Innovative Methods For Rare Disease Drug Development

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Innovative Methods for Rare Disease Drug Development

Shein-Chung Chow
Innovative Methods for Rare Disease Drug Development Book PDF

Author : Shein-Chung Chow
Publisher : CRC Press
Page : 306 pages
File Size : 41,9 Mb
Release : 2020-11-12
Category : Mathematics
ISBN : 9781000208214

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Innovative Methods for Rare Disease Drug Development by Shein-Chung Chow Pdf

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.

Innovative Methods for Rare Disease Drug Development

Shein-Chung Chow
Innovative Methods for Rare Disease Drug Development Book PDF

Author : Shein-Chung Chow
Publisher : CRC Press
Page : 286 pages
File Size : 48,7 Mb
Release : 2020-11-11
Category : Mathematics
ISBN : 9781000208337

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Innovative Methods for Rare Disease Drug Development by Shein-Chung Chow Pdf

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.

Drug Development for Rare Diseases

Bo Yang,Yang Song,Yijie Zhou
Drug Development for Rare Diseases Book PDF

Author : Bo Yang,Yang Song,Yijie Zhou
Publisher : CRC Press
Page : 233 pages
File Size : 51,5 Mb
Release : 2023-01-10
Category : Mathematics
ISBN : 9781000820225

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Drug Development for Rare Diseases by Bo Yang,Yang Song,Yijie Zhou Pdf

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.

Breakthrough Business Models

Institute of Medicine,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation,Robert Giffin,Sally Robinson,Theresa Wizemann
Breakthrough Business Models Book PDF

Author : Institute of Medicine,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation,Robert Giffin,Sally Robinson,Theresa Wizemann
Publisher : National Academies Press
Page : 151 pages
File Size : 47,5 Mb
Release : 2009-02-17
Category : Medical
ISBN : 9780309178167

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Breakthrough Business Models by Institute of Medicine,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation,Robert Giffin,Sally Robinson,Theresa Wizemann Pdf

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.

Rare Diseases and Orphan Products

Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development
Rare Diseases and Orphan Products Book PDF

Author : Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development
Publisher : National Academies Press
Page : 442 pages
File Size : 54,8 Mb
Release : 2011-04-03
Category : Medical
ISBN : 9780309158060

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Rare Diseases and Orphan Products by Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development Pdf

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 Disease Drug Development

Raymond A. Huml
Rare Disease Drug Development Book PDF

Author : Raymond A. Huml
Publisher : Springer Nature
Page : 418 pages
File Size : 40,5 Mb
Release : 2021-11-08
Category : Medical
ISBN : 9783030786052

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Rare Disease Drug Development by Raymond A. Huml Pdf

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.

Orphan Drugs and Rare Diseases

David C Pryde,Michael J Palmer
Orphan Drugs and Rare Diseases Book PDF

Author : David C Pryde,Michael J Palmer
Publisher : Royal Society of Chemistry
Page : 350 pages
File Size : 46,8 Mb
Release : 2014-07-30
Category : Science
ISBN : 9781782624202

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Orphan Drugs and Rare Diseases by David C Pryde,Michael J Palmer Pdf

Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world’s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target. Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from biopharmaceutical companies to tackle rare diseases. These developments have made rare diseases, and the orphan drugs that treat them, sufficiently attractive to pharmaceutical development and many pharmaceutical companies now have research units dedicated to this area of research. It is therefore timely to review the area of orphan drugs and some of the basic science, drug discovery and regulatory factors that underpin this important, and growing, area of biomedical research. Written by a combination of academic and industry experts working in the field, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat. Providing an up-to-date monograph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs and will appeal to medicinal and pharmaceutical chemists, biochemists and anyone working within the fields of rare disease research and drug development or pharmaceuticals in industry or academia.

Advanced Statistics in Regulatory Critical Clinical Initiatives

Wei Zhang,Fangrong Yan,Feng Chen,Shein-Chung Chow
Advanced Statistics in Regulatory Critical Clinical Initiatives Book PDF

Author : Wei Zhang,Fangrong Yan,Feng Chen,Shein-Chung Chow
Publisher : CRC Press
Page : 501 pages
File Size : 51,8 Mb
Release : 2022-05-25
Category : Mathematics
ISBN : 9781000568028

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Advanced Statistics in Regulatory Critical Clinical Initiatives by Wei Zhang,Fangrong Yan,Feng Chen,Shein-Chung Chow Pdf

Advanced Statistics in Regulatory Critical Clinical Initiatives is focused on the critical clinical initiatives introduced by the 21st Century Cure Act passed by the United States Congress in December 2016. The book covers everything from the outline of the initiatives to analysis on the effect on biopharmaceutical research and development. Advanced Statistics in Regulatory Critical Clinical Initiatives provides innovative ways to resolve common challenges in statistical research of rare diseases such small sample sizes and provides guidance for combined use of data. With analysis from regulatory and scientific perspectives this book is an ideal companion for researchers in biostatistics, pharmaceutical development, and policy makers in related fields. Key Features: Provides better understanding of innovative design and analysis of each critical clinical initiatives which may be used in regulatory review/approval of drug development. Makes recommendations to evaluate submissions accurately and reliably. Proposes innovative study designs and statistical methods for oncology and/or rare disease drug development. Provides insight regarding current regulatory guidance on drug development such as gene therapy and rare diseases.

The Role of NIH in Drug Development Innovation and Its Impact on Patient Access

National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Board on Health Care Services
The Role of NIH in Drug Development Innovation and Its Impact on Patient Access Book PDF

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Board on Health Care Services
Publisher : National Academies Press
Page : 103 pages
File Size : 49,6 Mb
Release : 2020-01-27
Category : Medical
ISBN : 9780309498517

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The Role of NIH in Drug Development Innovation and Its Impact on Patient Access by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Board on Health Care Services Pdf

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.

Precision Public Health

Tarun Weeramanthri,Hugh Dawkins,Gareth Baynam,Matthew Bellgard,Ori Gudes,James Semmens
Precision Public Health Book PDF

Author : Tarun Weeramanthri,Hugh Dawkins,Gareth Baynam,Matthew Bellgard,Ori Gudes,James Semmens
Publisher : Frontiers Media SA
Page : 149 pages
File Size : 41,5 Mb
Release : 2018-06-25
Category : Electronic
ISBN : 9782889455010

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Precision Public Health by Tarun Weeramanthri,Hugh Dawkins,Gareth Baynam,Matthew Bellgard,Ori Gudes,James Semmens Pdf

Precision Public Health is a new and rapidly evolving field, that examines the application of new technologies to public health policy and practice. It draws on a broad range of disciplines including genomics, spatial data, data linkage, epidemiology, health informatics, big data, predictive analytics and communications. The hope is that these new technologies will strengthen preventive health, improve access to health care, and reach disadvantaged populations in all areas of the world. But what are the downsides and what are the risks, and how can we ensure the benefits flow to those population groups most in need, rather than simply to those individuals who can afford to pay? This is the first collection of theoretical frameworks, analyses of empirical data, and case studies to be assembled on this topic, published to stimulate debate and promote collaborative work.

Innovative Medicine

Kazuwa Nakao,Nagahiro Minato,Shinji Uemoto
Innovative Medicine Book PDF

Author : Kazuwa Nakao,Nagahiro Minato,Shinji Uemoto
Publisher : Springer
Page : 330 pages
File Size : 41,5 Mb
Release : 2015-10-13
Category : Science
ISBN : 9784431556510

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Innovative Medicine by Kazuwa Nakao,Nagahiro Minato,Shinji Uemoto Pdf

This book is devoted to innovative medicine, comprising the proceedings of the Uehara Memorial Foundation Symposium 2014. It remains extremely rare for the findings of basic research to be developed into clinical applications, and it takes a long time for the process to be achieved. The task of advancing the development of basic research into clinical reality lies with translational science, yet the field seems to struggle to find a way to move forward. To create innovative medical technology, many steps need to be taken: development and analysis of optimal animal models of human diseases, elucidation of genomic and epidemiological data, and establishment of “proof of concept”. There is also considerable demand for progress in drug research, new surgical procedures, and new clinical devices and equipment. While the original research target may be rare diseases, it is also important to apply those findings more broadly to common diseases. The book covers a wide range of topics and is organized into three complementary parts. The first part is basic research for innovative medicine, the second is translational research for innovative medicine, and the third is new technology for innovative medicine. This book helps to understand innovative medicine and to make progress in its realization.

Rare Diseases

Nicolas Sireau
Rare Diseases Book PDF

Author : Nicolas Sireau
Publisher : Routledge
Page : 237 pages
File Size : 53,5 Mb
Release : 2017-09-08
Category : Business & Economics
ISBN : 9781351278386

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Rare Diseases by Nicolas Sireau Pdf

There are 7,000 rare diseases affecting 6%–8% of the global population. That's 3.5 million people in the UK alone. Yet only 200 rare diseases have approved treatments. In recent years, there has been a surge of interest from business and social entrepreneurs in the field of health – including looking at ways to treat rare disease patients better and faster. This book presents some of the latest developments in the world of rare disease entrepreneurship from a global group of experts. It examines the topic from the business angle, considering the drug development process and providing case studies of successful orphan drug enterprises. It also looks at rare diseases from the perspective of the patient, analysing the growing rare disease patient movement, a successful patient group that uses social enterprise techniques, and chapters on key requirements for helping patients with rare diseases through registries and centres of excellence. The book will be an essential toolkit for social and business entrepreneurs who are interested in the world of rare/orphan diseases. It has the rigour of an academic publication, along with the clarity of a lay publication. An original and timely book, Rare Diseases will help to add knowledge and awareness to a vastly under-published subject.

Case Studies in Innovative Clinical Trials

Kristine Broglio,Binbing Yu
Case Studies in Innovative Clinical Trials Book PDF

Author : Kristine Broglio,Binbing Yu
Publisher : CRC Press
Page : 331 pages
File Size : 41,6 Mb
Release : 2023-11-27
Category : Medical
ISBN : 9781000987249

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Case Studies in Innovative Clinical Trials by Kristine Broglio,Binbing Yu Pdf

Drug development is a strictly regulated area. As such, marketing approval of a new drug depends heavily, if not exclusively, on evidence generated from clinical trials. Drug development has seen tremendous innovation in science and technology that has revolutionized the treatment of some diseases. And yet, the statistical design and practical conduct of the clinical trials used to test new therapeutics for safety and efficacy have changed very little over the decades. Our approach to clinical trials is steeped in convention and tradition. The large, fixed, randomized controlled trial methods that have been the gold standard are well understood and expected by many trial stakeholders. However, this approach is not well suited to all aspects of modern drug development and the current competitive landscape. We now see new therapies that target a small fraction of the patient population, rare diseases with high unmet medical needs, and pediatric populations that must wait for years for new drug approvals from the time that therapies are approved in adults. Large randomized clinical trials are at best inefficient and at worst completely infeasible in many modern clinical settings. Advances in technology and data infrastructure call for innovations in clinical trial design. Despite advances in statistical methods, the availability of information, and computing power, the actual experience with innovative design in clinical trials across industry and academia is limited. This book will be an important showcase of the potential for these innovative designs in modern drug development and will be an important resource to guide those who wish to undertake them for themselves. This book is ideal for professionals in the pharmaceutical industry and regulatory agencies, but it will also be useful to academic researchers, faculty members, and graduate students in statistics, biostatistics, public health, and epidemiology due to its focus on innovation. Key Features: Is written by pharmaceutical industry experts, academic researchers, and regulatory reviewers; this is the first book providing a comprehensive set of case studies related to statistical methodology, implementation, regulatory considerations, and communication of complex innovative trial design. Has a broad appeal to a multitude of readers across academia, industry, and regulatory agencies. Each contribution is a practical case study that can speak to the benefits of an innovative approach but also balance that with the real-life challenges encountered. A complete understanding of what is actually being done in modern clinical trials will broaden the reader’s capabilities and provide examples to first mimic and then customize and expand upon when exploring these ideas on their own.

Orphan Drugs

Elizabeth Hernberg-Ståhl,Miroslav Reljanović
Orphan Drugs Book PDF

Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanović
Publisher : Elsevier
Page : 334 pages
File Size : 53,7 Mb
Release : 2013-11-15
Category : Medical
ISBN : 9781908818393

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Orphan Drugs by Elizabeth Hernberg-Ståhl,Miroslav Reljanović Pdf

This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patients A source of updated information, news and trends for those who are already active in this fast-evolving field Covers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe

Drug Repurposing

Farid A. Badria
Drug Repurposing Book PDF

Author : Farid A. Badria
Publisher : BoD – Books on Demand
Page : 236 pages
File Size : 51,5 Mb
Release : 2020-12-02
Category : Medical
ISBN : 9781839685200

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Drug Repurposing by Farid A. Badria Pdf

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.