Download or read book Treating Rare and Neglected Pediatric Diseases Promoting the Development of New Treatments and Cures written by Health, Education, Labor, and Pensions United States Senate Committee and published by . This book was released on 2013-04-28 with total page 134 pages. Available in PDF, EPUB and Kindle. Book excerpt: We meet today to discuss a profoundly important issue: the lack of effective treatments for rare and neglected diseases. Over the years, Congress has devoted extraordinary sums for research into major diseases that afflict millions of Americans. But, we've been less generous, and less successful, in mobilizing the research community to come up with therapies and cures for rare and neglected diseases. In the United States, rare diseases are defined as those that affect fewer than 200,000 people. According to the National Institutes of Health, there are nearly 7,000 rare diseases, affecting more than 25 million Americans. Yet, there are FDA-approved treatments for only as few as 200 of these diseases.

Download or read book Treating Rare and Neglected Pediatric Diseases written by United States. Congress and published by Createspace Independent Publishing Platform. This book was released on 2017-12-11 with total page 134 pages. Available in PDF, EPUB and Kindle. Book excerpt: Treating rare and neglected pediatric diseases : promoting the development of new treatments and cures : hearing of the Committee on Health, Education, Labor, and Pensions, United States Senate, One Hundred Eleventh Congress, second session ... July 21, 2010.

Download or read book Treating Rare and Neglected Pediatric Diseases Promoting the Development of New Treatments and Cures 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 130 pages. Available in PDF, EPUB and Kindle. Book excerpt:

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 130 pages. Available in PDF, EPUB and Kindle. Book excerpt:

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 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 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 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 Legislative Calendar written by United States. Congress. Senate. Committee on Health, Education, Labor, and Pensions and published by . This book was released on with total page 172 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book The Oxford Handbook of Intellectual Property Law written by Rochelle C. Dreyfuss and published by Oxford University Press. This book was released on 2018-04-12 with total page 1145 pages. Available in PDF, EPUB and Kindle. Book excerpt: We live in an age in which expressive, informational, and technological subject matter are becoming increasingly important. Intellectual property is the primary means by which the law seeks to regulate such subject matter. It aims to promote innovation and creativity, and in doing so to support solutions to global environmental and health problems, as well as freedom of expression and democracy. It also seeks to stimulate economic growth and competition, accounting for its centrality to EU Internal Market and international trade and development policies. Additionally, it is of enormous and increasing importance to business. As a result there is a substantial and ever-growing interest in intellectual property law across all spheres of industry and social policy, including an interest in its legal principles, its social and normative foundations, and its place and operation in the political economy. This handbook written by leading academics and practitioners from the field of intellectual property law, and suitable for both a specialist legal readership and an intelligent but non-specialist legal and non-legal readership, provides a comprehensive account of the following areas: - The foundations of IP law, including its emergence and development in different jurisdictions and regions; - The substantive rules and principles of IP; and - Important issues arising from the existence and operation of IP in the political economy.

Download or read book Congressional Record written by United States. Congress and published by . This book was released on 2010 with total page 172 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Congressional Record Daily Digest of the Congress written by United States. Congress and published by . This book was released on 2010 with total page 664 pages. Available in PDF, EPUB and Kindle. Book excerpt:

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 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 Food and Drug Administration s Review Process for Products to Treat Rare Diseases and Neglected Tropical Diseases written by United States. Congress. Senate. Committee on Appropriations. Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies and published by . This book was released on 2011 with total page 64 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Pandemic Planning written by J. Eric Dietz and published by CRC Press. This book was released on 2012-03-14 with total page 333 pages. Available in PDF, EPUB and Kindle. Book excerpt: Preparedness and rigorous planning on community, state, and regional levels are critical to containing the threat of pandemic illness. Steeped in research and recommendations from lessons learned, Pandemic Planning describes the processes necessary for the efficient and effective preparation, prevention, response, and recovery from a pandemic threa

Download or read book Investing to Overcome the Global Impact of Neglected Tropical Diseases written by World Health Organization and published by World Health Organization. This book was released on 2015-08-05 with total page 211 pages. Available in PDF, EPUB and Kindle. Book excerpt: "The presence, or absence, of neglected tropical diseases (NTDs) can be seen as a proxy for poverty and for the success of interventions aimed at reducing poverty. Today, coverage of the public-health interventions recommended by the World Health Organization (WHO) against NTDs may be interpreted as a proxy for universal health coverage and shared prosperity - in short, a proxy for coverage against neglect. As the world's focus shifts from development to sustainable development, from poverty eradication to shared prosperity, and from disease-specific goals to universal health coverage, control of NTDs will assume an important role towards the target of achieving universal health coverage, including individual financial risk protection. Success in overcoming NTDs is a "litmus test" for universal health coverage against NTDs in endemic countries. The first WHO report on NTDs (2010) set the scene by presenting the evidence for how these interventions had produced results. The second report (2013) assessed the progress made in deploying them and detailed the obstacles to their implementation. This third report analyses for the first time the investments needed to achieve the scale up of implementation required to achieve the targets of the WHO Roadmap on NTDs and universal coverage against NTDs. INVESTING TO OVERCOME THE GLOBAL IMPACT OF NEGLECTED TROPICAL DISEASES presents an investment strategy for NTDs and analyses the specific investment case for prevention, control, elimination and eradication of 12 of the 17 NTDs. Such an analysis is justified following the adoption by the Sixty-sixth World Health Assembly in 2013 of resolution WHA6612 on neglected tropical diseases, which called for sufficient and predictable funding to achieve the Roadmap's targets and sustain control efforts. The report cautions, however, that it is wise investment and not investment alone that will yield success. The report registers progress and challenges and signals those that lie ahead. Climate change is expected to increase the spread of several vector-borne NTDs, notably dengue, transmission of which is directly influenced by temperature, rainfall, relative humidity and climate variability primarily through their effects on the vector. Investments in vector-borne diseases will avoid the potentially catastrophic expenditures associated with their control. The presence of NTDs will thereby signal an early warning system for climate-sensitive diseases. The ultimate goal is to deliver enhanced and equitable interventions to the most marginalized populations in the context of a changing public-health and investment landscape to ensure that all peoples affected by NTDs have an opportunity to lead healthier and wealthier lives."--Publisher's description.