Download or read book Rare Disease Drug Development written by Raymond A. Huml and published by Springer Nature. This book was released on 2021-11-08 with total page 418 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Drug Development and Rare Pediatric Diseases written by Frederick Johnston and published by Nova Biomedical Books. This book was released on 2021-08 with total page 122 pages. Available in PDF, EPUB and Kindle. Book excerpt: Over the past few decades, a number of legislative actions have been taken to encourage the development of therapies to treat people affected by rare diseases and to encourage therapies for pediatric patients. During this same time period, the Food and Drug Administration (FDA) has invested substantial effort in support of these same goals. As described in this book, recent legislative actions have prompted new initiatives. Continuing the response to the laws' specific requirements and FDA's commitment under PDUFA, this book includes input from the public and FDA and discusses the many complex issues involved in medical product development for people with rare diseases. Following a brief overview of both legislative and FDA efforts over the years to foster development of therapies for these patients, the book summarizes the three days of the public meeting and presents FDA's strategic plan for accelerating the development of the urgently needed therapies for pediatric rare diseases.

Download or read book Drug Development for Rare Diseases written by Bo Yang and published by CRC Press. This book was released on 2023-01-10 with total page 233 pages. Available in PDF, EPUB and Kindle. Book excerpt: A disease is defined as rare if the prevalence is fewer than 200,000 in the United States. It is estimated that there are more than 7,000 rare diseases, which collectively affect 30 million Americans or 10% of the US population. This diverse and complex disease area poses challenges for patients, caregivers, regulators, drug developers, and other stakeholders. This book is proposed to give an overview of the common issues facing rare disease drug developers, summarize challenges specific to clinical development in small populations, discuss drug development strategies in the evolving regulatory environment, explain generation and utilization of different data and evidence inside and beyond clinical trials, and use recent examples to demonstrate these challenges and the development strategies that respond to the challenges. Key Features: • Rare disease. • Drug development. • Innovative clinical trial design. • Regulatory approval. • Real-world evidence.

Download or read book Rare Diseases and Orphan Products written by Institute of Medicine and published by National Academies Press. This book was released on 2011-04-03 with total page 442 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Pediatric Drug Development written by Andrew E. Mulberg and published by John Wiley & Sons. This book was released on 2013-05-20 with total page 782 pages. Available in PDF, EPUB and Kindle. Book excerpt: Most medicines have never been adequately tested for safety and efficacy in pediatric populations and preterm, infants and children are particularly vulnerable to adverse drug reactions. Pediatric Drug Development: Concepts and Applications, Second Edition, addresses the unique challenges in conducting effective drug research and development in pediatric populations. This new edition covers the legal and ethical issues of consent and assent, the additional legal and safety protections for children, and the appropriate methods of surveillance and assessment for children of varying ages and maturity, particularly for patient reported outcomes. It includes new developments in biomarkers and surrogate endpoints, developmental pharmacology and other novel aspects of global pediatric drug development. It also encompasses the new regulatory initiatives across EU, US and ROW designed to encourage improved access to safe and effective medicines for children globally. From an international team of expert contributors Pediatric Drug Development: Concepts and Applications is the practical guide to all aspects of the research and development of safe and effective medicines for children.

Download or read book Orphan Drugs and Rare Diseases written by David C Pryde and published by Royal Society of Chemistry. This book was released on 2014 with total page 488 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides an up-to-date monograph on the drug discovery and regulatory elements of therapuetics used to treat rare or "orphan" diseases.

Download or read book Principles and Practice of Clinical Trials written by Steven Piantadosi and published by Springer Nature. This book was released on 2022-07-19 with total page 2573 pages. Available in PDF, EPUB and Kindle. Book excerpt: This is a comprehensive major reference work for our SpringerReference program covering clinical trials. Although the core of the Work will focus on the design, analysis, and interpretation of scientific data from clinical trials, a broad spectrum of clinical trial application areas will be covered in detail. This is an important time to develop such a Work, as drug safety and efficacy emphasizes the Clinical Trials process. Because of an immense and growing international disease burden, pharmaceutical and biotechnology companies continue to develop new drugs. Clinical trials have also become extremely globalized in the past 15 years, with over 225,000 international trials ongoing at this point in time. Principles in Practice of Clinical Trials is truly an interdisciplinary that will be divided into the following areas: 1) Clinical Trials Basic Perspectives 2) Regulation and Oversight 3) Basic Trial Designs 4) Advanced Trial Designs 5) Analysis 6) Trial Publication 7) Topics Related Specific Populations and Legal Aspects of Clinical Trials The Work is designed to be comprised of 175 chapters and approximately 2500 pages. The Work will be oriented like many of our SpringerReference Handbooks, presenting detailed and comprehensive expository chapters on broad subjects. The Editors are major figures in the field of clinical trials, and both have written textbooks on the topic. There will also be a slate of 7-8 renowned associate editors that will edit individual sections of the Reference.

Download or read book Addressing the Barriers to Pediatric Drug Development written by Institute of Medicine and published by National Academies Press. This book was released on 2008-08-12 with total page 64 pages. Available in PDF, EPUB and Kindle. Book excerpt: Decades of research have demonstrated that children do not respond to medications in the same way as adults. Differences between children and adults in the overall response to medications are due to profound anatomical, physiological, and developmental differences. Although few would argue that children should receive medications that have not been adequately tested for safety and efficacy, the majority of drugs prescribed for children-50 to 75 percent-have not been tested in pediatric populations. Without adequate data from such testing, prescribing drugs appropriately becomes challenging for clinicians treating children, from infancy through adolescence. Addressing the Barriers to Pediatric Drug Development is the summary of a workshop, held in Washington, D.C. on June 13, 2006, that was organized to identify barriers to the development and testing of drugs for pediatric populations, as well as ways in which the system can be improved to facilitate better treatments for children.

Download or read book Pediatric Drug Development written by Andrew E. Mulberg and published by John Wiley & Sons. This book was released on 2011-09-20 with total page 843 pages. Available in PDF, EPUB and Kindle. Book excerpt: Pediatric Drug Development: Concepts and Applications is designed as a reference and textbook and is meant to address the science of differences between the pediatric and adult subject in the development of pharmaceutical products. Considered are the ethics and medical needs of proper understanding the pediatric and adult differences, the business case for proper development of drugs for children, as well as the technical feasibility studies and processes that are necessary for a proper pediatric drug development program. The applications of these approaches will benefit all stakeholders and ultimately not only educate but also provide better and safer drugs for pediatric patients.

Download or read book Breakthrough Business Models written by Institute of Medicine and published by National Academies Press. This book was released on 2009-02-17 with total page 151 pages. Available in PDF, EPUB and Kindle. Book excerpt: The process for developing new drug and biologic products is extraordinarily expensive and time-consuming. Although large pharmaceutical companies may be able to afford the cost of development because they can expect a large return on investment, organizations developing drugs to treat rare and neglected diseases are unable to rely on such returns. On June 23, 2008, the Institute of Medicine's Forum on Drug Discovery, Development, and Translation held a public workshop, "Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies," which sought to explore new and innovative strategies for developing drugs for rare and neglected diseases.

Download or read book Treating Rare and Neglected Pediatric Diseases written by United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions and published by . This book was released on 2012 with total page 144 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Registries for Evaluating Patient Outcomes written by Agency for Healthcare Research and Quality/AHRQ and published by Government Printing Office. This book was released on 2014-04-01 with total page 396 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Rare Diseases written by United States Government Accountability Office and published by Createspace Independent Publishing Platform. This book was released on 2017-09-23 with total page 32 pages. Available in PDF, EPUB and Kindle. Book excerpt: Almost 7,000 rare diseases, most of which are serious or life-threatening, affect more than 25 million Americans. About half of all rare diseases affect children, and few of these diseases have viable treatments. To encourage the development of drugs to treat or prevent rare pediatric diseases, the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012 authorized FDA to award a priority review voucher to a drug sponsor upon approval of that sponsor's drug to treat a rare pediatric disease. A drug sponsor can later redeem the voucher when submitting another new drug application to treat any disease or condition in adults or children, or sell or transfer the voucher to another sponsor. A voucher entitles a sponsor to a 6-month priority review by FDA rather than the 10-month standard review. FDASIA included a provision for GAO to study the pediatric voucher program. GAO examined what is known about the effectiveness of the program in encouraging the development of drugs to prevent or treat certain rare pediatric diseases. GAO reviewed relevant laws and documentation related to the program and its management, and identified drug sponsors who were awarded vouchers, the diseases their drugs were approved to treat, and whether the vouchers were redeemed, sold, or transferred. GAO also interviewed FDA officials, drug sponsors, patient advocacy groups

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 Drug Discovery and Development written by Vishwanath Gaitonde and published by BoD – Books on Demand. This book was released on 2020-03-11 with total page 166 pages. Available in PDF, EPUB and Kindle. Book excerpt: The process of drug discovery and development is a complex multistage logistics project spanned over 10-15 years with an average budget exceeding 1 billion USD. Starting with target identification and synthesizing anywhere between 10k to 15k synthetic compounds to potentially obtain the final drug that reaches the market involves a complicated maze with multiple inter- and intra-operative fields. Topics described in this book emphasize the progresses in computational applications, pharmacokinetics advances, and molecular modeling developments. In addition the book also contains special topics describing target deorphaning in Mycobacterium tuberculosis, therapy treatment of some rare diseases, and developments in the pediatric drug discovery process.

Download or read book Rare Diseases written by Marcia Crosse and published by . This book was released on 2016-05-04 with total page 32 pages. Available in PDF, EPUB and Kindle. Book excerpt: Almost 7,999 rare diseases, most of which are serious or life-threatening, affect more than 25 million Americans. About half of all rare diseases affect children, and few of these diseases have viable treatments. To encourage the development of drugs to treat or prevent rare pediatric diseases, the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012 authorized FDA to award a priority review voucher to a drug sponsor upon approval of that sponsor's drug to treat a rare pediatric disease. A drug sponsor can later redeem the voucher when submitting another new drug application to treat any disease or condition in adults or children, or sell or transfer the voucher to another sponsor. A voucher entitles a sponsor to a 6-month priority review by FDA rather than the 10-month standard review. This report examined what is known abut the effectiveness of the pediatric voucher program. Table and figure. This is a print on demand report.

Download or read book The Rare Disease Gazette 20 Special Edition written by Robert M. Califf and published by Primento Digital sprl. This book was released on 2024-04-23 with total page 17 pages. Available in PDF, EPUB and Kindle. Book excerpt: The journal Science (American Association for the Advancement of Science) in collaboration with Fondation Ipsen delivers international science webinars for the general public. In 2023, these webinars focused on advocacy in rare diseases. "The Rare Disease Gazette" is a magazine that broadcasts these discussions. In this special edition, Erika Berg, PhD, hosts a Fireside Chat about the intersection between the FDA and rare disease, with Robert M. Califf, Commissioner of Food and Drugs at the United States Food and Drug Administration (FDA). ​ !--StartFragment--ABOUT THE AUTHOR!--EndFragment-- !--StartFragment--Robert M. Califf, M.D., Commissioner of Food and Drugs; Head of the U.S. Food and Drug Administration !--EndFragment--