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Novel Designs of Early Phase Trials for Cancer Therapeutics

Novel Designs of Early Phase Trials for Cancer Therapeutics
A Book

by Shivaani Kummar,Chris Takimoto

  • Publisher : Academic Press
  • Release : 2018-05-22
  • Pages : 234
  • ISBN : 0128125705
  • Language : En, Es, Fr & De
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Novel Designs of Early Phase Trials for Cancer Therapeutics provides a comprehensive review by leaders in the field of the process of drug development, the integration of molecular profiling, the changes in early phase trial designs, and endpoints to optimally develop a new generation of cancer therapeutics. The book discusses topics such as statistical perspectives on cohort expansions, the role and application of molecular profiling and how to integrate biomarkers in early phase trials. Additionally, it discusses how to incorporate patient reported outcomes in phase one trials. This book is a valuable resource for medical oncologists, basic and translational biomedical scientists, and trainees in oncology and pharmacology who are interested in learning how to improve their research by using early phase trials. Brings a comprehensive review and recommendations for new clinical trial designs for modern cancer therapeutics Provides the reader with a better understanding on how to design and implement early phase oncology trials Presents a better and updated understanding of the process of developing new treatments for cancer, the exciting scientific advances and how they are informing drug development

Phase I Oncology Drug Development

Phase I Oncology Drug Development
A Book

by Timothy A. Yap,Jordi Rodon,David S. Hong

  • Publisher : Springer Nature
  • Release : 2020-10-18
  • Pages : 352
  • ISBN : 3030476820
  • Language : En, Es, Fr & De
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This book provides a detailed review of how oncology drug development has changed over the past decade, and serves as a comprehensive guide for the practicalities in setting up phase I trials. The book covers strategies to accelerate the development of novel antitumor compounds from the laboratory to clinical trials and beyond through the use of innovative mechanism-of-action pharmacodynamic biomarkers and pharmacokinetic studies. The reader will learn about all aspects of modern phase I trial designs, including the incorporation of precision medicine strategies, and approaches for rational patient allocation to novel anticancer therapies. Circulating biomarkers to assess mechanisms of response and resistance are changing the way we are assessing patient selection and are also covered in this book. The development of the different classes of antitumor agents are discussed, including chemotherapy, molecularly targeted agents, immunotherapies and also radiotherapy. The authors also discuss the lessons that the oncology field has learnt from the development of hematology-oncology drugs and how such strategies can be carried over into therapies for solid tumors. There is a dedicated chapter that covers the specialized statistical approaches necessary for phase I trial designs, including novel Bayesian strategies for dose escalation. This volume is designed to help clinicians better understand phase I clinical trials, but would also be of use to translational researchers (MDs and PhDs), and drug developers from academia and industry interested in cancer drug development. It could also be of use to phase I trial study coordinators, oncology nurses and advanced practice providers. Other health professionals interested in the treatment of cancer will also find this book of great value.

Cancer Clinical Trials

Cancer Clinical Trials
Current and Controversial Issues in Design and Analysis

by Stephen L. George,Xiaofei Wang,Herbert Pang

  • Publisher : CRC Press
  • Release : 2016-08-19
  • Pages : 474
  • ISBN : 1498706908
  • Language : En, Es, Fr & De
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Cancer Clinical Trials: Current and Controversial Issues in Design and Analysis provides statisticians with an understanding of the critical challenges currently encountered in oncology trials. Well-known statisticians from academic institutions, regulatory and government agencies (such as the U.S. FDA and National Cancer Institute), and the pharmaceutical industry share their extensive experiences in cancer clinical trials and present examples taken from actual trials. The book covers topics that are often perplexing and sometimes controversial in cancer clinical trials. Most of the issues addressed are also important for clinical trials in other settings. After discussing general topics, the book focuses on aspects of early and late phase clinical trials. It also explores personalized medicine, including biomarker-based clinical trials, adaptive clinical trial designs, and dynamic treatment regimes.

Oncology Clinical Trials

Oncology Clinical Trials
Successful Design, Conduct, and Analysis

by William Kevin Kelly, DO,Susan Halabi, PhD

  • Publisher : Springer Publishing Company
  • Release : 2018-03-28
  • Pages : 600
  • ISBN : 0826168736
  • Language : En, Es, Fr & De
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The second edition of Oncology Clinical Trials has been thoroughly revised and updated and now contains the latest designs and methods of conducting and analyzing cancer clinical trials in the era of precision medicine with biologic agents—including trials investigating the safety and efficacy of targeted therapies, immunotherapies, and combination therapies as well as novel radiation therapy modalities. Now divided into six sections this revamped book provides the necessary background and expert guidance from the principles governing oncology clinical trials to the innovative statistical design methods permeating the field; from conducting trials in a safe and effective manner, analyzing and interpreting the data, to a forward-looking assessment and discussion of regulatory issues impacting domestic, international, and global clinical trials. Considered by many as the gold standard reference on oncology clinical trials in the field, the second edition continues to provide examples of real-life flaws and real-world examples for how to successfully design, conduct and analyze quality clinical trials and interpret them. With chapters written by oncologists, researchers, biostatisticians, clinical research administrators, and industry and FDA representatives, this volume provides a comprehensive guide in the design, conduct, monitoring, analysis, and reporting of clinical trials in oncology. NEW TO THIS EDITION: Outlines how to design clinical trials with and without biomarker testing—including genomics-based “basket” trials, and adaptive trials for all phases during treatment and quality-of-life trials Includes new chapters on immunotherapy trials, radiation therapy trials, multi-arm trials, meta-analysis and adaptive design, use of genomics, dose modifications and use of ancillary treatments in investigational studies, establishing surrogate endpoints, practical issues with correlative studies, cost-effectiveness analysis, and more Comprehensively covers all regulatory aspects in the pursuit of global oncology trials Digital access to the ebook included

Drug Discovery and Development

Drug Discovery and Development
From Molecules to Medicine

by Omboon Vallisuta,Suleiman Olimat

  • Publisher : BoD – Books on Demand
  • Release : 2015-06-03
  • Pages : 330
  • ISBN : 9535121286
  • Language : En, Es, Fr & De
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It is very important for scientists all over the globe to enhance drug discovery research for better human health. This book demonstrates that various expertise are essential for drug discovery including synthetic or natural drugs, clinical pharmacology, receptor identification, drug metabolism, pharmacodynamic and pharmacokinetic research. The following 5 sections cover diverse chapter topics in drug discovery: Natural Products as Sources of Leading Molecules in Drug Discovery; Oncology and Drug Discovery; Receptors Involvement in Drug Discovery; Management and Development of Drugs against Infectious Diseases; Advanced Methodology.

Biomarkers of the Tumor Microenvironment

Biomarkers of the Tumor Microenvironment
Basic Studies and Practical Applications

by Lars A. Akslen,Randolph S. Watnick

  • Publisher : Springer
  • Release : 2017-08-02
  • Pages : 534
  • ISBN : 331939147X
  • Language : En, Es, Fr & De
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This book reviews different aspects of the cancer microenvironment, and its regulation and importance for tumor progression. Practical applications, in terms of how biomarkers are increasingly included in therapy protocols, will also be discussed. Biomarkers of the Tumour Microenvironment: Basic Studies and Practical Applications is aimed at research pathologists in the cancer field, and also cancer researchers from other backgrounds, especially those using morphology techniques and models focusing on cross-talk between different cell types in tumors.

Journal of the National Cancer Institute

Journal of the National Cancer Institute
JNCI.

by Anonim

  • Publisher : Unknown Publisher
  • Release : 2010-07
  • Pages : 329
  • ISBN : 9876543210XXX
  • Language : En, Es, Fr & De
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Blood and Marrow Transplantation Long Term Management

Blood and Marrow Transplantation Long Term Management
Survivorship after Transplant

by Bipin N. Savani,Andre Tichelli

  • Publisher : John Wiley & Sons
  • Release : 2021-03-02
  • Pages : 464
  • ISBN : 1119612748
  • Language : En, Es, Fr & De
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Hematopoietic cell transplantation (HCT) provides curative therapy for a variety of diseases. Over the past several decades, significant advances have been made in the field of HCT, to the point where HCT has become an integral part of treatment modality for a variety of hematologic malignancies and some nonmalignant diseases. HCT remains an important treatment option for a wide variety of hematologic and nonhematologic disorders, despite recent advances in the field of immunologic therapies. Factors driving this growth include expanded disease indications, greater donor options (expanding unrelated donor registries and haploidentical HCT), and accommodation of older and less fit recipients. The development of less toxic pretransplant conditioning regimens, more effective prophylaxis of graft-versus-host disease (GVHD), improved infection control, and other advances in transplant technology have resulted in a rapidly growing number of transplant recipients surviving long-term free of the disease for which they were transplanted. The changes over decades in the transplant recipient population and in the practice of HCT will have almost inevitably altered the composition of the long-term survivor population over time. Apart from an increasingly older transplant recipient cohort, the pattern of transplant indications has shifted from the 1990s when chronic myeloid leukemia made up a significant proportion of allo-HCT indications. Changes in cell source, donor types, conditioning regimens, GVHD prophylaxis, and supportive care have all occurred, with ongoing reductions in both relapse and non-relapse mortality (NRM) have been demonstrated. These patients have increased risks for a variety of late complications, which can cause morbidity and mortality. Most long-term survivors return to the care of their local hematologists/oncologists or primary care physicians, who may not be familiar with specialized monitoring and management of long complications after HCT for this patient population. As HCT survivorship increases, the focus of care has shifted to the identification and treatment of long-term complications that may affect quality of life and long-term morbidity and mortality. Preventive care as well as early detection and treatments are important aspects to reducing morbidity and mortality in long-term survivors after allo-HCT. This second edition, Blood and Marrow Transplantation Long-Term Management: Survivorship after Transplant, provides up-to-date information about diagnosis, screening, treatment, and long-term surveillance of long-term survivors after HCT.

A National Cancer Clinical Trials System for the 21st Century

A National Cancer Clinical Trials System for the 21st Century
Reinvigorating the NCI Cooperative Group Program

by Institute of Medicine,Board on Health Care Services,Committee on Cancer Clinical Trials and the NCI Cooperative Group Program

  • Publisher : National Academies Press
  • Release : 2010-07-08
  • Pages : 316
  • ISBN : 9780309157872
  • Language : En, Es, Fr & De
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The National Cancer Institute's (NCI) Clinical Trials Cooperative Group Program has played a key role in developing new and improved cancer therapies. However, the program is falling short of its potential, and the IOM recommends changes that aim to transform the Cooperative Group Program into a dynamic system that efficiently responds to emerging scientific knowledge; involves broad cooperation of stakeholders; and leverages evolving technologies to provide high-quality, practice-changing research.

Phase I Cancer Clinical Trials

Phase I Cancer Clinical Trials
A Practical Guide

by Elizabeth A. Eisenhauer,Christopher Twelves,Marc Buyse

  • Publisher : Oxford University Press
  • Release : 2015-03-20
  • Pages : 352
  • ISBN : 0199359024
  • Language : En, Es, Fr & De
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Phase I trials are a critical first step in the study of novel cancer therapeutic approaches. Their primary goals are to identify the recommended dose, schedule and pharmacologic behavior of new agents or new combinations of agents and to describe the adverse effects of treatment. In cancer therapeutics, such studies have particular challenges. Due to the nature of the effects of treatment, most such studies are conducted in patients with advanced malignancy, rather than in healthy volunteers. Further, the endpoints of these trials are usually measures adverse effects rather than molecular target or anti-tumor effects. These factors render the design, conduct, analysis and ethical aspects of phase I cancer trials unique. As the only comprehensive book on this topic, Phase I Cancer Clinical Trials is a useful resource for oncology trainees or specialists interested in understanding cancer drug development. New to this edition are chapters on Phase 0 Trials and Immunotherapeutics, and updated information on the process, pitfalls, and logistics of Phase I Trials

Phase I Cancer Clinical Trials

Phase I Cancer Clinical Trials
A Practical Guide

by Elizabeth A. Eisenhauer,Christopher Twelves,Marc Buyse

  • Publisher : Oxford University Press
  • Release : 2015-03-20
  • Pages : 352
  • ISBN : 0199359024
  • Language : En, Es, Fr & De
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Phase I trials are a critical first step in the study of novel cancer therapeutic approaches. Their primary goals are to identify the recommended dose, schedule and pharmacologic behavior of new agents or new combinations of agents and to describe the adverse effects of treatment. In cancer therapeutics, such studies have particular challenges. Due to the nature of the effects of treatment, most such studies are conducted in patients with advanced malignancy, rather than in healthy volunteers. Further, the endpoints of these trials are usually measures adverse effects rather than molecular target or anti-tumor effects. These factors render the design, conduct, analysis and ethical aspects of phase I cancer trials unique. As the only comprehensive book on this topic, Phase I Cancer Clinical Trials is a useful resource for oncology trainees or specialists interested in understanding cancer drug development. New to this edition are chapters on Phase 0 Trials and Immunotherapeutics, and updated information on the process, pitfalls, and logistics of Phase I Trials

Small Clinical Trials

Small Clinical Trials
Issues and Challenges

by Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Small-Number-Participant Clinical Research Trials

  • Publisher : National Academies Press
  • Release : 2001-01-01
  • Pages : 222
  • ISBN : 9780309171144
  • Language : En, Es, Fr & De
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Clinical trials are used to elucidate the most appropriate preventive, diagnostic, or treatment options for individuals with a given medical condition. Perhaps the most essential feature of a clinical trial is that it aims to use results based on a limited sample of research participants to see if the intervention is safe and effective or if it is comparable to a comparison treatment. Sample size is a crucial component of any clinical trial. A trial with a small number of research participants is more prone to variability and carries a considerable risk of failing to demonstrate the effectiveness of a given intervention when one really is present. This may occur in phase I (safety and pharmacologic profiles), II (pilot efficacy evaluation), and III (extensive assessment of safety and efficacy) trials. Although phase I and II studies may have smaller sample sizes, they usually have adequate statistical power, which is the committee's definition of a "large" trial. Sometimes a trial with eight participants may have adequate statistical power, statistical power being the probability of rejecting the null hypothesis when the hypothesis is false. Small Clinical Trials assesses the current methodologies and the appropriate situations for the conduct of clinical trials with small sample sizes. This report assesses the published literature on various strategies such as (1) meta-analysis to combine disparate information from several studies including Bayesian techniques as in the confidence profile method and (2) other alternatives such as assessing therapeutic results in a single treated population (e.g., astronauts) by sequentially measuring whether the intervention is falling above or below a preestablished probability outcome range and meeting predesigned specifications as opposed to incremental improvement.

Gene Therapy of Cancer

Gene Therapy of Cancer
Translational Approaches from Preclinical Studies to Clinical Implementation

by Stanton L. Gerson,Edmund C. Lattime

  • Publisher : Elsevier
  • Release : 2002-04-04
  • Pages : 534
  • ISBN : 9780080491363
  • Language : En, Es, Fr & De
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The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer. Key Features * Provides in-depth description of targeted systems and treatment strategies * Explains the underlying cancer biology necessary for understanding a given therapeutic approach * Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses * Presents translational focus with emphasis on requirements for clinical implementation * Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference

Oncology Clinical Trials

Oncology Clinical Trials
Successful Design, Conduct and Analysis

by Susan Halabi, PhD,Wm. Kevin Kelly, DO

  • Publisher : Demos Medical Publishing
  • Release : 2009-12-22
  • Pages : 168
  • ISBN : 9781935281764
  • Language : En, Es, Fr & De
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Clinical trials are the engine of progress in the development of new drugs and devices for the detection, monitoring, prevention and treatment of cancer. A well conceived, carefully designed and efficiently conducted clinical trial can produce results that change clinical practice overnight, deliver new oncology drugs and diagnostics to the marketplace, and expand the horizon of contemporary thinking about cancer biology. A poorly done trial does little to advance the field or guide clinical practice, consumes precious clinical and financial resources and challenges the validity of the ethical contract between investigators and the volunteers who willingly give their time and effort to benefit future patients. With chapters written by oncologists, researchers, biostatisticians, clinical research administrators, and industry and FDA representatives, Oncology Clinical Trials, provides a comprehensive guide for both early-career and senior oncology investigators into the successful design, conduct and analysis of an oncology clinical trial. Oncology Clinical Trials covers how to formulate a study question, selecting a study population, study design of Phase I, II, and III trials, toxicity monitoring, data analysis and reporting, use of genomics, cost-effectiveness analysis, systemic review and meta-analysis, and many other issues. Many examples of real-life flaws in clinical trials that have been reported in the literature are included throughout. The book discusses clinical trials from start to finish focusing on real-life examples in the development, design and analysis of clinical trials. Oncology Clinical Trials features: A systematic guide to all aspects of the design, conduct, analysis, and reporting of clinical trials in oncology Contributions from oncologists, researchers, biostatisticians, clinical research administrators, and industry and FDA representatives Hot topics in oncology trials including multi-arm trials, meta-analysis and adaptive design, use of genomics, and cost-effectiveness analysis Real-life examples from reported clinical trials included throughout

Novel Approaches and Strategies for Biologics, Vaccines and Cancer Therapies

Novel Approaches and Strategies for Biologics, Vaccines and Cancer Therapies
A Book

by Manmohan Singh,Maya Salnikova

  • Publisher : Academic Press
  • Release : 2014-12-30
  • Pages : 520
  • ISBN : 012416661X
  • Language : En, Es, Fr & De
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Novel Approaches and Strategies for Biologics, Vaccines and Cancer Therapies takes a look at the current strategies, successes and challenges involved with the development of novel formulations of biologics, vaccines and cancer therapy. This thorough reference on the latest trends in the development of diverse modalities will appeal to a broad community of scientists, students and clinicians. Written by leading authors across academia and industry, this book covers important topics such as unique drug delivery devices, non-parenteral delivery trends, novel approaches to the treatment of cancer, immunotherapy and more. It includes real-world cases and examples which highlight formulations with therapeutic proteins, monoclonal antibodies, peptides and biobetters, as well as cases on novel vaccines formulations including evolving pathogens, novel modalities of vaccines, universal vaccines. This book is a thorough and useful resource on the development of novel biologics, vaccines and cancer therapies. Provides strategies for the development of safe and efficacious novel formulations for various modalities of biologics, vaccines and for cancer therapy Highlights novel cases from current clinical trials as well as marketed products Reviews overall successes and challenges in the development of novel formulations, including new molecular targets for the treatment of diseases, design of target-specific therapies, regulatory considerations, individualized therapies

Cancer Drug Design and Discovery

Cancer Drug Design and Discovery
A Book

by Stephen Neidle

  • Publisher : Elsevier
  • Release : 2011-04-28
  • Pages : 496
  • ISBN : 9780080554952
  • Language : En, Es, Fr & De
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The ultimate source of information on the design of new anticancer agents, emphasizing small molecules, this newest work covers recent notable successes resulting from the human genome and cancer genomics projects. These advances have provided information on targets involved in specific cancers that are leading to effective medicines for at least some of the common solid tumors. Unique sections explain the basic underlying principles of cancer drug development and provide a practical introduction to modern methods of drug design. Appealing to a broad audience, this is an excellent reference for translational researchers interested in cancer biology and medicine as well as students in pharmacy, pharmacology, or medicinal and biological chemistry and clinicians taking oncology options. * Covers both currently available drugs as well as those under development * Provides a clinical perspective on trials of new anticancer agents * Presents drug discovery examples through the use of case histories

Group Sequential Methods with Applications to Clinical Trials

Group Sequential Methods with Applications to Clinical Trials
A Book

by Christopher Jennison,Bruce W. Turnbull

  • Publisher : CRC Press
  • Release : 1999-09-15
  • Pages : 416
  • ISBN : 9781584888581
  • Language : En, Es, Fr & De
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Group sequential methods answer the needs of clinical trial monitoring committees who must assess the data available at an interim analysis. These interim results may provide grounds for terminating the study-effectively reducing costs-or may benefit the general patient population by allowing early dissemination of its findings. Group sequential methods provide a means to balance the ethical and financial advantages of stopping a study early against the risk of an incorrect conclusion. Group Sequential Methods with Applications to Clinical Trials describes group sequential stopping rules designed to reduce average study length and control Type I and II error probabilities. The authors present one-sided and two-sided tests, introduce several families of group sequential tests, and explain how to choose the most appropriate test and interim analysis schedule. Their topics include placebo-controlled randomized trials, bio-equivalence testing, crossover and longitudinal studies, and linear and generalized linear models. Research in group sequential analysis has progressed rapidly over the past 20 years. Group Sequential Methods with Applications to Clinical Trials surveys and extends current methods for planning and conducting interim analyses. It provides straightforward descriptions of group sequential hypothesis tests in a form suited for direct application to a wide variety of clinical trials. Medical statisticians engaged in any investigations planned with interim analyses will find this book a useful and important tool.

Dose Finding by the Continual Reassessment Method

Dose Finding by the Continual Reassessment Method
A Book

by Ying Kuen Cheung

  • Publisher : CRC Press
  • Release : 2011-03-29
  • Pages : 200
  • ISBN : 1420091514
  • Language : En, Es, Fr & De
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As clinicians begin to realize the important role of dose-finding in the drug development process, there is an increasing openness to "novel" methods proposed in the past two decades. In particular, the Continual Reassessment Method (CRM) and its variations have drawn much attention in the medical community, though it has yet to become a commonplace tool. To overcome the status quo in phase I clinical trials, statisticians must be able to design trials using the CRM in a timely and reproducible manner. A self-contained theoretical framework of the CRM for researchers and graduate students who set out to learn and do research in the CRM and dose-finding methods in general, Dose Finding by the Continual Reassessment Method features: Real clinical trial examples that illustrate the methods and techniques throughout the book Detailed calibration techniques that enable biostatisticians to design a CRM in timely manner Limitations of the CRM are outlined to aid in correct use of method This book supplies practical, efficient dose-finding methods based on cutting edge statistical research. More than just a cookbook, it provides full, unified coverage of the CRM in addition to step-by-step guidelines to automation and parameterization of the methods used on a regular basis. A detailed exposition of the calibration of the CRM for applied statisticians working with dose-finding in phase I trials, the book focuses on the R package ‘dfcrm’ for the CRM and its major variants. The author recognizes clinicians’ skepticism of model-based designs, and addresses their concerns that the time, professional, and computational resources necessary for accurate model-based designs can be major bottlenecks to the widespread use of appropriate dose-finding methods in phase I practice. The theoretically- and empirically-based methods in Dose Finding by the Continual Reassessment Method will lessen the statistician’s burden and encourage the continuing development and implementation of model-based dose-finding methods.

Cancer Clinical Trials

Cancer Clinical Trials
A Commonsense Guide to Experimental Cancer Therapies and Clinical Trials

by Tomasz M. Beer,Larry W. Axmaker

  • Publisher : Diamed Llc
  • Release : 2012
  • Pages : 166
  • ISBN : 9780982321973
  • Language : En, Es, Fr & De
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Cancer Clinical Trials is a comprehensive, no-nonsense, and readable guide for anyone who is considering therapeutic options in addition to standard cancer therapy. The book seeks to share knowledge about cancer clinical trials with people living with cancer, their families and loved ones. It will help readers decide if a clinical trial is a good option for them, to choose an appropriate trial, and to navigate through the clinical trial process. It includes lists of questions to ask, things to look for, things to watch out for, and places to look for information. The book begins with a discussion of what cancer is and the many ways in which it is treated – including surgery, chemotherapy, radiation, and new strategies now in use or that will be available in the near future. It considers all aspects of clinical trials, including how they are designed, how to find and evaluate them, how to become a participant, and details of what they will involve in terms of time and commitment. A chapter on available drugs now used to treat cancer will be especially helpful.

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Exploring Novel Clinical Trial Designs for Gene-Based Therapies
Proceedings of a Workshop

by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Regenerative Medicine

  • Publisher : National Academies Press
  • Release : 2020-08-27
  • Pages : 126
  • ISBN : 0309672988
  • Language : En, Es, Fr & De
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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.