Perspectives on Biomarker and Surrogate Endpoint Evaluation

Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 9780309187008
Format: PDF, ePub, Docs
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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.

Pharmaco Imaging in Drug and Biologics Development

Author: Brian R. Moyer
Publisher: Springer Science & Business Media
ISBN: 146148247X
Format: PDF, ePub, Docs
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The volume aim to be a comprehensive overview of the drug and biologic development process that is often called “the valley of death” (pre-IND through approval) where high costs of studies and high rates of product failure are part of the drug development landscape. Imaging tools can serve in this period by adding high value data, the images and the kinetic information they can provide, and cost-effective development alternative tools which potentially improve pivotal study designs. Imaging may identify safety issues early such as unwanted organ or tissue distributions, and then can serve advanced development with added certainty of a drug or biologic’s success to senior corporate management and investors. There are numerous textbooks, reference texts and treatises on medical imaging technologies, teaching tools on medical cases and physics books on the science of detector and computer interface systems. Rarely, in each of these are examples of medical imaging protocols and animal models of disease i.e. a text on methodology in drug development is currently unavailable.

Science Is Not What You Think

Author: Henry H. Bauer
Publisher: McFarland
ISBN: 1476669104
Format: PDF, Mobi
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 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: Committee on Qualifications of Biomarkers and Surrogate Endpoints in Chronic Disease
Publisher: National Academies Press
ISBN: 0309151295
Format: PDF, ePub, Docs
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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.

Sodium Intake in Populations

Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309282985
Format: PDF, ePub, Docs
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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

Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309457971
Format: PDF, ePub, Mobi
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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.

Rational Therapeutics for Infants and Children

Author: Roundtable on Research and Development of Drugs, Biologics, and Medical Devices
Publisher: National Academies Press
ISBN: 0309069378
Format: PDF, Kindle
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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.

Clinical Trials

Author: Steven Piantadosi
Publisher: John Wiley & Sons
ISBN: 1118959213
Format: PDF, Docs
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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.

Braunwald Tratado de cardiolog a ExpertConsult

Author: Douglas L. Mann
Publisher: Elsevier España
ISBN: 8490229112
Format: PDF, ePub
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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.