New Therapeutic Options For Rare Diseases
DOWNLOAD
FREE 30 Days
Download New Therapeutic Options For Rare Diseases PDF/ePub or read online books in Mobi eBooks. Click Download or Read Online button to get New Therapeutic Options For Rare Diseases book now. This website allows unlimited access to, at the time of writing, more than 1.5 million titles, including hundreds of thousands of titles in various foreign languages. If the content not found or just blank you must refresh this page
New Therapeutic Options For Rare Diseases
DOWNLOAD
FREE 30 Days
Author : Sascha Meyer
language : en
Publisher: Frontiers Media SA
Release Date : 2022-02-14
New Therapeutic Options For Rare Diseases written by Sascha Meyer and has been published by Frontiers Media SA this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-02-14 with Medical categories.
Rare Diseases And Orphan Products
DOWNLOAD
FREE 30 Days
Author : Institute of Medicine
language : en
Publisher: National Academies Press
Release Date : 2011-04-03
Rare Diseases And Orphan Products written by Institute of Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2011-04-03 with Medical categories.
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.
Rare Diseases
DOWNLOAD
FREE 30 Days
Author : Wanda Ramirez
language : en
Publisher:
Release Date : 2017
Rare Diseases written by Wanda Ramirez and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017 with Rare diseases categories.
This book provides new research insights on rare diseases. Chapter One reviews the use of patients' registries as a key tool in rare disease management. Chapter Two discusses the worldwide experience with cytoreductive surgery (CRS) and hyperthermic intraperitoneal chemotherapy (HIPEC) for patients with rare abdominal and pelvic neoplasms with peritoneal metastases that should be evaluated for long-term benefit. Chapter Three explores the contribution of psychology-related research on Congenital Anomalies (CAs) from the analysis of international scientific literature.
Rare Disease Drug Development
DOWNLOAD
FREE 30 Days
Author : Raymond A. Huml
language : en
Publisher: Springer Nature
Release Date : 2021-11-08
Rare Disease Drug Development written by Raymond A. Huml and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-11-08 with Medical categories.
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.
Registries For Evaluating Patient Outcomes
DOWNLOAD
FREE 30 Days
Author : Agency for Healthcare Research and Quality/AHRQ
language : en
Publisher: Government Printing Office
Release Date : 2014-04-01
Registries For Evaluating Patient Outcomes written by Agency for Healthcare Research and Quality/AHRQ and has been published by Government Printing Office this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-04-01 with Medical categories.
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.
Drug Repurposing
DOWNLOAD
FREE 30 Days
Author : Farid A. Badria
language : en
Publisher: BoD – Books on Demand
Release Date : 2020-12-02
Drug Repurposing written by Farid A. Badria and has been published by BoD – Books on Demand this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-12-02 with Medical categories.
Drug repurposing or drug repositioning is a new approach to presenting new indications for common commercial and clinically approved existing drugs. For example, chloroquine, an old antimalarial drug, showed promising results for treating COVID-19, interfering with MDR in several types of cancer, and chemosensitizing human leukemic cells.This book focuses on the hypothesis, risk/benefits, and economic impacts of drug repurposing on drug discovery in dermatology, infectious diseases, neurological disorders, cancer, and orphan diseases. It brings together up-to-date research to provide readers with an informative, illustrative, and easy-to-read book useful for students, clinicians, and the pharmaceutical industry.
Rare Diseases
DOWNLOAD
FREE 30 Days
Author : Mani T. Valarmathi
language : en
Publisher: BoD – Books on Demand
Release Date : 2021-09-22
Rare Diseases written by Mani T. Valarmathi and has been published by BoD – Books on Demand this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-09-22 with Medical categories.
A rare disease is any disease or condition that affects a small percentage of the population. Many rare conditions are life-threatening or chronically debilitating, and unfortunately do not have appropriate treatments, rendering them incurable. In recent years, there has been substantial development in the area of rare disease research and its clinical applications, for instance, rare disease biology and genomics, epidemiology and preventions, early detection and screening, and diagnosis and treatment. In this context, this book consolidates the recent advances in rare disease biology and therapeutics, covering a wide spectrum of interrelated topics, and disseminates this essential knowledge in a comprehensible way to a greater scientific and clinical audience as well as patients, caregivers, and drug and device manufacturers, especially to support rare disease product development. Chapters cover such diseases as Felty’s syndrome, Löfgren’s syndrome, mesothelioma, epidermolysis bullosa, and more. This book is a valuable resource not only for medical and allied health students but also for researchers, clinical and nurse geneticists, genetic counselors, and physician assistants.
Rare Diseases
DOWNLOAD
FREE 30 Days
Author : Meral Özgüç
language : en
Publisher: Springer
Release Date : 2014-09-08
Rare Diseases written by Meral Özgüç and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-09-08 with Science categories.
This book focuses on predictive, preventative and personalized medicine (PPPM) and how it is related to the healthcare of rare diseases. Readers will discover how advanced rare diseases healthcare provides an excellent “proof-of-principles” for the personalisation of healthcare systems on a global scale. Chapters look at national plans for rare disease, at biobanking, gene identification, rare cancers, virus gene therapy , induced pluripotency for cell therapy amongst other topics. There is a chapter dedicated to personalized medicine for hereditary deafness and another exploring the complexity of genotype-phenotype correlations. Specific diseases such as Fabry's, Gauchers and mitochondrial cytopathies are highlighted and we look at enzyme replacement therapy in lysosomal storage diseases. This work is part of a series, produced with the involvement of the European Association for Predictive, Preventive and Personalised Medicine. The series focusses on the concept of an integrative medical approach by PPPM. This volume is dedicated to all aspects related to the prediction, prevention and personalised treatments of rare diseases, and in doing so it explores developments relevant to all medical branches. The authors cover ethical considerations, the creation of a robust platform for professional communication, synergies with patient organisations, “doctor-patient” collaboration and a new philosophy of integrative medicine by PPPM. This volume serves as a reference source for scientific and medical centres in the field and can be used both at medical curricula and graduate level in the life sciences. Those who place a special emphasis on healthcare promotion and innovations intended to combat rare diseases, save the affected lives and enhance life quality will all find this book of great value.
The Prevention And Treatment Of Missing Data In Clinical Trials
DOWNLOAD
FREE 30 Days
Author : National Research Council
language : en
Publisher: National Academies Press
Release Date : 2010-12-21
The Prevention And Treatment Of Missing Data In Clinical Trials written by National Research Council and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2010-12-21 with Medical categories.
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.
Innovative Methods For Rare Disease Drug Development
DOWNLOAD
FREE 30 Days
Author : Shein-Chung Chow
language : en
Publisher: CRC Press
Release Date : 2020-11-11
Innovative Methods For Rare Disease Drug Development written by Shein-Chung Chow and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-11-11 with Mathematics categories.
In the United States, a rare disease is defined by the Orphan Drug Act as a disorder or condition that affects fewer than 200,000 persons. For the approval of "orphan" drug products for rare diseases, the traditional approach of power analysis for sample size calculation is not feasible because there are only limited number of subjects available for clinical trials. In this case, innovative approaches are needed for providing substantial evidence meeting the same standards for statistical assurance as drugs used to treat common conditions. Innovative Methods for Rare Disease Drug Development focuses on biostatistical applications in terms of design and analysis in pharmaceutical research and development from both regulatory and scientific (statistical) perspectives. Key Features: Reviews critical issues (e.g., endpoint/margin selection, sample size requirements, and complex innovative design). Provides better understanding of statistical concepts and methods which may be used in regulatory review and approval. Clarifies controversial statistical issues in regulatory review and approval accurately and reliably. Makes recommendations to evaluate rare diseases regulatory submissions. Proposes innovative study designs and statistical methods for rare diseases drug development, including n-of-1 trial design, adaptive trial design, and master protocols like platform trials. Provides insight regarding current regulatory guidance on rare diseases drug development like gene therapy.