Rare Disease Drug Development

Author : Raymond A. Huml
Publisher : Springer Nature
Page : 418 pages
File Size : 53,5 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.

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 : 47,7 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.

Author : Shein-Chung Chow
Publisher : CRC Press
Page : 286 pages
File Size : 47,7 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.

Author : Bo Yang,Yang Song,Yijie Zhou
Publisher : CRC Press
Page : 233 pages
File Size : 41,9 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.

Author : Shein-Chung Chow
Publisher : CRC Press
Page : 306 pages
File Size : 54,9 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.

Author : David C Pryde,Michael J Palmer
Publisher : Royal Society of Chemistry
Page : 350 pages
File Size : 53,5 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.

Author : Jocelyn Jennings,Linda McBride
Publisher : Unknown
Page : 128 pages
File Size : 41,8 Mb
Release : 2021-11-26
Category : Electronic
ISBN : 1947493736

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Global Pediatric Development of Drugs, Biologics, and Medical Devices by Jocelyn Jennings,Linda McBride 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 : 41,6 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.

Author : Farid A. Badria
Publisher : BoD – Books on Demand
Page : 236 pages
File Size : 40,9 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.

Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanović
Publisher : Elsevier
Page : 334 pages
File Size : 42,7 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

Author : I. Herbert Scheinberg,J. M. Walshe
Publisher : Manchester University Press
Page : 244 pages
File Size : 41,6 Mb
Release : 1986
Category : Medical
ISBN : 0719022967

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Orphan Diseases and Orphan Drugs by I. Herbert Scheinberg,J. M. Walshe Pdf

Author : Gerard Marshall Raj,Ramasamy Raveendran
Publisher : Springer Nature
Page : 410 pages
File Size : 45,9 Mb
Release : 2019-11-16
Category : Medical
ISBN : 9789813297791

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Introduction to Basics of Pharmacology and Toxicology by Gerard Marshall Raj,Ramasamy Raveendran Pdf

This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology.

Author : Nicolas Sireau
Publisher : Routledge
Page : 237 pages
File Size : 44,5 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.

Author : Anonim
Publisher : Unknown
Page : 48 pages
File Size : 48,6 Mb
Release : 1982
Category : Drugs
ISBN : STANFORD:36105119622699

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Preliminary Report of the Survey on Drugs for Rare Disease by Anonim Pdf

Author : Manuel Posada de la Paz,Domenica Taruscio,Stephen C. Groft
Publisher : Springer
Page : 667 pages
File Size : 45,7 Mb
Release : 2017-12-06
Category : Medical
ISBN : 9783319671444

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Rare Diseases Epidemiology: Update and Overview by Manuel Posada de la Paz,Domenica Taruscio,Stephen C. Groft Pdf

The fields of rare diseases research and orphan products development continue to expand with more products in research and development status. In recent years, the role of the patient advocacy groups has evolved into a research partner with the academic research community and the bio-pharmaceutical industry. Unique approaches to research and development require epidemiological data not previously available to assist in protocol study design and patient recruitment for clinical trials required by regulatory agencies prior to approval for access by patents and practicing physicians.