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Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309455650
Category : Medical
Languages : en
Pages : 103
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Book Description
The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Potential effects on medical product development in the context of this wealth of real-world data could be numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to the ability to assess how therapeutics affect patients' lives through measurement of lifestyle changes. In October 2016, the National Academies of Sciences, Engineering, and Medicine held a workshop to facilitate dialogue among stakeholders about the opportunities and challenges for incorporating real-world evidence into all stages in the process for the generation and evaluation of therapeutics. Participants explored unmet stakeholder needs and opportunities to generate new kinds of evidence that meet those needs. This publication summarizes the presentations and discussions from the workshop.
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309455650
Category : Medical
Languages : en
Pages : 103
Get Book Here
Book Description
The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Potential effects on medical product development in the context of this wealth of real-world data could be numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to the ability to assess how therapeutics affect patients' lives through measurement of lifestyle changes. In October 2016, the National Academies of Sciences, Engineering, and Medicine held a workshop to facilitate dialogue among stakeholders about the opportunities and challenges for incorporating real-world evidence into all stages in the process for the generation and evaluation of therapeutics. Participants explored unmet stakeholder needs and opportunities to generate new kinds of evidence that meet those needs. This publication summarizes the presentations and discussions from the workshop.
Author: Harry Yang
Publisher: CRC Press
ISBN: 0429676824
Category : Mathematics
Languages : en
Pages : 191
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Book Description
Real-world evidence (RWE) has been at the forefront of pharmaceutical innovations. It plays an important role in transforming drug development from a process aimed at meeting regulatory expectations to an operating model that leverages data from disparate sources to aid business, regulatory, and healthcare decision making. Despite its many benefits, there is no single book systematically covering the latest development in the field. Written specifically for pharmaceutical practitioners, Real-World Evidence in Drug Development and Evaluation, presents a wide range of RWE applications throughout the lifecycle of drug product development. With contributions from experienced researchers in the pharmaceutical industry, the book discusses at length RWE opportunities, challenges, and solutions. Features Provides the first book and a single source of information on RWE in drug development Covers a broad array of topics on outcomes- and value-based RWE assessments Demonstrates proper Bayesian application and causal inference for real-world data (RWD) Presents real-world use cases to illustrate the use of advanced analytics and statistical methods to generate insights Offers a balanced discussion of practical RWE issues at hand and technical solutions suitable for practitioners with limited data science expertise
Author: Agency for Healthcare Research and Quality/AHRQ
Publisher: Government Printing Office
ISBN: 1587634333
Category : Medical
Languages : en
Pages : 385
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Book Description
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.
Author: Raymond A. Huml
Publisher: Springer Nature
ISBN: 3030786056
Category : Medical
Languages : en
Pages : 418
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Book Description
This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.
Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309113695
Category : Medical
Languages : en
Pages : 202
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Book Description
Drawing on the work of the Roundtable on Evidence-Based Medicine, the 2007 IOM Annual Meeting assessed some of the rapidly occurring changes in health care related to new diagnostic and treatment tools, emerging genetic insights, the developments in information technology, and healthcare costs, and discussed the need for a stronger focus on evidence to ensure that the promise of scientific discovery and technological innovation is efficiently captured to provide the right care for the right patient at the right time. As new discoveries continue to expand the universe of medical interventions, treatments, and methods of care, the need for a more systematic approach to evidence development and application becomes increasingly critical. Without better information about the effectiveness of different treatment options, the resulting uncertainty can lead to the delivery of services that may be unnecessary, unproven, or even harmful. Improving the evidence-base for medicine holds great potential to increase the quality and efficiency of medical care. The Annual Meeting, held on October 8, 2007, brought together many of the nation's leading authorities on various aspects of the issues - both challenges and opportunities - to present their perspectives and engage in discussion with the IOM membership.
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309457971
Category : Medical
Languages : en
Pages : 145
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Book Description
Advances in cancer research have led to an improved understanding of the molecular mechanisms underpinning the development of cancer and how the immune system responds to cancer. This influx of research has led to an increasing number and variety of therapies in the drug development pipeline, including targeted therapies and associated biomarker tests that can select which patients are most likely to respond, and immunotherapies that harness the body's immune system to destroy cancer cells. Compared with standard chemotherapies, these new cancer therapies may demonstrate evidence of benefit and clearer distinctions between efficacy and toxicity at an earlier stage of development. However, there is a concern that the traditional processes for cancer drug development, evaluation, and regulatory approval could impede or delay the use of these promising cancer treatments in clinical practice. This has led to a number of effortsâ€"by patient advocates, the pharmaceutical industry, and the Food and Drug Administration (FDA)â€"to accelerate the review of promising new cancer therapies, especially for cancers that currently lack effective treatments. However, generating the necessary data to confirm safety and efficacy during expedited drug development programs can present a unique set of challenges and opportunities. To explore this new landscape in cancer drug development, the National Academies of Sciences, Engineering, and Medicine developed a workshop held in December 2016. This workshop convened cancer researchers, patient advocates, and representatives from industry, academia, and government to discuss challenges with traditional approaches to drug development, opportunities to improve the efficiency of drug development, and strategies to enhance the information available about a cancer therapy throughout its life cycle in order to improve its use in clinical practice. This publication summarizes the presentations and discussions from the workshop.
Author: Agency for Health Care Research and Quality (U.S.)
Publisher: Government Printing Office
ISBN: 1587634236
Category : Medical
Languages : en
Pages : 236
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Book Description
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)
Author: Bernadette Mazurek Melnyk, PhD, APRN-CNP, FAANP, FNAP, FAAN
Publisher: Springer Publishing Company
ISBN: 0826109586
Category : Medical
Languages : en
Pages : 498
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Book Description
2012 First Place AJN Book of the Year Award Winner in Nursing Research! "This is a resource for success and should be a part of any researcher's library."--Doody's Medical Reviews This book is a practical, user-friendly guide for health care researchers across multiple disciplines who are involved in intervention research. It provides all of the essential elements needed for understanding how to design, conduct, analyze, and fund intervention studies that are replicable and can withstand the scrutiny of the Institutional Review Board and peer review. Developed from an annual continuing education workshop on intervention studies conducted by Dr. Melnyk, this text is the most comprehensive body of information available on this topic. Contributors address the design of interventions that are ethically considerate and sensitive to culture, race/ethnicity, and gender, minimizing threats to external and internal validity, measurement, and budgeting. The guide explores such implementation issues as subject recruitment and retention, data management, and specialized settings, cost analysis, and explaining intervention effects. The text also guides readers in writing grant applications that fund , and addresses how to move intervention study findings into the real world. A unique addition to the book is the availability of digital examples of progress reports, final reports, and research grant applications that have received funding from the National Institutes of Health and other relevant organizations. This text is a valuable resource for all health care professionals conducting research and for doctoral students in health care studies. Key Features: Presents the essential tools for designing, conducting, analyzing, and funding intervention studies Designed for use by health care professionals conducting intervention research Provides comprehensive, accessible guidelines for doctoral students across all health care disciplines Instructs readers on writing grant applications that fund Includes digital examples of funded research grants, progress reports, and final reports
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309455626
Category : Medical
Languages : en
Pages : 103
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Book Description
The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Potential effects on medical product development in the context of this wealth of real-world data could be numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to the ability to assess how therapeutics affect patients' lives through measurement of lifestyle changes. In October 2016, the National Academies of Sciences, Engineering, and Medicine held a workshop to facilitate dialogue among stakeholders about the opportunities and challenges for incorporating real-world evidence into all stages in the process for the generation and evaluation of therapeutics. Participants explored unmet stakeholder needs and opportunities to generate new kinds of evidence that meet those needs. This publication summarizes the presentations and discussions from the workshop.
Author: Rachel Richesson
Publisher: Springer Science & Business Media
ISBN: 1848824475
Category : Medical
Languages : en
Pages : 415
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Book Description
The purpose of the book is to provide an overview of clinical research (types), activities, and areas where informatics and IT could fit into various activities and business practices. This book will introduce and apply informatics concepts only as they have particular relevance to clinical research settings.
