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 Safe and Effective Medicines for Children written by Institute of Medicine and published by National Academies Press. This book was released on 2012-10-13 with total page 432 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were designed to encourage more pediatric studies of drugs used for children. The FDA asked the IOM to review aspects of pediatric studies and changes in product labeling that resulted from BPCA and PREA and their predecessor policies, as well as assess the incentives for pediatric studies of biologics and the extent to which biologics have been studied in children. The IOM committee concludes that these policies have helped provide clinicians who care for children with better information about the efficacy, safety, and appropriate prescribing of drugs. The IOM suggests that more can be done to increase knowledge about drugs used by children and thereby improve the clinical care, health, and well-being of the nation's children.

Download or read book Observation and Experiment written by Paul Rosenbaum and published by Harvard University Press. This book was released on 2017-08-14 with total page 395 pages. Available in PDF, EPUB and Kindle. Book excerpt: A daily glass of wine prolongs life—yet alcohol can cause life-threatening cancer. Some say raising the minimum wage will decrease inequality while others say it increases unemployment. Scientists once confidently claimed that hormone replacement therapy reduced the risk of heart disease but now they equally confidently claim it raises that risk. What should we make of this endless barrage of conflicting claims? Observation and Experiment is an introduction to causal inference by one of the field’s leading scholars. An award-winning professor at Wharton, Paul Rosenbaum explains key concepts and methods through lively examples that make abstract principles accessible. He draws his examples from clinical medicine, economics, public health, epidemiology, clinical psychology, and psychiatry to explain how randomized control trials are conceived and designed, how they differ from observational studies, and what techniques are available to mitigate their bias. “Carefully and precisely written...reflecting superb statistical understanding, all communicated with the skill of a master teacher.” —Stephen M. Stigler, author of The Seven Pillars of Statistical Wisdom “An excellent introduction...Well-written and thoughtful...from one of causal inference’s noted experts.” —Journal of the American Statistical Association “Rosenbaum is a gifted expositor...an outstanding introduction to the topic for anyone who is interested in understanding the basic ideas and approaches to causal inference.” —Psychometrika “A very valuable contribution...Highly recommended.” —International Statistical Review

Download or read book Medications for Opioid Use Disorder Save Lives written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2019-06-16 with total page 175 pages. Available in PDF, EPUB and Kindle. Book excerpt: The opioid crisis in the United States has come about because of excessive use of these drugs for both legal and illicit purposes and unprecedented levels of consequent opioid use disorder (OUD). More than 2 million people in the United States are estimated to have OUD, which is caused by prolonged use of prescription opioids, heroin, or other illicit opioids. OUD is a life-threatening condition associated with a 20-fold greater risk of early death due to overdose, infectious diseases, trauma, and suicide. Mortality related to OUD continues to escalate as this public health crisis gathers momentum across the country, with opioid overdoses killing more than 47,000 people in 2017 in the United States. Efforts to date have made no real headway in stemming this crisis, in large part because tools that already existâ€"like evidence-based medicationsâ€"are not being deployed to maximum impact. To support the dissemination of accurate patient-focused information about treatments for addiction, and to help provide scientific solutions to the current opioid crisis, this report studies the evidence base on medication assisted treatment (MAT) for OUD. It examines available evidence on the range of parameters and circumstances in which MAT can be effectively delivered and identifies additional research needed.

Download or read book Drugs and the FDA written by Mikkael A. Sekeres and published by MIT Press. This book was released on 2024-02-06 with total page 319 pages. Available in PDF, EPUB and Kindle. Book excerpt: How the FDA was shaped by public health crises and patient advocacy, told against a background of the contentious hearings on the breast cancer drug Avastin. Food and Drug Administration approval for COVID-19 vaccines and the controversial Alzheimer’s drug Aduhelm made headlines, but few of us know much about how the agency does its work. Why is the FDA the ultimate US authority on a drug’s safety and efficacy? In Drugs and the FDA, Mikkael Sekeres—a leading oncologist and former chair of the FDA’s cancer drug advisory committee—tells the story of how the FDA became the most trusted regulatory agency in the world. It took a series of tragedies and health crises, as well as patient advocacy, for the government to take responsibility for ensuring the efficacy and safety of drugs and medical devices. Before the FDA existed, drug makers could hawk any potion, claim treatment of any ailment, and make any promise on a label. But then, throughout the twentieth century, the government was forced to take action when children were poisoned by contaminated diphtheria and smallpox vaccines, an early antibiotic contained antifreeze, a drug prescribed for morning sickness in pregnancy caused babies to be born disfigured, and access to AIDS drugs was limited to a few clinical trials while thousands died. Sekeres describes all these events against the backdrop of the contentious 2011 hearings on the breast cancer drug Avastin, in which he participated as a panel member. The Avastin hearings, he says, put to the test a century of the FDA’s evolution, demonstrating how its system of checks and balances works—or doesn’t work.

Download or read book Treating Drug Problems written by Committee for the Substance Abuse Coverage Study and published by National Academies Press. This book was released on 1992-01-01 with total page 332 pages. Available in PDF, EPUB and Kindle. Book excerpt: Treating Drug Problems, Volume 2 presents a wealth of incisive and accessible information on the issue of drug abuse and treatment in America. Several papers lay bare the relationship between drug treatment and other aspects of drug policy, including a powerful overview of twentieth century narcotics use in America and a unique account of how the federal government has built and managed the drug treatment system from the 1960s to the present. Two papers focus on the criminal justice system. The remaining papers focus on Employer policies and practices toward illegal drugs. Patterns and cycles of cocaine use in subcultures and the popular culture. Drug treatment from a marketing, supply-and-demand perspective, including an analysis of policy options. Treating Drug Problems, Volume 2 provides important information to policy makers and administrators, drug treatment specialists, and researchers.

Download or read book Improving and Accelerating Therapeutic Development for Nervous System Disorders written by Institute of Medicine and published by National Academies Press. This book was released on 2014-02-06 with total page 107 pages. Available in PDF, EPUB and Kindle. Book excerpt: Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.

Download or read book Physics Pharmacology and Physiology for Anaesthetists written by Matthew E. Cross and published by Cambridge University Press. This book was released on 2014-03-06 with total page 439 pages. Available in PDF, EPUB and Kindle. Book excerpt: A quick reference to basic science for anaesthetists, containing all the key information needed for FRCA exams.

Download or read book Drug Efficacy written by United States. Congress. House. Committee on Government Operations. Intergovernmental Relations Subcommittee and published by . This book was released on 1970 with total page 264 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Drug Efficacy Safety and Biologics Discovery written by Sean Ekins and published by John Wiley & Sons. This book was released on 2009-01-28 with total page 442 pages. Available in PDF, EPUB and Kindle. Book excerpt: Drug Efficacy, Safety, and Biologics Discovery: Emerging Technologies and Tools covers key emerging technologies in pharmaceutical R & D and how they have substantially impacted (or are currently impacting) drug discovery. The cross-disciplinary collaborations implicit in integrating these technologies with drug discovery operations will fuel the engine for future innovations. This book cuts across the multiple areas of drug discovery, each chapter authored by pioneers in that field, making for a broad appeal to the chemical and biological scientists and technologists involved in drug discovery and development.

Download or read book The Role of NIH in Drug Development Innovation and Its Impact on Patient Access written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-01-27 with total page 103 pages. Available in PDF, EPUB and Kindle. Book excerpt: To explore the role of the National Institutes of Health (NIH) in innovative drug development and its impact on patient access, the Board on Health Care Services and the Board on Health Sciences Policy of the National Academies jointly hosted a public workshop on July 24â€"25, 2019, in Washington, DC. Workshop speakers and participants discussed the ways in which federal investments in biomedical research are translated into innovative therapies and considered approaches to ensure that the public has affordable access to the resulting new drugs. This publication summarizes the presentations and discussions from the workshop.

Download or read book Basic Principles of Drug Discovery and Development written by Benjamin E. Blass and published by Academic Press. This book was released on 2021-03-30 with total page 712 pages. Available in PDF, EPUB and Kindle. Book excerpt: Basic Principles of Drug Discovery and Development presents the multifaceted process of identifying a new drug in the modern era, which requires a multidisciplinary team approach with input from medicinal chemists, biologists, pharmacologists, drug metabolism experts, toxicologists, clinicians, and a host of experts from numerous additional fields. Enabling technologies such as high throughput screening, structure-based drug design, molecular modeling, pharmaceutical profiling, and translational medicine are critical to the successful development of marketable therapeutics. Given the wide range of disciplines and techniques that are required for cutting edge drug discovery and development, a scientist must master their own fields as well as have a fundamental understanding of their collaborator’s fields. This book bridges the knowledge gaps that invariably lead to communication issues in a new scientist’s early career, providing a fundamental understanding of the various techniques and disciplines required for the multifaceted endeavor of drug research and development. It provides students, new industrial scientists, and academics with a basic understanding of the drug discovery and development process. The fully updated text provides an excellent overview of the process and includes chapters on important drug targets by class, in vitro screening methods, medicinal chemistry strategies in drug design, principles of in vivo pharmacokinetics and pharmacodynamics, animal models of disease states, clinical trial basics, and selected business aspects of the drug discovery process. Provides a clear explanation of how the pharmaceutical industry works, as well as the complete drug discovery and development process, from obtaining a lead, to testing the bioactivity, to producing the drug, and protecting the intellectual property Includes a new chapter on the discovery and development of biologics (antibodies proteins, antibody/receptor complexes, antibody drug conjugates), a growing and important area of the pharmaceutical industry landscape Features a new section on formulations, including a discussion of IV formulations suitable for human clinical trials, as well as the application of nanotechnology and the use of transdermal patch technology for drug delivery Updated chapter with new case studies includes additional modern examples of drug discovery through high through-put screening, fragment-based drug design, and computational chemistry

Download or read book Drug Efficacy written by United States. Congress. House. Committee on Government Operations. Intergovernmental Relations Subcommittee and published by . This book was released on 1970 with total page 442 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Oral Drug Absorption written by Jennifer B. Dressman and published by CRC Press. This book was released on 2016-04-19 with total page 432 pages. Available in PDF, EPUB and Kindle. Book excerpt: Oral Drug Absorption, Second Edition thoroughly examines the special equipment and methods used to test whether drugs are released adequately when administered orally. The contributors discuss methods for accurately establishing and validating in vitro/in vivo correlations for both MR and IR formulations, as well as alternative approaches for MR an

Download or read book Bottle of Lies written by Katherine Eban and published by HarperCollins. This book was released on 2020-06-23 with total page 512 pages. Available in PDF, EPUB and Kindle. Book excerpt: A NEW YORK TIMES BESTSELLER New York Times 100 Notable Books of 2019 New York Public Library Best Books of 2019 Kirkus Reviews Best Health and Science Books of 2019 Science Friday Best Books of 2019 New postscript by the author From an award-winning journalist, an explosive narrative investigation of the generic drug boom that reveals fraud and life-threatening dangers on a global scale—The Jungle for pharmaceuticals Many have hailed the widespread use of generic drugs as one of the most important public-health developments of the twenty-first century. Today, almost 90 percent of our pharmaceutical market is comprised of generics, the majority of which are manufactured overseas. We have been reassured by our doctors, our pharmacists and our regulators that generic drugs are identical to their brand-name counterparts, just less expensive. But is this really true? Katherine Eban’s Bottle of Lies exposes the deceit behind generic-drug manufacturing—and the attendant risks for global health. Drawing on exclusive accounts from whistleblowers and regulators, as well as thousands of pages of confidential FDA documents, Eban reveals an industry where fraud is rampant, companies routinely falsify data, and executives circumvent almost every principle of safe manufacturing to minimize cost and maximize profit, confident in their ability to fool inspectors. Meanwhile, patients unwittingly consume medicine with unpredictable and dangerous effects. The story of generic drugs is truly global. It connects middle America to China, India, sub-Saharan Africa and Brazil, and represents the ultimate litmus test of globalization: what are the risks of moving drug manufacturing offshore, and are they worth the savings? A decade-long investigation with international sweep, high-stakes brinkmanship and big money at its core, Bottle of Lies reveals how the world’s greatest public-health innovation has become one of its most astonishing swindles.

Download or read book Drug Transporters written by Martin F. Fromm and published by Springer Science & Business Media. This book was released on 2010-11-19 with total page 457 pages. Available in PDF, EPUB and Kindle. Book excerpt: It is increasingly recognized that various transporter proteins are expressed throughout the body and determine absorption, tissue distribution, biliary and renal elimination of endogenous compounds and drugs and drug effects. This book will give an overview on the transporter families which are most important for drug therapy. Most chapters will focus on one transporter family highlighting tissue expression, substrates, inhibitors, knock-out mouse models and clinical studies.

Download or read book Female Sexual Function and Dysfunction written by Elisabetta Costantini and published by Springer. This book was released on 2017-06-19 with total page 209 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book discusses all aspects of sexuality in women and in particular explores sexual function and dysfunction in a variety of settings, including the different stages of life and a wide range of major diseases and local conditions. The aim is to refocus attention on the needs and sexual realities of women, providing a fresh point of view that will assist gynecologists, sexual medicine physicians, and urologists in delivery of high-quality care and help women themselves to understand and address sexual problems relating to desire, arousal, orgasm, and sexual pain. Psychological aspects of female sexuality and the impacts of the aging process, pregnancy, and childbirth are carefully examined. Extensive consideration is then given to the effects on sexual function of such conditions as cardiovascular disease, cancer, diabetes, neurological disease, endometriosis, pelvic organ prolapse, urinary incontinence, reproductive disorders, sexual abuse, and drug abuse. Issues of sexual identity and female dysmorphophobias are also considered. The authors are all experts in the field and have a deep understanding of the complexities of female sexuality.