Considering the Patient in Pediatric Drug Development

Considering the Patient in Pediatric Drug Development: How Good Intentions Turned into Harm addresses a fundamental challenge in drug development and healthcare for young patients. In clinical trials and clinical practice, the term "children" is used ambiguously to confer physiological characteristics to a chronological age limit, which in reality does not exist. This book outlines why the United States (US) and European Union's (EU) regulatory authorities, pediatric academia, and the pharmaceutical industry demand, support and perform pediatric drug studies, along with the key flaws of this demand that blurs the different administrative and physiological meanings of the term "child." In addition, the book covers why most pediatric regulatory studies lack medical sense and many even harm young patients and the conflicts of interest behind pediatric drug studies. It includes relevant information about the maturation of the human body regarding absorption, distribution, metabolism and excretion of food and drugs as well as key differences between newborns, infants, older children and adolescents. Explains relevant information about the maturation of the human body regarding absorption, distribution, metabolism and excretion of food and drugs, including key differences between newborns, infants, older children and adolescents Discusses historical roots of separate drug approval in officially labeled "children" and conflicts of interest in performing and publishing "pediatric" research Helps to decipher justifications for pediatric studies to help people navigate the relevance of the information

Pediatric Drug Development, Second Edition, encompasses the new regulatory initiatives across EU, US and ROW designed to encourage improved access to safe and effective medicines for children. It includes new developments in biomarkers and surrogate endpoints, developmental pharmacology and other novel aspects of pediatric drug development.

Pediatric Drug Development, Second Edition, encompasses the new regulatory initiatives across EU, US and ROW designed to encourage improved access to safe and effective medicines for children. It includes new developments in biomarkers and surrogate endpoints, developmental pharmacology and other novel aspects of pediatric drug development.

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.

Clinical Research in Paediatric Psychopharmacology: An Overview of the Ethical, Scientific and Regulatory Aspects provides a practical guide and overview of the ethical, scientific and regulatory aspects of clinical research in pediatric psychopharmacology, also discussing practical points to consider when developing clinical research in this field. The book is ideal for professionals involved in clinical research in pediatric psychopharmacology, i.e., including, but not limited to pediatricians, health care professionals, researchers, investigators, pharmaceutical company personals and potentially ethics committee members. Topics discussed include the role of patient organization and advocacy groups in research, the role of families and patients: ‘should I involve my kid in clinical research, and historical, ethical, regulatory, clinical, scientific, intercultural and practical aspects of clinical research in child and adolescent psychopharmacology. Covers both theoretical and practical aspects of clinical research in paediatric psychopharmacology Approaches the topic from different angles from the regulatory framework to the patient perspective Discusses ethical and safety considerations for research in paediatric psychopharmacology Offers future perspective for paediatric development

This concise book is addressed to researchers, clinical investigators, as well as practicing physicians and surgeons who are interested in the fields of clinical research and trials. It covers some important topics related to clinical trials including an introduction to clinical trials, some aspects concerning clinical trials in pediatric age group, and the unique aspects of the design of clinical trials on stem cell therapy.

Aimed at those already involved in drug development or those considering entering the field, Clinical Drug Trials and Tribulations, Second Edition comprehensibly addresses the new, day-to-day challenges of drug development with valuable assessments of the areas affecting the conduction of nonclinical and clinical studies. Addressing which decisions should be made during drug development, this updated and expanded text/reference carefully guides readers through the various trials and tribulations that emerge phase-by-phase and are pertinent to all levels of pharmaceutical or clinical drug management. Bringing together the latest information on drug development, the Second Edition contains: new material on... international regulation and deregulation venture capitalist investment the IND process informed consent changes in manufacturing and updated and extended coverage of... pediatric drug trial design the advantages and disadvantages of orphan drug designations the maximization of package inserts for marketing post approval safety surveillance withdrawals from the drug market Clinical Drug Trials and Tribulations, Second Edition will prove an invaluable reference for pharmacologists, pharmacists, clinical chemists, clinical coordinators, clinical monitors, government drug regulatory personnel, and bioethicists as well as a useful text for medical or pharmacy school courses on pharmaceutical development and research.

Focused on pediatric physiology, pharmacology, pharmacokinetics and pharmacodynamics, this book illustrates the differences between the pediatric population and adults; knowledge of extreme importance not only during pediatric drug development but also in the clinical practice. Physicians, nurses, clinical pharmacologists, researchers and healthcare professionals will find this an invaluable resource. With the advent of pediatric exclusivity, and requirements to conduct clinical studies in children, an emphasis has been placed on finding a safe and efficacious dose of a drug in children. Children are not ‘small adults’, and drug dosing in this population requires special consideration. There are subtle physiological and biochemical differences among neonates, infants, children, adolescents and adults and dosing in pediatrics requires proper understanding of these factors. Furthermore, dosing in children, as in adults, should be based on pharmacokinetic and pharmacodynamic data. This is an evolving area, as pediatric pharmacokinetic studies are becoming mandatory for getting approval of new drugs in this population.

It is very important for scientists all over the globe to enhance drug discovery research for better human health. This book demonstrates that various expertise are essential for drug discovery including synthetic or natural drugs, clinical pharmacology, receptor identification, drug metabolism, pharmacodynamic and pharmacokinetic research. The following 5 sections cover diverse chapter topics in drug discovery: Natural Products as Sources of Leading Molecules in Drug Discovery; Oncology and Drug Discovery; Receptors Involvement in Drug Discovery; Management and Development of Drugs against Infectious Diseases; Advanced Methodology.

In recent decades, advances in biomedical research have helped save or lengthen the lives of children around the world. With improved therapies, child and adolescent mortality rates have decreased significantly in the last half century. Despite these advances, pediatricians and others argue that children have not shared equally with adults in biomedical advances. Even though we want children to benefit from the dramatic and accelerating rate of progress in medical care that has been fueled by scientific research, we do not want to place children at risk of being harmed by participating in clinical studies. Ethical Conduct of Clinical Research Involving Children considers the necessities and challenges of this type of research and reviews the ethical and legal standards for conducting it. It also considers problems with the interpretation and application of these standards and conduct, concluding that while children should not be excluded from potentially beneficial clinical studies, some research that is ethically permissible for adults is not acceptable for children, who usually do not have the legal capacity or maturity to make informed decisions about research participation. The book looks at the need for appropriate pediatric expertise at all stages of the design, review, and conduct of a research project to effectively implement policies to protect children. It argues persuasively that a robust system for protecting human research participants in general is a necessary foundation for protecting child research participants in particular.

Every year, hundreds of thousands of healthy volunteers and patients worldwide undertake the journey through the maze that can be clinical trials. Research participants take part in clinical trials for a variety of reasons. The healthy volunteers may be seeking extra money to pay off college tuition, or they may know someone who is suffering and would potentially benefit from the results of the trial. The patient who is terminally ill might participate in a clinical trial simply as a last hope for a cure. Whatever the goals, though, most participants will experience the same sense of bewilderment as they encounter the jargon and medical terminology that they will hear and have to read about and understand during the course of the clinical trial. Clinical Trials: What Patients and Volunteers Need to Know demystifies the entire process, focusing on the process of drug development, and the clinical trial itself. Writing from a lifetime of experience, the author provides important questions to ask those running a clinical trial, key definitions and terms for a participant to know and understand, as well as anecdotes illustrating the clinical trial process. The author also grapples with the idea of "informed consent," providing mechanisms for patients and volunteers to feel fully informed before signing up for the trial. A vital resource for those who are considering enrolling in a clinical trial, or for the parents, friends, or relatives of those involved in a clinical trial, this book takes away the mystery and allows the participant to enter a clinical trial feeling both informed and confident.

The very rapid pace of advances in biomedical research promises us a wide range of new drugs, medical devices, and clinical procedures. The extent to which these discoveries will benefit the public, however, depends in large part on the methods we choose for developing and testing them. Modern Methods of Clinical Investigation focuses on strategies for clinical evaluation and their role in uncovering the actual benefits and risks of medical innovation. Essays explore differences in our current systems for evaluating drugs, medical devices, and clinical procedures; health insurance databases as a tool for assessing treatment outcomes; the role of the medical profession, the Food and Drug Administration, and industry in stimulating the use of evaluative methods; and more. This book will be of special interest to policymakers, regulators, executives in the medical industry, clinical researchers, and physicians.

The objective of this volume is to give an overview of the present state of the art of pediatric clinical pharmacology including developmental physiology, pediatric-specific pathology, special tools and methods for development of drugs for children (assessment of efficacy, toxicity, long-term safety etc.) as well as regulatory and ethical knowledge and skills. In the future, structural and educational changes have to lead back to a closer cooperation and interaction of pediatrics with (clinical) pharmacology and pharmacy.

Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

Prescribing for children is a particularly challenging discipline due to specific issues of drug absorption, metabolism, distribution and excretion. The aim of this book is to improve understanding in all aspects of paediatric prescribing, from the development of suitable drugs through to their practical administration. With its origins in the EU-funded Global Research in Paediatrics (GRiP) project this is the first truly international textbook to provide guidance on the principles behind optimal neonatal and paediatric prescribing. Harnessing the international expertise of paediatricians and pharmacists in the field, Prescribing Medicines for Children compliments the British National Formulary for Children (BNFC), facilitating translation of essential pharmacological principles into good prescribing practice. It incorporates specific information on how to promote safe and effective prescribing in paediatrics, including how to avoid medication errors and adverse drug reactions in children. Highlights include the differences in prescribing habits between countries and the shared principles that underpin rational prescribing in paediatrics and neonatology. The book is divided into two sections: Section A provides concise educational material relating to paediatric pharmacology and optimising how medicines are developed and prescribed for children. Section B considers key clinical prescribing areas and can be used as a quick reference guide. Each chapter is focused on the key issues in prescribing for a respective clinical specialty or context. Prescribing Medicines for Children is essential reading for all those who are involved in prescribing medicines to neonates and children. This includes undergraduate and postgraduate pharmacists, nurses, paediatricians and primary care physicians, academic scientists, and those working in the pharmaceutical industry and drug regulation.

#1 NEW YORK TIMES BESTSELLER • “The story of modern medicine and bioethics—and, indeed, race relations—is refracted beautifully, and movingly.”—Entertainment Weekly NOW A MAJOR MOTION PICTURE FROM HBO® STARRING OPRAH WINFREY AND ROSE BYRNE • ONE OF THE “MOST INFLUENTIAL” (CNN), “DEFINING” (LITHUB), AND “BEST” (THE PHILADELPHIA INQUIRER) BOOKS OF THE DECADE • ONE OF ESSENCE’S 50 MOST IMPACTFUL BLACK BOOKS OF THE PAST 50 YEARS • WINNER OF THE CHICAGO TRIBUNE HEARTLAND PRIZE FOR NONFICTION NAMED ONE OF THE BEST BOOKS OF THE YEAR BY The New York Times Book Review • Entertainment Weekly • O: The Oprah Magazine • NPR • Financial Times • New York • Independent (U.K.) • Times (U.K.) • Publishers Weekly • Library Journal • Kirkus Reviews • Booklist • Globe and Mail Her name was Henrietta Lacks, but scientists know her as HeLa. She was a poor Southern tobacco farmer who worked the same land as her slave ancestors, yet her cells—taken without her knowledge—became one of the most important tools in medicine: The first “immortal” human cells grown in culture, which are still alive today, though she has been dead for more than sixty years. HeLa cells were vital for developing the polio vaccine; uncovered secrets of cancer, viruses, and the atom bomb’s effects; helped lead to important advances like in vitro fertilization, cloning, and gene mapping; and have been bought and sold by the billions. Yet Henrietta Lacks remains virtually unknown, buried in an unmarked grave. Henrietta’s family did not learn of her “immortality” until more than twenty years after her death, when scientists investigating HeLa began using her husband and children in research without informed consent. And though the cells had launched a multimillion-dollar industry that sells human biological materials, her family never saw any of the profits. As Rebecca Skloot so brilliantly shows, the story of the Lacks family—past and present—is inextricably connected to the dark history of experimentation on African Americans, the birth of bioethics, and the legal battles over whether we control the stuff we are made of. Over the decade it took to uncover this story, Rebecca became enmeshed in the lives of the Lacks family—especially Henrietta’s daughter Deborah. Deborah was consumed with questions: Had scientists cloned her mother? Had they killed her to harvest her cells? And if her mother was so important to medicine, why couldn’t her children afford health insurance? Intimate in feeling, astonishing in scope, and impossible to put down, The Immortal Life of Henrietta Lacks captures the beauty and drama of scientific discovery, as well as its human consequences.

The book is not a classic pediatric text book. It shows different approaches to some pediatric topics. Our aim in this book, as understood from its title, is to describe some specific issues related to nursing, psychiatric and surgical issues. The book Pediatric Nursing, Psychiatric and Surgical Issues has 8 chapters which are placed in 4 different sections, to enlighten patients and pediatricians on current developments on specific pediatric issues.

Cardiotoxicity may be caused by radiotherapy and/or anticancer agents for many malignancies, adverse effects of some drugs in the context of medical intervention or heavy metal intake, especially during the anticancer therapy. This book intends to bring forward the recent development in toxicities from cancer treatment. It updates the possible mechanisms of cardiotoxicities of some anticancer agents and the suggested prevention and treatment strategies. This book contains many valuable contributions from the researchers in oncology and cardiology as well as the clinicians who are experts in this field.

Social and behavioral science has for decades studied and recognized leadership as a social exchange between leaders and followers. But leadership is rather complex, and as such, it tends to lead to an increased interest within and across different disciplines. This book is an attempt to provide theoretical and empirical framework to better understand leadership challenges in various contexts. The authors cover an array of themes that span from an individual level to an organizational and societal level. In this volume, two sections are presented. The first section based on individual level focuses on different leadership styles and abilities, and the other section provides theories to understand leadership in public administration, in industrial settings and in nonprofit organizations.