New Drug Development

A Regulatory Overview

Author: Mark P. Mathieu,Christopher-Paul Milne

Publisher: Barnett Educational Services / Chi

ISBN: 9781882615858

Category: Medical

Page: 362

View: 2264

"Go inside the drug development and FDA regulatory process with today's most authoritative and popular reference on the topic. In its all-new 2008 edition, New Drug Development: A Regulatory Overview addresses the most cutting-edge developments redefining how new drugs are developed and regulated today, including: how the FDA Amendments Act of 2007 will affect everything from drug reviews to postmarketing requirements; how the CDER's efforts to integrate a culture of drug safety has affected the center's structure and its new drug review and approval processes; how CDER's much-anticipated January 2008 transition to the eCTD as the only valid esubmission format will affect the FDA's drug submission and review process; how the FDA and industry are already integrating pharmacogenomics, computer simulation, and other emerging technologies to inform key decisions; and which drug development strategies are fulfilling their promise and offering optimal returns for industry, given the explosion of accelerated development/approval programs and pilot programs to speed the drug development and review process."--Publisher's description.
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Early Drug Development

Strategies and Routes to First-in-Human Trials

Author: Mitchell N. Cayen

Publisher: John Wiley & Sons

ISBN: 9781118035207

Category: Medical

Page: 654

View: 2272

The focus of early drug development has been the submission of an Investigational New Drug application to regulatory agencies. Early Drug Development: Strategies and Routes to First-in-Human Trials guides drug development organizations in preparing and submitting an Investigational New Drug (IND) application. By explaining the nuts and bolts of preclinical development activities and their interplay in effectively identifying successful clinical candidates, the book helps pharmaceutical scientists determine what types of discovery and preclinical research studies are needed in order to support a submission to regulatory agencies.
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Global New Drug Development

An Introduction

Author: Jan A. Rosier,Mark A. Martens,Josse R. Thomas

Publisher: John Wiley & Sons

ISBN: 1118414853

Category: Medical

Page: 424

View: 9594

The development of new drugs is very complex, costly and risky. Its success is highly dependent on an intense collaboration and interaction between many departments within the drug development organization, external investigators and service providers, in constant dialogue with regulatory authorities, payers, academic experts, clinicians and patient organizations. Within the different phases of the drug life cycle, drug development is by far the most crucial part for the initial and continued success of a drug on the market. This book offers an introduction to the field of drug development with a clear overview of the different processes that lead to a successful new medicine and of the regulatory pathways that are used to launch a new drug that are both safe and efficacious. “This is the most comprehensive and detailed book on drug development I have ever read and I feel that it is likely to become a staple of drug development courses, such as those taught at Masters Level in my own University…. I think in the light of increasing integration of company and academic approaches to drug development both sides can read this book.. (and, therefore)… this book could not be more timely. “ Professor Mike Coleman, University of Aston., UK ( from his review of the final manuscript)
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New Drug Development

An Introduction to Clinical Trials: Second Edition

Author: J. Rick Turner

Publisher: Springer Science & Business Media

ISBN: 9781441964182

Category: Medical

Page: 256

View: 5167

New Drug Development: Second Edition provides an overview of the design concepts and statistical practices involved in therapeutic drug development. This wide spectrum of activities begins with identifying a potentially useful drug candidate that can perhaps be used in the treatment or prevention of a condition of clinical concern, and ends with marketing approval being granted by one or more regulatory agencies. In between, it includes drug molecule optimization, nonclinical and clinical evaluations of the drug’s safety and efficacy profiles, and manufacturing considerations. The more inclusive term lifecycle drug development can be used to encompass the postmarketing surveillance that is conducted all the time that a drug is on the market and being prescribed to patients with the relevant clinical condition. Information gathered during this time can be used to modify the drug (for example, dose prescribed, formulation, and mode of administration) in terms of its safety and its effectiveness. The central focus of the first edition of this book is captured by its subtitle, 'Design, Methodology, and Analysis'. Optimum quality study design and experimental research methodology must be employed if the data collected—numerical representations of biological information—are to be of optimum quality. Optimum quality data facilitate optimum quality statistical analysis and interpretation of the results obtained, which in turn permit optimum quality decisions to be made: Rational decision making is predicated on appropriate research questions and optimum quality numerical information. The book took a non-computational approach to statistics, presenting instead a conceptual framework and providing readers with a sound working knowledge of the importance of design, methodology, and analysis. Not everyone needs to be an expert in statistical analysis, but it is very helpful for work (or aspire to work) in the pharmaceutical and biologics industries to be aware of the fundamental importance of a sound scientific and clinical approach to the planning, conduct, and analysis of clinical trials.
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Drugs

From Discovery to Approval

Author: Rick Ng

Publisher: John Wiley & Sons

ISBN: 1118907191

Category: Science

Page: 552

View: 1895

The third edition of this best-selling book continues to offer a user-friendly, step-by-step introduction to all the key processes involved in bringing a drug to the market, including the performance of pre-clinical studies, the conduct of human clinical trials, regulatory controls, and even the manufacturing processes for pharmaceutical products. Concise and easy to read, Drugs: From Discovery to Approval, Third Edition quickly introduces basic concepts, then moves on to discuss target selection and the drug discovery process for both small and large molecular drugs. The third edition incorporates the latest developments and updates in the pharmaceutical community, provides more comprehensive coverage of topics, and includes more materials and case studies suited to college and university use. Biotechnology is a dynamic field with changes across R&D, clinical trials, manufacturing and regulatory processes, and the third edition of the text provides timely updates for those in this rapidly growing field.
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Biological Drug Products

Development and Strategies

Author: Wei Wang,Manmohan Singh

Publisher: John Wiley & Sons

ISBN: 1118695224

Category: Medical

Page: 744

View: 7114

Tested and proven solutions to the challenges of biological drug product development Biological drug products play a central role in combating human diseases; however, developing new successful biological drugs presents many challenges, including labor intensive production processes, tighter regulatory controls, and increased market competition. This book reviews the current state of the science, offering readers a single resource that sets forth the fundamentals as well as tested and proven development strategies for biological drugs. Moreover, the book prepares readers for the challenges that typically arise during drug development, offering straightforward solutions to improve their ability to pass through all the regulatory hurdles and deliver new drug products to the market. Biological Drug Products begins with general considerations for the development of any biological drug product and then explores the strategies and challenges involved in the development of specific types of biologics. Divided into five parts, the book examines: Part 1: General Aspects Part 2: Proteins and Peptides Part 3: Vaccines Part 4: Novel Biologics Part 5: Product Administration/Delivery Each chapter has been prepared by one or more leading experts in biological drug development. Contributions are based on a comprehensive review and analysis of the current literature as well as the authors' first-hand experience developing and testing new drugs. References at the end of each chapter serve as a gateway to original research papers and reviews in the field. By incorporating lessons learned and future directions for research, Biological Drug Products enables pharmaceutical scientists and students to improve their success rate in developing new biologics to treat a broad range of human diseases.
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Pharmacovigilance Medical Writing

A Good Practice Guide

Author: Justina Orleans-Lindsay

Publisher: John Wiley & Sons

ISBN: 1118302060

Category: Medical

Page: 288

View: 8820

Pharmacovigilance Medical Writing covers the preparation of pharmacovigilance documents for all stages of the drug development process (i.e. from clinical development through to applications for marketing authorisations to the post-marketing stage). For each document, the book presents a review of the regulatory framework that governs the content of the document, followed by practical guidance (e.g. scheduling, source data, department/functions involved in document preparation/review, appropriate timelines and planning activities), ending with a generic model document compliant with the current guidelines, which can be modified to meet specific company and product requirements.
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Medical Device Development

Regulation and Law

Author: Jonathan S. Kahan

Publisher: Barnett Educational Services / Chi

ISBN: 9781882615926

Category: Law

Page: 546

View: 6522

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Applied Linear Models with SAS

Author: Daniel Zelterman

Publisher: Cambridge University Press

ISBN: 1139489003

Category: Medical

Page: N.A

View: 9871

This textbook for a second course in basic statistics for undergraduates or first-year graduate students introduces linear regression models and describes other linear models including Poisson regression, logistic regression, proportional hazards regression, and nonparametric regression. Numerous examples drawn from the news and current events with an emphasis on health issues illustrate these concepts. Assuming only a pre-calculus background, the author keeps equations to a minimum and demonstrates all computations using SAS. Most of the programs and output are displayed in a self-contained way, with an emphasis on the interpretation of the output in terms of how it relates to the motivating example. Plenty of exercises conclude every chapter. All of the datasets and SAS programs are available from the book's website, along with other ancillary material.
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FDA Regulatory Affairs

Third Edition

Author: David Mantus,Douglas J. Pisano

Publisher: CRC Press

ISBN: 1841849200

Category: Medical

Page: 400

View: 3158

FDA Regulatory Affairs is a roadmap to prescription drug, biologics, and medical device development in the United States. Written in plain English, the concise and jargon-free text demystifies the inner workings of the US Food and Drug Administration (FDA) and facilitates an understanding of how the agency operates with respect to compliance and product approval, including clinical trial exemptions, fast track status, advisory committee procedures, and more. The Third Edition of this highly successful publication: Examines the harmonization of the US Federal Food, Drug, and Cosmetic Act with international regulations on human drug, biologics and device development, research, manufacturing, and marketing Includes contributions from experts at organizations such as the FDA, National Institutes of Health (NIH), and PAREXEL Focuses on the new drug application (NDA) process, cGMPs, GCPs, quality system compliance, and corresponding documentation requirements Provides updates to the FDA Safety and Innovation Act (FDASIA), incorporating pediatric guidelines and follow-on biologics regulations from the 2012 Prescription Drug User Fee Act (PDUFA) V Explains current FDA inspection processes, enforcement options, and how to handle FDA meetings and required submissions Co-edited by an industry leader (Mantus) and a respected academic (Pisano), FDA Regulatory Affairs, Third Edition delivers a compilation of the selected US laws and regulations as well as a straightforward commentary on the FDA product approval process that’s broadly useful to both business and academia.
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Real World Drug Discovery

A Chemist's Guide to Biotech and Pharmaceutical Research

Author: Robert M. Rydzewski

Publisher: Elsevier

ISBN: 9780080914886

Category: Medical

Page: 600

View: 5513

Drug discovery increasingly requires a common understanding by researchers of the many and diverse factors that go into the making of new medicines. The scientist entering the field will immediately face important issues for which his education may not have prepared him: project teams, patent law, consultants, target product profiles, industry trends, Gantt charts, target validation, pharmacokinetics, proteomics, phenotype assays, biomarkers, and many other unfamiliar topics for which a basic understanding must somehow be obtained. Even the more experienced scientist can find it frustratingly difficult to get an overview of the many factors involved in modern drug discovery and often only after years of exploring does a whole and integrated picture emerge in the mind of the researcher. Real World Drug Discovery: A Chemist’s Guide to Biotech and Pharmaceutical Research presents this kind of map of the landscape of drug discovery. In a single, readable volume it outlines processes and explains essential concepts and terms for the recent science graduate wondering what to expect in pharma or biotech, the medicinal chemist seeking a broader and more timely understanding of the industry, or the contractor or collaborator whose understanding of the commercial drug discovery process could increase the value of his contribution to it. Interviews with well-known experts in many of the fields involved, giving insightful comments from authorities on many of the sub-disciplines important to cutting edge drug discovery. Helpful suggestions gleaned from years of experience in biotech and pharma, which represents a repository drug discovery "lore" not previously available in any book. "Periodic Table of Drugs" listing current top-selling drugs arranged by target and laid out so that structural similarities and differences are plain and clear. Extensive use of diagrams to illustrate concepts like biotech startup models, preteomic profiling for target identification, Gantt charts for project planning, etc.
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Patent Valuation

Improving Decision Making Through Analysis

Author: William J. Murphy,John L. Orcutt,Paul C. Remus

Publisher: John Wiley & Sons

ISBN: 1118027345

Category: Business & Economics

Page: 379

View: 4760

A practical resource for valuing patents that is accessible to the complete spectrum of decision makers in the patent process In today's economy, patents tend to be the most important of the intellectual property (IP) assets. It is often the ability to create, manage, defend, and extract value from patents that can distinguish competitive success and significant wealth creation from competitive failure and economic waste. Patent Valuation enhances the utility and value of patents by providing IP managers, IP creators, attorneys, and government officials with a useable resource that allows them to use actual or implied valuations when making patent-related decisions. Involves a combination of techniques for describing patent valuation Includes descriptions of various topics, illustrative cases, step-by-step valuation techniques, user-friendly procedures and checklists, and examples Serves as a useable resource that allows IP managers to use actual or implied valuations when making patent-related decisions One of the most fundamental premises of the book is that these valuation skills can be made accessible to each of the various decision makers in the patent process. Patent Valuation involves narrative descriptions of the various topics, illustrative cases, step-by-step valuation techniques, user-friendly procedures and checklists, and an abundance of examples to demonstrate the more complex concepts.
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Food and Drug Law and Regulation

Author: David G. Adams,Richard M. Cooper,Martin J. Hahn,Jonathan S. Kahan

Publisher: N.A

ISBN: 9781935065739

Category: Law

Page: 995

View: 3392

Written by highly experienced food and drug attorneys and edited by four of the most distinguished authorities in the field, Food and Drug Law and Regulation is the most comprehensive guidebook covering an area that accounts for more than 20 percent of all consumer spending in the United States.
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In vivo Models for Drug Discovery

Author: Jos? Miguel Vela,Rafael Maldonado,Michel Hamon

Publisher: John Wiley & Sons

ISBN: 3527679367

Category: Medical

Page: 594

View: 8368

This one-stop reference is the first to present the complete picture -- covering all relevant organisms, from single cells to mammals, as well as all major disease areas, including neurological disorders, cancer and infectious diseases. Addressing the needs of the pharmaceutical industry, this unique handbook adopts a broad perspective on the use of animals in the early part of the drug development process, including regulatory rules and limitations, as well as numerous examples from real-life drug development projects. After a general introduction to the topic, the expert contributors from research-driven pharmaceutical companies discuss the basic considerations of using animal models, including ethical issues. The main part of the book systematically surveys the most important disease areas for current drug development, from cardiovascular to endocrine disorders, and from infectious to neurological diseases. For each area, the availability of animal models for target validation, hit finding and lead profiling is reviewed, backed by numerous examples of both successes and failures among the use of animal models. The whole is rounded off with a discussion of perspectives and challenges. Key knowledge for drug researchers in industry as well as academia.
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Complement Therapeutics

Author: John D. Lambris,V. Michael Holers,Daniel Ricklin

Publisher: Springer Science & Business Media

ISBN: 146144117X

Category: Medical

Page: 320

View: 5690

This book highlights progress and trends in the rapidly evolving field of complement-related drug discovery and spotlights examples of clinical applications. As an integral part of innate immunity and critical mediator in homeostatic and inflammatory processes, the human complement system has been identified as contributor to a large number of disorders including ocular, cardiovascular, metabolic, autoimmune, and inflammatory diseases as well as in ischemia/reperfusion injury, cancer and sepsis. In addition, complement is often involved in adverse immune reactions to biomaterials, cell and organ transplants or drug delivery systems. Although the complement cascade with its close to 50 extracellular protein targets has long been recognized as an attractive system for therapeutic modulation, the past few years have seen a particularly strong boost in interest. Fueled by novel research insight and the marketing of the first complement-targeted drugs, a plethora of highly creative treatment approaches and potent drug candidates have recently emerged and are currently evaluated in disease models and clinical trials. The chapters in this book cover a wide range of topics related to the development of complement therapeutics, ranging from the molecular and functional description of complement targets to the presentation of novel inhibitors, improved treatment strategies as well as examples of disease models and clinical applications. The broad and up-to-date overview on a highly versatile and dynamic field renders this book an indispensable source of information for researchers and clinicians dealing with therapeutic and disease-related aspects of the human complement system.
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Guide to Drug Development

A Comprehensive Review and Assessment

Author: Bert Spilker

Publisher: Lippincott Williams & Wilkins

ISBN: 9780781774246

Category: Business & Economics

Page: 1277

View: 6668

Written by one of the foremost authorities on clinical trials, drug development, and regulatory affairs, Guide to Drug Development is a comprehensive review of the principles and activities involved in developing new drugs, devices, and other medical products. The book covers many topics not discussed in any other textbook and includes timely discussions on electronic clinical trials, registries of clinical trials, data mining, computer simulations and modeling, and changing regulatory standards. Each chapter includes practical tips, lessons, guides, firsthand stories, quotes from experts, and three to six questions for group discussion. The last three chapters present twelve case studies each on clinical trials, regulatory affairs, and management of drug development. Spilker's Guide to Drug Development will be the standard reference text for everyone working on or studying drug discovery or development, in industry, academia, hospitals, government, and independent laboratories.
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Combination Products

Regulatory Challenges and Successful Product Development

Author: Smita Gopalaswamy,Venky Gopalaswamy

Publisher: CRC Press

ISBN: 9781420064513

Category: Medical

Page: 264

View: 834

The field of combination product development (products born of the integration of medical devices, biologics, and drugs) is so new that, while literature abounds on each part individually, there are very few publications, including FDA documents, available concerning the unique challenges posed by this nascent but fast-growing area. Providing the first in-depth look at this breakthrough field, Combination Products includes practical guidelines and a detailed step-by-step process for the development of these novel technologies. It addresses the technical, scientific, regulatory, and quality issues that arise when combining drugs, biologics, and medical devices into a single product. It takes a practical, readily applicable approach to discussing the challenges, victories, and pitfalls associated with merging technologies and systems and how to implement these products into the market successfully and in a timely manner. Specifically, this text explores the process from start to finish, establishing a workable design and development plan complete with relevant definitions. It reviews FDA and other regulatory expectations and covers resource requirements, manufacturing pitfalls, post-launch compliance requirements, and agency audits and challenges. Drawing on the experience and expertise of two leaders in their respective fields, Combination Products boasts the credentials of Dr. Smita Gopalaswamy, a 20 year veteran of technical consulting responsibilities in medical device, biologics, and pharmaceutical industries as well as combination products, along with the support of Dr. Venky Gopalaswamy, an expert in business improvement methodologies such as six sigma, lean, and change management, to provide a comprehensive assessment of the field and an efficient and effective approach to the creation and implementation of combination products.
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Biobetters

Protein Engineering to Approach the Curative

Author: Amy Rosenberg,Barthélemy Demeule

Publisher: Springer

ISBN: 1493925431

Category: Medical

Page: 378

View: 4820

“Biobetters: Protein Engineering to Approach the Curative” discusses the optimization of protein therapeutic products for treatment of human diseases. It is based on the fact that though numerous important therapeutic protein products have been developed for life threatening and chronic diseases that possess acceptable safety and efficacy profiles, these products have generally not been reexamined and modified for an improved clinical performance, with enhancements both to safety and efficacy profiles. Advances in protein engineering, coupled with greatly enhanced understanding of critical product quality attributes for efficacy and safety, make it possible to optimize predecessor products for clinical performance, thereby enhancing patient quality of life and with the potential for great savings in health care costs. Yet despite such knowledge, there is little movement towards such modifications. This book examines engineering protein therapeutic products such that they exhibit an optimal, not just an adequate, clinical performance profile. Two product classes, therapeutic enzymes for lysosomal storage diseases (enzyme replacement therapies, ERT) and monoclonal antibodies (mAbs), are used as examples of what modifications to such proteins could be made to enhance clinical performance, “closer to a cure” as it were. For ERT, the key to optimizing clinical performance is to ensure the ERT is endowed with moieties that target the protein to the relevant target tissue. Thus, for Gaucher Disease, our best example of how to optimize an ERT to address a disease that manifests in specific target tissues (macrophages and monocytes), the enzyme has been extensively modified to target macrophages. For diseases such as Pompe Disease, largely a disorder of muscle, optimal performance of ERT will depend on endowing the enzyme with the ability to be taken up via the Mannose 6 Phosphate Receptor, and so one of the chapters in the book will discuss such approaches. Moreover, a major failure of biotechnology based products is to gain access to the CNS, a key target tissue in numerous diseases. Thus, a chapter has been devoted to strategies to access the CNS. Additionally, immune responses to therapeutic proteins can be highly problematic, eliminating the efficacy of life saving or highly effective protein therapeutics. This is especially poignant in the case of Pompe Disease wherein great improvement in muscle strength and functionality is lost following development of an immune response to the ERT with consequent patient deterioration and death. Thus, a chapter regarding protein engineering, as well as other non-clinical approaches to diminishing immunogenicity is a valuable part of the book. Monoclonal antibodies (mAbs) can be engineered to bind targets relevant to a wide variety of diseases; binding affinity, however, is only part of the equation and one of the chapters will present a molecular assessment approach that balances affinity with pharmacokinetics and manufacturability. As with other proteins immunogenicity can be problematic, being responsible for loss of efficacy of anti-TNF mAbs, often after prolonged successful treatment. The authors will also share their perspective on the consequences of physico-chemical modifications occurring to mAbs once they reach the circulation or their target, a research area open to further development from a protein engineering as well as analytical perspective. This book will also discuss novel platforms for protein therapeutics, technologies that exceed mAbs with respect to potency, and hence, potentially efficacy. These platforms consist largely of repeat domain proteins with very high affinity for their target ligands, but while potentially more efficacious, immunogenicity may be a major problem limiting use. The economics surrounding the issue of biobetters is another high-profile issue - this final chapter will explore the incentives and disincentives for developing biobetters and consider incentives that might make their pursuit more rewarding.
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