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Author: Maria Cristina Galli Publisher: Springer Nature ISBN: 3031345673 Category : Science Languages : en Pages : 243
Book Description
This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization. This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, as well as pertinent ‘must-know’ guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: a Global Perspective is part of the American Society of Gene & Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.
Author: Maria Cristina Galli Publisher: Springer Nature ISBN: 3031345673 Category : Science Languages : en Pages : 243
Book Description
This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization. This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, as well as pertinent ‘must-know’ guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: a Global Perspective is part of the American Society of Gene & Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.
Author: Maria Cristina Galli Publisher: Springer ISBN: 3319186183 Category : Medical Languages : en Pages : 230
Book Description
This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.
Author: Maria Cristina Galli Publisher: ISBN: 9783031345692 Category : Languages : en Pages : 0
Book Description
This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization. This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, as well as pertinent 'must-know' guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: a Global Perspective is part of the American Society of Gene & Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.
Author: Maria Cristina Galli Publisher: ISBN: 9783319186191 Category : Languages : en Pages :
Book Description
Medical literature for health care practitioners on the evaluation and treatment of breastfeeding issues has been disjointed, conflicting, and difficult to find. The field of breastfeeding medicine itself is nonexistent--there are no "breastfeeding doctors" who are specifically trained to understand this complex and interactive process. While much of the literature about breastfeeding describes how it "should" work, there is currently nothing available to explain why it often fails and how to treat it. Clinician's Guide to Breastfeeding: Evidence-based Evaluation and Management is written for health care practitioners who work with breastfeeding mothers; physicians, nurses, nurse practitioners, and lactation consultants. It provides clear information and clinically tested strategies to help professionals guide new mothers to breastfeed successfully. The first of its kind to consider the entirety of the breastfeeding experience, Clinician's Guide to Breastfeeding is written by Dr. Linda D. Dahl, a leading expert on the subject. It is a comprehensive review of breastfeeding, covering objective analyses of ideal or "normal" nursing, as well as the evaluation and treatment of abnormal nursing, including case studies to illustrate the treatment decision-making process.
Author: Miguel-Angel Perales Publisher: Springer ISBN: 3319543687 Category : Medical Languages : en Pages : 288
Book Description
In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee.
Author: National Academies of Sciences, Engineering, and Medicine Publisher: National Academies Press ISBN: 0309672988 Category : Medical Languages : en Pages : 127
Book Description
Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.
Author: Franz-Xaver Reichl Publisher: Springer ISBN: 9783642353734 Category : Medical Languages : en Pages : 0
Book Description
This book will be written by experts for professionals, scientists and all those involved in toxicological data generation and decision-making. It is the updated and expanded version of a monograph published in German in 2004. Chemical safety is regulated on various levels including production, storage, transport, handling, disposal or labelling. This book deals comprehensively with the safety-ensuring methods and concepts employed by regulatory agencies, industry and academics. Toxicologists use experimental and scientific approaches for data collection, e.g. about chemical hazards, physicochemical features or toxicokinetics. The respective experimental methods are described in the book. Toxicologists also deal with much insecurity in the exposure and effect scenarios during risk assessment. To overcome these, they have different extrapolation methods and estimation procedures at their disposal. The book describes these methods in an accessible manner. Differing concepts from one regulation area to another are also covered. Reasons and consequences become evident when reading the book. Altogether, the book Regulatory Toxicology will serve as an excellent reference.
Author: Institute of Medicine Publisher: National Academies Press ISBN: 030929665X Category : Medical Languages : en Pages : 134
Book Description
Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.
Author: Hazel Aranha Publisher: CRC Press ISBN: 1000864251 Category : Science Languages : en Pages : 444
Book Description
This handbook provides an in-depth review of information across the developmental spectrum of gene and cell therapy products. From introductory information to state-of-the-art technologies and concepts, the book provides insights into upstream processes such as vector design and construction, purification, formulation and fill/finish, as well as delivery options. Planning steps for compliance with current good manufacturing practice (cGMP) to readiness for chemistry, manufacturing and controls (CMC) are also discussed. This book wraps up with examples of successes and pitfalls addressed by experts who have navigated the multiple challenges that are part of any innovative endeavor. Features Provides the most up-to-date information on the development of gene therapy, from the technology involved to gene correction and genome editing Discusses siRNA, mRNA, and plasmid manufacturing Describes the importance of supplier-sponsor synergies on the path to commercialization Written for a diverse audience with a large number of individuals in the core technologies and supportive practices It is intended as a one-stop resource for the availability of state-of-the-art information related to cell and gene therapy products for researchers, scientists, management and other academic and research institutions.
Author: National Academies of Sciences, Engineering, and Medicine Publisher: National Academies Press ISBN: 0309452880 Category : Medical Languages : en Pages : 329
Book Description
Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.
Author: Maria Cristina Galli
Author: Maria Cristina Galli
Author: Maria Cristina Galli
Author: Maria Cristina Galli
Author: Maria Cristina Galli
Author: Miguel-Angel Perales
Author: National Academies of Sciences, Engineering, and Medicine
Author: Franz-Xaver Reichl
Author: Institute of Medicine
Author: Hazel Aranha
Author: National Academies of Sciences, Engineering, and Medicine