Download or read book Planning and Analyzing Clinical Trials with Composite Endpoints written by Geraldine Rauch and published by Springer. This book was released on 2018-05-22 with total page 255 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book addresses the most important aspects of how to plan and evaluate clinical trials with a composite primary endpoint to guarantee a clinically meaningful and valid interpretation of the results. Composite endpoints are often used as primary efficacy variables for clinical trials, particularly in the fields of oncology and cardiology. These endpoints combine several variables of interest within a single composite measure, and as a result, all variables that are of major clinical relevance can be considered in the primary analysis without the need to adjust for multiplicity. Moreover, composite endpoints are intended to increase the size of the expected effects thus making clinical trials more powerful. The book offers practical advice for statisticians and medical experts involved in the planning and analysis of clinical trials. For readers who are mainly interested in the application of the methods, all the approaches are illustrated with real-world clinical trial examples, and the software codes required for fast and easy implementation are provided. The book also discusses all the methods in the context of relevant guidelines related to the topic. To benefit most from the book, readers should be familiar with the principles of clinical trials and basic statistical methods.

Download or read book Methods for Planning and Evaluating Clinical Trials with Composite Endpoints written by Geraldine Rauch and published by . This book was released on 2014 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Methods for Planning and Analyzing Clinical Trials with Composite Endpoints written by Svenja Schüler and published by . This book was released on 2020 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Developments in Statistical Evaluation of Clinical Trials written by Kees van Montfort and published by Springer. This book was released on 2014-10-07 with total page 364 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book describes various ways of approaching and interpreting the data produced by clinical trial studies, with a special emphasis on the essential role that biostatistics plays in clinical trials. Over the past few decades the role of statistics in the evaluation and interpretation of clinical data has become of paramount importance. As a result the standards of clinical study design, conduct and interpretation have undergone substantial improvement. The book includes 18 carefully reviewed chapters on recent developments in clinical trials and their statistical evaluation, with each chapter providing one or more examples involving typical data sets, enabling readers to apply the proposed procedures. The chapters employ a uniform style to enhance comparability between the approaches.

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 Composite Endpoints in Clinical Trials Computational Tools Practical Guidelines and Methodological Extensions written by Moisés Gómez-Mateu and published by . This book was released on 2016 with total page 168 pages. Available in PDF, EPUB and Kindle. Book excerpt: The conclusions from randomized clinical trials (RCT) rely on the primary endpoint (PE), which is chosen at the design stage of the study; thus, it is of utmost importance to select it appropriately. In RCT, there should generally be only one PE, and it should be able to provide the most clinically relevant and scientific evidence regarding the potential efficacy of the new treatment. Composite endpoints (CE) consist of the union of two or more outcomes and are often used in RCT. When the focus is time-to-event analysis, CE refer to the elapse time from randomization until the first component of the CE. In oncology trials, for instance, progression-free survival is defined as the time to disease progression or death. The decision on whether to use a CE versus a single component as the PE is controversial. The advantages and drawbacks regarding the use of CE have been extensively discussed in the literature. Gómez and Lagakos develop a statistical methodology to evaluate the convenience of using a relevant endpoint RE versus a CE consisting of the union of the RE plus another additional endpoint (AE). Their strategy is based on the value of the asymptotic relative efficiency (ARE), which relates the efficiency of using the logrank test based on the RE versus the efficiency based on the CE. The ARE is expressed as a function of the marginal laws of the time to each component RE and AE, the probabilities of observing each component in the control group, the hazard ratios measured by each component of the CE between the two treatment groups, and the correlation between components. This thesis explores, elaborates on, implements and applies the ARE method. We have also developed a new online platform named CompARE that facilitates the practical use of this method. The ARE method has been applied to cardiovascular studies. We have made further progress into the theoretical meaning of the ARE and have explored how to handle the probability and the hazard ratio of a combination of endpoints. In cardiovascular trials, it is common to use CE. We systematically examine the use of CE in this field by means of a literature search and the discussion of several case studies. Based on the ARE methodology, we provide guidelines for the informed choice of the PE. We prove that the usual interpretation of the ARE as the ratio of sample sizes holds and that it can be applied to evaluate the efficiency of the RE versus the CE. Furthermore, we carry out a simulation study to empirically check the proximity between the ratio of finite sample sizes and the ARE. We discuss how to derive the probabilities and hazard ratios when they come from a combination of several components. Furthermore, it is shown that the combined hazard ratio (HR*) is, in general, not constant over time, even if the hazard ratio of the marginal components are. This non-constant behaviour might have a strong influence on the interpretation of treatment effect and on sample size assessment. We evaluate the behaviour of the HR* in respect to the marginal parameters, and we study its departure from constancy, depending on different scenarios. This thesis has implemented the ARE methodology on the online platform CompARE. Clinicians and biostatisticians can use CompARE to study the performance of different endpoints in a variety of scenarios. CompARE has an intuitive interface and it is a convenient tool for better informed decisions regarding the PE. Results from different parameter settings are shown immediately by means of tables and plots. CompARE is extended to quantify specific values for the combined probability and hazard ratios. When the user cannot anticipate some of the needed parameters, CompARE provides a range of plausible values. Moreover, the departure from constancy of a combined hazard ratio can be explored by visualizing its shape over time. Sample size computations are implemented as well.

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 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 396 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 Composite Time to event Endpoints in Clinical and Epidemiologic Trials written by Ann-Kathrin Ozga and published by . This book was released on 2020 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Primary Pulmonary Hypertension written by Lewis J. Rubin and published by CRC Press. This book was released on 1996-11-06 with total page 384 pages. Available in PDF, EPUB and Kindle. Book excerpt: Detailing state-of-the-art developments in the various aspects of primary pulmonary hypertension (PPH), this practical reference explores the history, most current scientific concepts, and treatments of this disease. Includes new advances not yet formally published! Written by nearly 30 of the top international experts in the field, Primary Pulmonary Hypertension addresses the general histological features of the normal and hypertensive pulmonary vasculature and the pathology of PPH discusses etiological possibilities of pathogenesis, common morphological features, and findings in experimental models examines risks factors for PPH and looks separately at familial PPH and PPH in children presents an approach to the differential diagnosis of pulmonary hypertension, emphasizing the recognition of PPH elucidates the invasive and noninvasive modalities available for obtaining qualitative and quantitative hemodynamic data for the diagnosis of PPH covers a variety of therapeutic options and much more!

Download or read book Multiple Testing Problems in Pharmaceutical Statistics written by Alex Dmitrienko and published by CRC Press. This book was released on 2009-12-08 with total page 323 pages. Available in PDF, EPUB and Kindle. Book excerpt: Useful Statistical Approaches for Addressing Multiplicity IssuesIncludes practical examples from recent trials Bringing together leading statisticians, scientists, and clinicians from the pharmaceutical industry, academia, and regulatory agencies, Multiple Testing Problems in Pharmaceutical Statistics explores the rapidly growing area of multiple c

Download or read book Adaptive Design Methods in Clinical Trials written by Shein-Chung Chow and published by CRC Press. This book was released on 2011-12-01 with total page 368 pages. Available in PDF, EPUB and Kindle. Book excerpt: With new statistical and scientific issues arising in adaptive clinical trial design, including the U.S. FDA's recent draft guidance, a new edition of one of the first books on the topic is needed. Adaptive Design Methods in Clinical Trials, Second Edition reflects recent developments and regulatory positions on the use of adaptive designs in clini

Download or read book Developing a Protocol for Observational Comparative Effectiveness Research A User s Guide written by Agency for Health Care Research and Quality (U.S.) and published by Government Printing Office. This book was released on 2013-02-21 with total page 236 pages. Available in PDF, EPUB and Kindle. Book excerpt: This User’s Guide is a resource for investigators and stakeholders who develop and review observational comparative effectiveness research protocols. It explains how to (1) identify key considerations and best practices for research design; (2) build a protocol based on these standards and best practices; and (3) judge the adequacy and completeness of a protocol. Eleven chapters cover all aspects of research design, including: developing study objectives, defining and refining study questions, addressing the heterogeneity of treatment effect, characterizing exposure, selecting a comparator, defining and measuring outcomes, and identifying optimal data sources. Checklists of guidance and key considerations for protocols are provided at the end of each chapter. 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. More more information, please consult the Agency website: www.effectivehealthcare.ahrq.gov)

Download or read book Pharmaceutical Medicine written by Adrian Kilcoyne and published by OUP Oxford. This book was released on 2013-05-23 with total page 473 pages. Available in PDF, EPUB and Kindle. Book excerpt: The breadth of the pharmaceutical medicine can be daunting, but this book is designed to navigate a path through the speciality. Providing a broad overview of all topics relevant to the discipline of pharmaceutical medicine, it gives you the facts fast, in a user-friendly format, without having to dive through page upon page of dense text. With 136 chapters spread across 8 sections, the text offers a thorough grounding in issues ranging from medicines regulation to clinical trial design and data management. This makes it a useful revision aid for exams as well as giving you a taster of areas of pharmaceutical medicine adjacent to your current role. For healthcare professionals already working in the field, this book offers a guiding hand in difficult situations as well as supplying rapid access to the latest recommendations and guidelines. Written by authors with experience in the industry and drug regulation, this comprehensive and authoritative guide provides a shoulder to lean on throughout your pharmaceutical career.

Download or read book Oxford Textbook of Global Public Health written by Roger Detels and published by Oxford University Press. This book was released on 2017 with total page 1717 pages. Available in PDF, EPUB and Kindle. Book excerpt: Sixth edition of the hugely successful, internationally recognised textbook on global public health and epidemiology, with 3 volumes comprehensively covering the scope, methods, and practice of the discipline

Download or read book Prognosis Research in Healthcare written by Richard D. Riley and published by Oxford University Press. This book was released on 2019-01-17 with total page 384 pages. Available in PDF, EPUB and Kindle. Book excerpt: "What is going to happen to me?" Most patients ask this question during a clinical encounter with a health professional. As well as learning what problem they have (diagnosis) and what needs to be done about it (treatment), patients want to know about their future health and wellbeing (prognosis). Prognosis research can provide answers to this question and satisfy the need for individuals to understand the possible outcomes of their condition, with and without treatment. Central to modern medical practise, the topic of prognosis is the basis of decision making in healthcare and policy development. It translates basic and clinical science into practical care for patients and populations. Prognosis Research in Healthcare: Concepts, Methods and Impact provides a comprehensive overview of the field of prognosis and prognosis research and gives a global perspective on how prognosis research and prognostic information can improve the outcomes of healthcare. It details how to design, carry out, analyse and report prognosis studies, and how prognostic information can be the basis for tailored, personalised healthcare. In particular, the book discusses how information about the characteristics of people, their health, and environment can be used to predict an individual's future health. Prognosis Research in Healthcare: Concepts, Methods and Impact, addresses all types of prognosis research and provides a practical step-by-step guide to undertaking and interpreting prognosis research studies, ideal for medical students, health researchers, healthcare professionals and methodologists, as well as for guideline and policy makers in healthcare wishing to learn more about the field of prognosis.

Download or read book Assessing Medical Technologies written by Institute of Medicine and published by National Academies Press. This book was released on 1985-02-01 with total page 592 pages. Available in PDF, EPUB and Kindle. Book excerpt: New drugs, new devices, improved surgical techniques, and innovative diagnostic procedures and equipment emerge rapidly. But development of these technologies has outpaced evaluation of their safety, efficacy, cost-effectiveness, and ethical and social consequences. This volume, which is "strongly recommended" by The New England Journal of Medicine "to all those interested in the future of the practice of medicine," examines how new discoveries can be translated into better care, and how the current system's inefficiencies prevent effective health care delivery. In addition, the book offers detailed profiles of 20 organizations currently involved in medical technology assessment, and proposes ways to organize U.S. efforts and create a coordinated national system for evaluating new medical treatments and technology.