Perspectives on Biomarker and Surrogate Endpoint Evaluation

Discussion Forum Summary

Author: Institute of Medicine,Food and Nutrition Board,Board on Health Sciences Policy,Board on Health Care Services,Committee on Qualification of Biomarkers and Surrogate Endpoints in Chronic Disease

Publisher: National Academies Press

ISBN: 9780309187008

Category: Medical

Page: 140

View: 7437

In 2010 the Institute of Medicine (IOM) recommended a framework for the evaluation of biomarkers in the chronic disease setting. Published in the book Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease, the framework is intended to bring consistency and transparency to the previously disparate process of biomarker evaluation. Following the book's release, the IOM convened a 2-day discussion forum in Washington, DC, in order to provide an opportunity for stakeholders to learn about, react to, and discuss the book. Presentations reviewed the authoring committee's work process, recommendations, and provided perspectives on the book from the point of view of participants. Thomas Fleming, professor of biostatistics and statistics at the University of Washington, gave a keynote presentation on the critical issues in the validation of surrogate endpoints, a specific use of a biomarker. The present volume recounts the discussion forum proceedings, focusing in turn on each represented sector. A summary of Dr. Fleming's presentation then sets the committee's recommendations within the context of biomarker utilization. Lastly, this summary examines the main themes raised by stakeholders, and the challenges and opportunities presented to stakeholders by the book's recommendations.

Science and the Citizen

Author: Marco Mamone Capria

Publisher: Lulu.com

ISBN: 1291446834

Category:

Page: N.A

View: 2397

Science Is Not What You Think

How It Has Changed, Why We Can't Trust It, How It Can Be Fixed

Author: Henry H. Bauer

Publisher: McFarland

ISBN: 1476669104

Category: Reference

Page: 260

View: 2081

 This book discusses the ways in which science, the touchstone of reliable knowledge in modern society, changed dramatically in the second half of the 20th century, becoming less trustworthy through conflicts of interest and excessive competitiveness. Fraud became common enough that organized efforts to combat it now include a federal Office of Research Integrity. Competent minority opinions are sometimes thereby suppressed, with the result that policy makers, the media and the public are presented with biased or incomplete information. Evidence tending to challenge established theories is sometimes rejected without addressing its substance. While most would agree in the abstract that science can go wrong, few would consider--despite interesting contrary evidence--that official consensus about the origins of the universe or the causes of global warming might be mistaken.

Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease

Author: Institute of Medicine,Food and Nutrition Board,Board on Health Sciences Policy,Board on Health Care Services,Committee on Qualification of Biomarkers and Surrogate Endpoints in Chronic Disease

Publisher: National Academies Press

ISBN: 0309157277

Category: Medical

Page: 334

View: 3414

Many people naturally assume that the claims made for foods and nutritional supplements have the same degree of scientific grounding as those for medication, but that is not always the case. The IOM recommends that the FDA adopt a consistent scientific framework for biomarker evaluation in order to achieve a rigorous and transparent process.

Braunwald. Tratado de cardiología + ExpertConsult

Texto de medicina cardiovascular

Author: Douglas L. Mann,Douglas P. Zipes,Peter Libby,Robert O. Bonow

Publisher: Elsevier España

ISBN: 8490229112

Category: Medical

Page: 2048

View: 3606

Obra de referencia en Cardiología que pretende seguir siendo un recurso fundamental en todos los aspectos de la Cardiología contemporánea proporcionando a clínicos, médicos en formació y estudiantes de todos los niveles las herramientas críticas para mantenerse al día en las bases científicas y los avances clínicos de la medicina cardiovascular. Ayuda al lector a aplicar los conocimientos más actuales en biología molecular y genética, imagen, farmacología, cardiología intervencional y electrofisiología entre otras cuestiones. Del total de los 89 capítulos que constituyen esta nueva edición, 27 son nuevos. Igualmente colaboran en esta nueva edición 53 nuevos autores, todos ellos reconocidos expertos internacionales en sus respectivas disciplinas. Todos los capítulos han sido revisados y actualizados en profundidad para recoger todas las novedades registradas en el área de la medicina cardiovascular. La nueva edición de esta obra de referencia incluye un total de 2.600 figuras, 600 tablas y 240 vídeos. Algunas de las novedades que incorpora esta nueva edición hacen referencia a imagen molecular, ultrasonido intravascular, regeneración cardiovascular e ingeniería de tejidos, terapias avanzadas para fallo cardiaco, manejo de la fibrilación atrial, enfermedad cardiaca estructural, cuestiones éticas en Medicina cardiovascular, cuestiones relacionadas con ensayos clínicos y otras cuestiones de interés. La nueva edición de este tratado de Cardiología presenta un acceso on line a través de www.ExpertConsult.com en el que los usuarios podrán acceder a la videoteca completa, actualizaciones en línea, lecturas adicionales y audios.

Sodium Intake in Populations

Assessment of Evidence

Author: Institute of Medicine,Board on Population Health and Public Health Practice,Food and Nutrition Board,Committee on the Consequences of Sodium Reduction in Populations

Publisher: National Academies Press

ISBN: 0309282985

Category: Medical

Page: 224

View: 8260

Despite efforts over the past several decades to reduce sodium intake in the United States, adults still consume an average of 3,400 mg of sodium every day. A number of scientific bodies and professional health organizations, including the American Heart Association, the American Medical Association, and the American Public Health Association, support reducing dietary sodium intake. These organizations support a common goal to reduce daily sodium intake to less than 2,300 milligrams and further reduce intake to 1,500 mg among persons who are 51 years of age and older and those of any age who are African-American or have hypertension, diabetes, or chronic kidney disease. A substantial body of evidence supports these efforts to reduce sodium intake. This evidence links excessive dietary sodium to high blood pressure, a surrogate marker for cardiovascular disease (CVD), stroke, and cardiac-related mortality. However, concerns have been raised that a low sodium intake may adversely affect certain risk factors, including blood lipids and insulin resistance, and thus potentially increase risk of heart disease and stroke. In fact, several recent reports have challenged sodium reduction in the population as a strategy to reduce this risk. Sodium Intake in Populations recognizes the limitations of the available evidence, and explains that there is no consistent evidence to support an association between sodium intake and either a beneficial or adverse effect on most direct health outcomes other than some CVD outcomes (including stroke and CVD mortality) and all-cause mortality. Some evidence suggested that decreasing sodium intake could possibly reduce the risk of gastric cancer. However, the evidence was too limited to conclude the converse-that higher sodium intake could possibly increase the risk of gastric cancer. Interpreting these findings was particularly challenging because most studies were conducted outside the United States in populations consuming much higher levels of sodium than those consumed in this country. Sodium Intake in Populations is a summary of the findings and conclusions on evidence for associations between sodium intake and risk of CVD-related events and mortality.

The Drug Development Paradigm in Oncology

Proceedings of a Workshop

Author: National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Care Services,National Cancer Policy Forum

Publisher: National Academies Press

ISBN: 0309457971

Category: Medical

Page: 144

View: 6710

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.

Evolution of Translational Omics:

Lessons Learned and the Path Forward

Author: Committee on the Review of Omics-Based Tests for Predicting Patient Outcomes in Clinical Trials,Board on Health Care Services,Board on Health Sciences Policy,Institute of Medicine

Publisher: National Academies Press

ISBN: 0309224187

Category: Science

Page: 338

View: 801

Technologies collectively called omics enable simultaneous measurement of an enormous number of biomolecules; for example, genomics investigates thousands of DNA sequences, and proteomics examines large numbers of proteins. Scientists are using these technologies to develop innovative tests to detect disease and to predict a patient's likelihood of responding to specific drugs. Following a recent case involving premature use of omics-based tests in cancer clinical trials at Duke University, the NCI requested that the IOM establish a committee to recommend ways to strengthen omics-based test development and evaluation. This report identifies best practices to enhance development, evaluation, and translation of omics-based tests while simultaneously reinforcing steps to ensure that these tests are appropriately assessed for scientific validity before they are used to guide patient treatment in clinical trials.

Novel Designs of Early Phase Trials for Cancer Therapeutics

Author: Shivaani Kummar,Chris Takimoto

Publisher: Academic Press

ISBN: 0128125705

Category: Medical

Page: 234

View: 9421

Novel Designs of Early Phase Trials for Cancer Therapeutics provides a comprehensive review by leaders in the field of the process of drug development, the integration of molecular profiling, the changes in early phase trial designs, and endpoints to optimally develop a new generation of cancer therapeutics. The book discusses topics such as statistical perspectives on cohort expansions, the role and application of molecular profiling and how to integrate biomarkers in early phase trials. Additionally, it discusses how to incorporate patient reported outcomes in phase one trials. This book is a valuable resource for medical oncologists, basic and translational biomedical scientists, and trainees in oncology and pharmacology who are interested in learning how to improve their research by using early phase trials. Brings a comprehensive review and recommendations for new clinical trial designs for modern cancer therapeutics Provides the reader with a better understanding on how to design and implement early phase oncology trials Presents a better and updated understanding of the process of developing new treatments for cancer, the exciting scientific advances and how they are informing drug development

Clinical Trials

A Methodologic Perspective

Author: Steven Piantadosi

Publisher: John Wiley & Sons

ISBN: 1118959213

Category: Mathematics

Page: 896

View: 1652

Presents elements of clinical trial methods that are essential in planning, designing, conducting, analyzing, and interpreting clinical trials with the goal of improving the evidence derived from these important studies This Third Edition builds on the text’s reputation as a straightforward, detailed, and authoritative presentation of quantitative methods for clinical trials. Readers will encounter the principles of design for various types of clinical trials, and are then skillfully guided through the complete process of planning the experiment, assembling a study cohort, assessing data, and reporting results. Throughout the process, the author alerts readers to problems that may arise during the course of the trial and provides common sense solutions. All stages of therapeutic development are discussed in detail, and the methods are not restricted to a single clinical application area. The authors bases current revisions and updates on his own experience, classroom instruction, and feedback from teachers and medical and statistical professionals involved in clinical trials. The Third Edition greatly expands its coverage, ranging from statistical principles to new and provocative topics, including alternative medicine and ethics, middle development, comparative studies, and adaptive designs. At the same time, it offers more pragmatic advice for issues such as selecting outcomes, sample size, analysis, reporting, and handling allegations of misconduct. Readers familiar with the First and Second Editions will discover revamped exercise sets; an updated and extensive reference section; new material on endpoints and the developmental pipeline, among others; and revisions of numerous sections. In addition, this book: • Features accessible and broad coverage of statistical design methods—the crucial building blocks of clinical trials and medical research -- now complete with new chapters on overall development, middle development, comparative studies, and adaptive designs • Teaches readers to design clinical trials that produce valid qualitative results backed by rigorous statistical methods • Contains an introduction and summary in each chapter to reinforce key points • Includes discussion questions to stimulate critical thinking and help readers understand how they can apply their newfound knowledge • Provides extensive references to direct readers to the most recent literature, and there are numerous new or revised exercises throughout the book Clinical Trials: A Methodologic Perspective, Third Edition is a textbook accessible to advanced undergraduate students in the quantitative sciences, graduate students in public health and the life sciences, physicians training in clinical research methods, and biostatisticians and epidemiologists. Steven Piantadosi, MD, PhD, is the Phase One Foundation Distinguished Chair and Director of the Samuel Oschin Cancer Institute, and Professor of Medicine at Cedars-Sinai Medical Center in Los Angeles, California. Dr. Piantadosi is one of the world’s leading experts in the design and analysis of clinical trials for cancer research. He has taught clinical trials methods extensively in formal courses and short venues. He has advised numerous academic programs and collaborations nationally regarding clinical trial design and conduct, and has served on external advisory boards for the National Institutes of Health and other prominent cancer programs and centers. The author of more than 260 peer-reviewed scientific articles, Dr. Piantadosi has published extensively on research results, clinical applications, and trial methodology. While his papers have contributed to many areas of oncology, he has also collaborated on diverse studies outside oncology including lung disease and degenerative neurological disease.

Handbook of Neuroemergency Clinical Trials

Author: Brett E. Skolnick,Wayne M. Alves

Publisher: Academic Press

ISBN: 0128041013

Category: Science

Page: 400

View: 5221

Handbook of Neuroemergency Clinical Trials, Second Edition, focuses on the practice of clinical trials in acute neuroscience populations, or what have been called neuroemergencies. Neuroemergencies are complex, life-threatening diseases and disorders, often with devastating consequences, including death or disability. The overall costs are staggering in terms of annual incidence and costs associated with treatment and survival, yet despite their significance as public health issues, there are few drugs and devices available for definitive treatment. The book focuses on novel therapies and the unique challenges their intended targets pose for the design and analysis of clinical trials. This volume provides neurologists, neuroscientists, and drug developers with a more complete understanding of the scientific and medical issues of relevance in designing and initiating clinical development plans for novel drugs intended for acute neuroscience populations. The editors provide the best understanding of the pitfalls associated with acute CNS drug development and the best information on how to approach and solve issues that have plagued drug development. Presents a comprehensive overview on clinical trials and drug development challenges in acute neuroscience populations Provides neurologists, neuroscientists and drug developers with a complete understanding of scientific and medical issues related to designing clinical trials Edited by leaders in the field who have designed and managed over 50 neuroemergency clinical trials

Emerging Technologies for Nutrition Research

Potential for Assessing Military Performance Capability

Author: Institute of Medicine,Committee on Military Nutrition Research

Publisher: National Academies Press

ISBN: 9780309174534

Category: Medical

Page: 728

View: 3210

The latest of a series of publications based on workshops sponsored by the Committee on Military Nutrition Research, this book's focus on emerging technologies for nutrition research arose from a concern among scientists at the U.S. Army Research Institute of Environmental Medicine that traditional nutrition research, using standard techniques, centered more on complex issues of the maintenance or enhancement of performance, and might not be sufficiently substantive either to measure changes in performance or to predict the effects on performance of stresses soldiers commonly experience in operational environments. The committee's task was to identify and evaluate new technologies to determine whether they could help resolve important issues in military nutrition research. The book contains the committee's summary and recommendations as well as individually authored chapters based on presentations at a 1995 workshop. Other chapters cover techniques of body composition assessment, tracer techniques for the study of metabolism, ambulatory techniques for the determination of energy expenditure, molecular and cellular approaches to nutrition, the assessment of immune function, and functional and behavioral measures of nutritional status.

The Evaluation of Surrogate Endpoints

Author: Tomasz Burzykowski,Geert Molenberghs,Marc Buyse

Publisher: Springer Science & Business Media

ISBN: 0387270809

Category: Medical

Page: 410

View: 1269

Covers the latest research on a sensitive and controversial topic in a professional and well researched manner. Provides practical outlook as well as model guidelines and software tools that should be of interest to people who use the software tools described and those who do not. Related title by Co-author Geert Molenbergh has sold more than 3500 copies world wide. Provides dual viewpoints: from scientists in the industry as well as regulatory authorities.

Statistics in a Nutshell

Author: Sarah Boslaugh

Publisher: "O'Reilly Media, Inc."

ISBN: 1449316824

Category: Mathematics

Page: 569

View: 4268

A clear and concise introduction and reference for anyone new to the subject of statistics.

Accelerating the Development of Biomarkers for Drug Safety:

Workshop Summary

Author: Forum on Drug Discovery, Development, and Translation,Board on Health Sciences Policy,Institute of Medicine

Publisher: National Academies Press

ISBN: 0309131243

Category: Medical

Page: 100

View: 2583

Biomarkers can be defined as indicators of any biologic state, and they are central to the future of medicine. As the cost of developing drugs has risen in recent years, reducing the number of new drugs approved for use, biomarker development may be a way to cut costs, enhance safety, and provide a more focused and rational pathway to drug development. On October 24, 2008, the IOM's Forum on Drug Discovery, Development, and Translation held "Assessing and Accelerating Development of Biomarkers for Drug Safety," a one-day workshop, summarized in this volume, on the value of biomarkers in helping to determine drug safety during development.

Rational Therapeutics for Infants and Children

Workshop Summary

Author: Institute of Medicine,Division of Health Sciences Policy,Roundtable on Research and Development of Drugs, Biologics, and Medical Devices,Jonathan R. Davis,Sarah Pitluck,Peter Bouxsein,Ronald W. Estabrook

Publisher: National Academies Press

ISBN: 9780309183642

Category: Medical

Page: 136

View: 4995

The Institute of Medicine's (IOM's) Roundtable on Research and Development of Drugs, Biologics, and Medical Devices evolved from the Forum on Drug Development, which was established in 1986. Sponsor representatives and IOM determined the importance of maintaining a neutral setting for discussions regarding long-term and politically sensitive issues justified the need to revise and enhance past efforts. The new Roundtable is intended to be a mechanism by which a broad group of experts from the public* and private sectors can be convened to conduct a dialogue and exchange information related to the development of drugs, biologics, and medical devices. Members have expertise in clinical medicine, pediatrics, clinical pharmacology, health policy, health insurance, industrial management, and product development; and they represent interests that address all facets of public policy issues. From time to time, the Roundtable requests that a workshop be conducted for the purpose of exploring a specific topic in detail and obtaining the views of additional experts. The first workshop for the Roundtable was held on April 14 and 15, 1998, and was entitled Assuring Data Quality and Validity in Clinical Trials for Regulatory Decision Making. The summary on that workshop is available from IOM. This workshop summary covers the second workshop, which was held on May 24 and 25, 1999, and which was aimed at facilitating the development and proper use of drugs, biologics, and medical devices for infants and children. It explores the scientific underpinnings and clinical needs, as well as the regulatory, legal, and ethical issues, raised by this area of research and development.

Integrating Clinical Research into Epidemic Response

The Ebola Experience

Author: National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Board on Global Health,Committee on Clinical Trials During the 2014-2015 Ebola Outbreak

Publisher: National Academies Press

ISBN: 0309457769

Category: Medical

Page: 342

View: 9126

The 2014â€"2015 Ebola epidemic in western Africa was the longest and most deadly Ebola epidemic in history, resulting in 28,616 cases and 11,310 deaths in Guinea, Liberia, and Sierra Leone. The Ebola virus has been known since 1976, when two separate outbreaks were identified in the Democratic Republic of Congo (then Zaire) and South Sudan (then Sudan). However, because all Ebola outbreaks prior to that in West Africa in 2014â€"2015 were relatively isolated and of short duration, little was known about how to best manage patients to improve survival, and there were no approved therapeutics or vaccines. When the World Heath Organization declared the 2014-2015 epidemic a public health emergency of international concern in August 2014, several teams began conducting formal clinical trials in the Ebola affected countries during the outbreak. Integrating Clinical Research into Epidemic Response: The Ebola Experience assesses the value of the clinical trials held during the 2014â€"2015 epidemic and makes recommendations about how the conduct of trials could be improved in the context of a future international emerging or re-emerging infectious disease events.