the first edition: "Given the author’s years of experience as a statistician and as a founder of the first DMC in pharmaceutical industry trials, I highly recommend this book—not only for experts because of its cogent and organized presentation, but more importantly for young investigators who are seeking information about the logistical and philosophical aspects of a DMC." -S. T. Ounpraseuth, The American Statistician ? In the first edition of this well-regarded book, the author provided a groundbreaking and definitive guide to best practices in pharmaceutical industry data monitoring committees (DMCs). Maintaining all the material from the first edition and adding substantial new material, Data and Safety Monitoring Committees in Clinical Trials, Second Edition is ideal for training professionals to serve on their first DMC as well as for experienced clinical and biostatistical DMC members, sponsor and regulatory agency staff. The second edition guides the reader through newly emerging DMC responsibilities brought about by regulations emphasizing risk vs benefit and the emergence of risk-based monitoring. It also provides the reader with many new statistical methods, clinical trial designs and clinical terminology that have emerged since the first edition. The references have been updated and the very popular end-of-chapter Q&A section has been supplemented with many new experiences since the first edition. ? New to the Second Edition: Presents statistical methods, tables, listings and graphs appropriate for safety review, efficacy analysis and risk vs benefit analysis, SPERT and PRISMA initiatives. Newly added interim analysis for efficacy and futility section. DMC responsibilities in SUSARs (Serious Unexpected Serious Adverse Reactions), basket trials, umbrella trials, dynamic treatment strategies /SMART trials, pragmatic trials, biosimilar trials, companion diagnostics, etc. DMC responsibilities for data quality and fraud detection (Fraud Recovery Plan) Use of patient reported outcomes of safety Use of meta analysis and data outside the trial New ideas for training and compensation of DMC members ? Jay Herson is Senior Associate, Biostatistics, Johns Hopkins Bloomberg School of Public Health where he teaches courses on clinical trials and drug development based on his many years experience in clinical trials in academia and the pharmaceutical industry. ? ? ? ? ? ? ? ? ? ? ? ? ?

Download or read book The Statistical Evaluation of Medical Tests for Classification and Prediction written by Margaret Sullivan Pepe and published by OUP Oxford. This book was released on 2003-03-13 with total page 319 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book describes statistical techniques for the design and evaluation of research studies on medical diagnostic tests, screening tests, biomarkers and new technologies for classification and prediction in medicine.

Download or read book Registries for Evaluating Patient Outcomes written by Agency for Healthcare Research and Quality/AHRQ and published by Government Printing Office. This book was released on 2014-04-01 with total page 385 pages. Available in PDF, EPUB and Kindle. Book excerpt: This User’s Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase understanding of patient outcomes. For the purposes of this guide, a patient registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes. A registry database is a file (or files) derived from the registry. Although registries can serve many purposes, this guide focuses on registries created for one or more of the following purposes: to describe the natural history of disease, to determine clinical effectiveness or cost-effectiveness of health care products and services, to measure or monitor safety and harm, and/or to measure quality of care. Registries are classified according to how their populations are defined. For example, product registries include patients who have been exposed to biopharmaceutical products or medical devices. Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization. Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews.

Download or read book Planning Writing and Reviewing Medical Device Clinical and Performance Evaluation Reports CERs PERs written by Joy Frestedt and published by Elsevier. This book was released on 2024-09-27 with total page 547 pages. Available in PDF, EPUB and Kindle. Book excerpt: A Practical Guide to Planning, Writing, and Reviewing Medical Device Clinical Evaluation Reports guides readers through clinical data evaluation of medical devices, in compliance with the EU MDR requirements and other similar regulatory requirements throughout the world. This book brings together knowledge learned as the author constructed hundreds of CERs and taught thousands of learners on how to conduct clinical data evaluations. This book will support training for clinical engineers, clinical evaluation scientists, and experts reviewing medical device CERs, and will help individual writers, teams and companies to develop stronger, more robust CERs. Identifies and explains data analysis for clinical evaluation of medical devices Teaches readers how to understand and evaluate medical device performance and safety in the context of new regulations Provides analysis of new clinical evaluation criteria in the context of medical device design as well as in-hospital deployment and servicing

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 Sharing Clinical Trial Data written by Institute of Medicine and published by National Academies Press. This book was released on 2015-04-20 with total page 236 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Strategy and Statistics in Clinical Trials written by Joseph Tal and published by Academic Press. This book was released on 2011-07-14 with total page 279 pages. Available in PDF, EPUB and Kindle. Book excerpt: Delineates the statistical building blocks and concepts of clinical trials.

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 The Prevention and Treatment of Missing Data in Clinical Trials written by National Research Council and published by National Academies Press. This book was released on 2010-12-21 with total page 163 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Handbook of Statistical Methods for Randomized Controlled Trials written by KyungMann Kim and published by CRC Press. This book was released on 2021-08-23 with total page 655 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book AMSTAT News written by American Statistical Association and published by . This book was released on 2008 with total page 504 pages. Available in PDF, EPUB and Kindle. Book excerpt: