Author : Robert D. Gibbons
Publisher : CRC Press
Page : 297 pages
File Size : 23,8 MB
Release : 2015-07-28
Category : Mathematics
ISBN : 1466561858
Explore Important Tools for High-Quality Work in Pharmaceutical SafetyStatistical Methods for Drug Safety presents a wide variety of statistical approaches for analyzing pharmacoepidemiologic data. It covers both commonly used techniques, such as proportional reporting ratios for the analysis of spontaneous adverse event reports, and newer approach
Author : Thomas D. Cook
Publisher : CRC Press
Page : 465 pages
File Size : 10,75 MB
Release : 2007-11-19
Category : Mathematics
ISBN : 1584880279
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.
Author : Stephen S. Senn
Publisher : John Wiley & Sons
Page : 523 pages
File Size : 36,3 MB
Release : 2008-02-28
Category : Medical
ISBN : 9780470723579
Drug development is the process of finding and producingtherapeutically useful pharmaceuticals, turning them into safe andeffective medicine, and producing reliable information regardingthe appropriate dosage and dosing intervals. With regulatoryauthorities demanding increasingly higher standards in suchdevelopments, statistics has become an intrinsic and criticalelement in the design and conduct of drug development programmes. Statistical Issues in Drug Development presents anessential and thought provoking guide to the statistical issues andcontroversies involved in drug development. This highly readable second edition has been updated toinclude: Comprehensive coverage of the design and interpretation ofclinical trials. Expanded sections on missing data, equivalence, meta-analysisand dose finding. An examination of both Bayesian and frequentist methods. A new chapter on pharmacogenomics and expanded coverage ofpharmaco-epidemiology and pharmaco-economics. Coverage of the ICH guidelines, in particular ICH E9,Statistical Principles for Clinical Trials. It is hoped that the book will stimulate dialogue betweenstatisticians and life scientists working within the pharmaceuticalindustry. The accessible and wide-ranging coverage make itessential reading for both statisticians and non-statisticiansworking in the pharmaceutical industry, regulatory bodies andmedical research institutes. There is also much to benefitundergraduate and postgraduate students whose courses include amedical statistics component.
Author : Council for International Organizations of Medical Sciences (CIOMS)
Publisher : World Health Organization
Page : 0 pages
File Size : 47,2 MB
Release : 2016
Category : Drugs
ISBN : 9789290360858
At any point in the drug development process, systematic reviews and meta-analysis can provide important information to guide the future path of the development program and any actions that might be needed in the post-marketing setting. This report gives the rationale for why and when a meta-analysis should be considered, all in the context of regulatory decision-making, and the tasks, data collection, and analyses that need to be carried out to inform those decisions. There is increasing demand by decision-makers in health care, the bio-pharmaceutical industry, and society at large to have access to the best available evidence on benefits and risks of medicinal products. The best strategy will take an overview of all the evidence and where it is possible and sensible, combine the evidence and summarize the results. For efficacy, the outcomes generally use the same or very similar predefined events for each of the trials to be included. Most regulatory guidance and many Cochrane Collaboration reviews have usually given more attention to assessment of benefits, while issues around combining evidence on harms have not been as well-covered. However, the (inevitably) unplanned nature of the data on safety makes the process more difficult. Combining evidence on adverse events (AEs), where these were not the focus of the original studies, is more challenging than combining evidence on pre-specified benefits. This focus on AEs represents the main contribution of the current CIOMS X report. The goal of the CIOMS X report is to provide principles on appropriate application of meta-analysis in assessing safety of pharmaceutical products to inform regulatory decision-making. This report is about meta-analysis in this narrow area, but the present report should also provide conceptually helpful points to consider for a wider range of applications, such as vaccines, medical devices, veterinary medicines or even products that are combinations of medicinal products and medical devices. Although some of the content of this report describes highly technical statistical concepts and methods (in particular Chapter 4), the ambition of the working group has been to make it comprehensible to non-statisticians for its use in clinical epidemiology and regulatory science. To that end, Chapters 3 and 4, which contain the main technical statistical aspects of the appropriate design, analysis and reporting of a meta-analysis of safety data are followed by Chapter 5 with a thought process for evaluating the findings of a meta-analysis and how to communicate these.
Author : Institute of Medicine
Publisher : National Academies Press
Page : 221 pages
File Size : 43,71 MB
Release : 2001-01-01
Category : Medical
ISBN : 0309171148
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.
Author : William Wang
Publisher : CRC Press
Page : 347 pages
File Size : 16,38 MB
Release : 2021-12-30
Category : Mathematics
ISBN : 0429949995
Quantitative Methodologies and Process for Safety Monitoring and Ongoing Benefit Risk Evaluation provides a comprehensive coverage on safety monitoring methodologies, covering both global trends and regional initiatives. Pharmacovigilance has traditionally focused on the handling of individual adverse event reports however recently there had been a shift towards aggregate analysis to better understand the scope of product risks. Written to be accessible not only to statisticians but also to safety scientists with a quantitative interest, this book aims to bridge the gap in knowledge between medical and statistical fields creating a truly multi-disciplinary approach that is very much needed for 21st century safety evaluation.
Author : National Research Council
Publisher : National Academies Press
Page : 163 pages
File Size : 38,44 MB
Release : 2010-12-21
Category : Medical
ISBN : 030918651X
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.
Author : William Wang
Publisher : CRC Press
Page : 382 pages
File Size : 44,62 MB
Release : 2021-12-31
Category : Mathematics
ISBN : 9780429488801
"Quantitative Methodologies and Process for Safety Monitoring and Ongoing Benefit Risk Evaluation provides a comprehensive coverage on safety monitoring methodologies, covering both global trends and regional initiatives. Pharmacovigilance has traditionally focused on the handling of individual adverse event reports however recently there had been a shift towards aggregate analysis to better understand the scope of product risks. Written to be accessible not only to statisticians but also to safety scientists with a quantitative interest, this book aims to bridge the gap in knowledge between medical and statistical fields creating a truly multi-disciplinary approach that is very much needed for 21st century safety evaluation"--
Author : Institute of Medicine
Publisher : National Academies Press
Page : 236 pages
File Size : 15,39 MB
Release : 2015-04-20
Category : Medical
ISBN : 0309316324
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.
Author : Frederick W. Faltin
Publisher : John Wiley & Sons
Page : 533 pages
File Size : 41,22 MB
Release : 2012-07-24
Category : Medical
ISBN : 1119942047
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.
