Case Studies In Innovative Clinical Trials

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Case Studies in Innovative Clinical Trials

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

Author : Binbing Yu,Kristine Broglio
Publisher : Unknown
Page : 0 pages
File Size : 54,8 Mb
Release : 2023
Category : MEDICAL
ISBN : 1003288642

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

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

Innovative Strategies, Statistical Solutions and Simulations for Modern Clinical Trials

Mark Chang,John Balser,Jim Roach,Robin Bliss
Innovative Strategies Statistical Solutions and Simulations for Modern Clinical Trials Book PDF

Author : Mark Chang,John Balser,Jim Roach,Robin Bliss
Publisher : CRC Press
Page : 218 pages
File Size : 48,9 Mb
Release : 2019-03-20
Category : Mathematics
ISBN : 9781351214520

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Innovative Strategies, Statistical Solutions and Simulations for Modern Clinical Trials by Mark Chang,John Balser,Jim Roach,Robin Bliss Pdf

"This is truly an outstanding book. [It] brings together all of the latest research in clinical trials methodology and how it can be applied to drug development.... Chang et al provide applications to industry-supported trials. This will allow statisticians in the industry community to take these methods seriously." Jay Herson, Johns Hopkins University The pharmaceutical industry's approach to drug discovery and development has rapidly transformed in the last decade from the more traditional Research and Development (R & D) approach to a more innovative approach in which strategies are employed to compress and optimize the clinical development plan and associated timelines. However, these strategies are generally being considered on an individual trial basis and not as part of a fully integrated overall development program. Such optimization at the trial level is somewhat near-sighted and does not ensure cost, time, or development efficiency of the overall program. This book seeks to address this imbalance by establishing a statistical framework for overall/global clinical development optimization and providing tactics and techniques to support such optimization, including clinical trial simulations. Provides a statistical framework for achieve global optimization in each phase of the drug development process. Describes specific techniques to support optimization including adaptive designs, precision medicine, survival-endpoints, dose finding and multiple testing. Gives practical approaches to handling missing data in clinical trials using SAS. Looks at key controversial issues from both a clinical and statistical perspective. Presents a generous number of case studies from multiple therapeutic areas that help motivate and illustrate the statistical methods introduced in the book. Puts great emphasis on software implementation of the statistical methods with multiple examples of software code (both SAS and R). It is important for statisticians to possess a deep knowledge of the drug development process beyond statistical considerations. For these reasons, this book incorporates both statistical and "clinical/medical" perspectives.

Randomised Clinical Trials

David Machin,Peter M. Fayers,Bee Choo Tai
Randomised Clinical Trials Book PDF

Author : David Machin,Peter M. Fayers,Bee Choo Tai
Publisher : John Wiley & Sons
Page : 86 pages
File Size : 48,8 Mb
Release : 2021-07-26
Category : Medical
ISBN : 9781119524649

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Randomised Clinical Trials by David Machin,Peter M. Fayers,Bee Choo Tai Pdf

Randomised Clinical Trials: Design, Practice and Reporting provides a detailed overview of the methodology for conducting clinical trials, including developing protocols, data capture, randomisation, analysis and reporting. Assuming no prior background, this user-friendly resource describes the statistical, regulatory, and practical components required for conducting randomised clinical trials. Numerous examples and case studies from industry, academia, and the research literature help readers understand each stage of the clinical trial process. This second edition contains extensively revised material throughout, including new chapters covering designs for repeated measures, non-inferiority, cluster and stepped wedge trials. Other new chapters describe data and safety monitoring, biomarker studies, and feasibility studies. Updated and expanded sections discuss situations where multiple organs, different body locations or competing risks are involved, subgroup analysis, and multiple outcomes. Written by an author team with extensive experience in conducting clinical trials, this book: Provides comprehensive coverage of randomised clinical trials, ranging from basic to advanced Features several new chapters, updated case studies and examples, and references to changes in regulations Explains basic randomised trials, including the parallel two-group controlled trial with a single outcome measure Covers paired trial designs and trials with more than two interventions Includes a chapter on miscellaneous topics such as adaptive designs, large simple trials, Bayesian methods for very small trials, alpha-spending functions and the predictive probability test Randomised Clinical Trials is essential reading for clinicians, nurses, data managers, and medical statisticians involved in clinical trials, and for health practitioners responsible for direct patient care in a clinical trial setting.

Case Studies in Innovative Clinical Trials

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

Author : Kristine Broglio,Binbing Yu
Publisher : CRC Press
Page : 303 pages
File Size : 55,7 Mb
Release : 2023-11-27
Category : Mathematics
ISBN : 9781000987218

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

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

Clinical Evaluation of Medical Devices

Karen M. Becker,John J. Whyte
Clinical Evaluation of Medical Devices Book PDF

Author : Karen M. Becker,John J. Whyte
Publisher : Springer Science & Business Media
Page : 360 pages
File Size : 55,7 Mb
Release : 2007-11-05
Category : Technology & Engineering
ISBN : 9781597450041

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Clinical Evaluation of Medical Devices by Karen M. Becker,John J. Whyte Pdf

The original edition of this text, Clinical Evaluation of Medical Devices: Principles and Case Studies, provided the first overview of key pr- ciples and approaches to medical device clinical trials, illustrated with a series of detailed, real-world case studies. The book is designed as a resource for clinical professionals and regulatory specialists working in the field of new medical device development and marketing. Since the first edition of this text was published in 1997, the rapid pace of inno- tion in health care technologies continues to yield exciting and important new products. The regulatory landscape has also evolved, reflecting some of the changes and needs within the medical device industry. The purpose of Clinical Evaluation of Medical Devices: Principles and Case Studies, Second Edition is to provide an updated and expanded presentation of the scientific methods and regulatory requirements applied to the study of new significant risk medical devices. The text now includes (1) new information on the requirements and process for gaining reimbursement of new products from Medicare and private insurers, with case studies of research specifically designed for this p- pose as well as health care technology assessment methods; (2) infor- tion on new statistical methodologies applied to medical device trials; and (3) all new case studies, including examples of combination pr- ucts, three-phase development models (i. e. , feasibility, FDA approval, Medicare reimbursement), and novel study designs.

Clinical Research

John G. Brock-Utne
Clinical Research Book PDF

Author : John G. Brock-Utne
Publisher : Springer
Page : 0 pages
File Size : 41,6 Mb
Release : 2015-07-24
Category : Medical
ISBN : 1493925156

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Clinical Research by John G. Brock-Utne Pdf

This book provides insights into how to be a productive clinical researcher via real-life case examples of successful clinical research -- and also clinical research gone awry. Through these examples of success and failure, the book develops a blueprint for building a career in clinical research. Future medical practice depends on the quality of the clinical trials to which drugs, devices, and treatment procedures are subjected today. However, clinical trials are not easy to do, and many physicians and health care providers who attempt clinical research struggle in this endeavor, primarily because of lack of instruction. Clinical Research aims to fill the gap between training and research through case studies of a long-time clinical researcher’s rich and varied experiences.

Global Clinical Trials

Richard Chin,Menghis Bairu
Global Clinical Trials Book PDF

Author : Richard Chin,Menghis Bairu
Publisher : Academic Press
Page : 522 pages
File Size : 49,9 Mb
Release : 2011-05-06
Category : Science
ISBN : 012381538X

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Global Clinical Trials by Richard Chin,Menghis Bairu Pdf

This book will explore the great opportunities and challenges which exist in conducting clinical trials in developing countries. By exploring the various regulations specific to the major players and providing insight into the logistical challenges including language barriers, this book provides a working tool for clinical researchers and administrators to navigate the intricacies of clinical trials in developing countries. Important topics such as ethical issues will be handled very carefully to highlight the significant differences of conducting this work in various jurisdictions. Overall, it will present a clear and comprehensive guide to the ins-and-outs of clinical trials in various countries to assist in design, development, and effectiveness of these trials. Contributors include high-profile, respected figures who have paved the way for clinical trials in developing countries Provides hands-on tools for regulatory and legal requirements and qualification, design, management, and reporting Case studies outline successes, failures, lessons learned and prospects for future collaboration Includes country-specific guidelines for the most utilized countries Foreword by David Feigel, former Head of CDRH at FDA

Critical Thinking in Clinical Research

Felipe Fregni,Ben M. W. Illigens
Critical Thinking in Clinical Research Book PDF

Author : Felipe Fregni,Ben M. W. Illigens
Publisher : Oxford University Press
Page : 537 pages
File Size : 41,8 Mb
Release : 2018
Category : Medical
ISBN : 9780199324491

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Critical Thinking in Clinical Research by Felipe Fregni,Ben M. W. Illigens Pdf

Critical Thinking in Clinical Research explains the fundamentals of clinical research in a case-based approach. The core concept is to combine a clear and concise transfer of information and knowledge with an engagement of the reader to develop a mastery of learning and critical thinking skills. The book addresses the main concepts of clinical research, basics of biostatistics, advanced topics in applied biostatistics, and practical aspects of clinical research, with emphasis on clinical relevance across all medical specialties.

Introduction to Adaptive Trial Designs and Master Protocols

Jay J. H. Park,Edward J. Mills,J. Kyle Wathen
Introduction to Adaptive Trial Designs and Master Protocols Book PDF

Author : Jay J. H. Park,Edward J. Mills,J. Kyle Wathen
Publisher : Cambridge University Press
Page : 189 pages
File Size : 48,7 Mb
Release : 2023-03-31
Category : Medical
ISBN : 9781108934442

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Introduction to Adaptive Trial Designs and Master Protocols by Jay J. H. Park,Edward J. Mills,J. Kyle Wathen Pdf

This practical high-level guidebook offers an in-depth understanding of the newly emerging clinical trial designs in adaptive trial designs and master protocols. Both concise and readable without shying away from technical discussion, the book introduces the most innovative approaches in clinical trial research such as adaptive trial designs, master protocols, platform trial, basket trial, and umbrella trial designs. Featuring a revisionist history of clinical research before moving on to case-study based discussion and practical considerations from collective experience. The book enables readers to understand the strengths and limitations of these novel designs as well as their application to individual areas of research and clinical practice. Supplemented by real-world examples from the recent developments in medical research efficiency instigated by both personalized medicine and high-profile diseases like COVID-19 and cancer. The first book of its kind, it is the go-to resource for medical students and researchers working in clinical trial research.

Innovation in Clinical Trial Methodologies

Peter Schueler
Innovation in Clinical Trial Methodologies Book PDF

Author : Peter Schueler
Publisher : Academic Press
Page : 202 pages
File Size : 55,8 Mb
Release : 2020-11-01
Category : Medical
ISBN : 9780323860062

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Innovation in Clinical Trial Methodologies by Peter Schueler Pdf

Innovation in Clinical Trial Methodologies: Lessons Learned during the Corona Pandemic presents a selection of updated chapters from Re-Engineering Clinical Trials that feature innovative options and methods in clinical trials. The Coronavirus pandemic is an accelerator for digitalization in many industries, including clinical trials. This book considers best practices, alternative study concepts requiring fewer patients, studies with less patient interaction, the design of "virtualized" protocols, and moving from data to decisions. This book will be helpful to pharmacologists, physicians and clinical researchers involved in the process of clinical development and clinical trial design. Considers multiple digital and virtual strategies Explores best practices, including the use of reduced patient involvement Brings together expert, trusted information to increase the efficiency and effectiveness of clinical trials

Analysis of Clinical Trials Using SAS

Alex Dmitrienko,Gary G. Koch
Analysis of Clinical Trials Using SAS Book PDF

Author : Alex Dmitrienko,Gary G. Koch
Publisher : SAS Institute
Page : 455 pages
File Size : 54,9 Mb
Release : 2017-07-17
Category : Computers
ISBN : 9781635261448

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Analysis of Clinical Trials Using SAS by Alex Dmitrienko,Gary G. Koch Pdf

Analysis of Clinical Trials Using SAS®: A Practical Guide, Second Edition bridges the gap between modern statistical methodology and real-world clinical trial applications. Tutorial material and step-by-step instructions illustrated with examples from actual trials serve to define relevant statistical approaches, describe their clinical trial applications, and implement the approaches rapidly and efficiently using the power of SAS. Topics reflect the International Conference on Harmonization (ICH) guidelines for the pharmaceutical industry and address important statistical problems encountered in clinical trials. Commonly used methods are covered, including dose-escalation and dose-finding methods that are applied in Phase I and Phase II clinical trials, as well as important trial designs and analysis strategies that are employed in Phase II and Phase III clinical trials, such as multiplicity adjustment, data monitoring, and methods for handling incomplete data. This book also features recommendations from clinical trial experts and a discussion of relevant regulatory guidelines. This new edition includes more examples and case studies, new approaches for addressing statistical problems, and the following new technological updates: SAS procedures used in group sequential trials (PROC SEQDESIGN and PROC SEQTEST) SAS procedures used in repeated measures analysis (PROC GLIMMIX and PROC GEE) macros for implementing a broad range of randomization-based methods in clinical trials, performing complex multiplicity adjustments, and investigating the design and analysis of early phase trials (Phase I dose-escalation trials and Phase II dose-finding trials) Clinical statisticians, research scientists, and graduate students in biostatistics will greatly benefit from the decades of clinical research experience and the ready-to-use SAS macros compiled in this book.

Innovative Methods for Rare Disease Drug Development

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

Author : Shein-Chung Chow
Publisher : CRC Press
Page : 321 pages
File Size : 51,5 Mb
Release : 2020-11-11
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.

Randomized Clinical Trials

David Machin,Peter M. Fayers
Randomized Clinical Trials Book PDF

Author : David Machin,Peter M. Fayers
Publisher : John Wiley & Sons
Page : 375 pages
File Size : 48,7 Mb
Release : 2010-05-20
Category : Medical
ISBN : 9780470319222

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Randomized Clinical Trials by David Machin,Peter M. Fayers Pdf

Using examples and case studies from industry, academia and research literature, Randomized Clinical Trials provides a detailed overview of the key issues involved in designing, conducting, analysing and reporting randomized clinical trials. It examines the methodology for conducting Phase III clinical trials, developing the protocols, the practice for capturing, measuring, and analysing the resulting clinical data and their subsequent reporting. Randomized clinical trials are the principal method for determining the relative efficacy and safety of alternative treatments, interventions or medical devices. They are conducted by groups comprising one or more of pharmaceutical and allied health-care organisations, academic institutions, and charity supported research groups. In many cases such trials provide the key evidence necessary for the regulatory approval of a new product for future patient use. Randomized Clinical Trials provides comprehensive coverage of such trials, ranging from elementary to advanced level. Written by authors with considerable experience of clinical trials, Randomized Clinical Trials is an authoritative guide for clinicians, nurses, data managers and medical statisticians involved in clinical trials research and for health care professionals directly involved in patient care in a clinical trial context.

Textbook of Clinical Trials in Oncology

Susan Halabi,Stefan Michiels
Textbook of Clinical Trials in Oncology Book PDF

Author : Susan Halabi,Stefan Michiels
Publisher : CRC Press
Page : 701 pages
File Size : 49,5 Mb
Release : 2019-04-24
Category : Medical
ISBN : 9781351620963

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Textbook of Clinical Trials in Oncology by Susan Halabi,Stefan Michiels Pdf

There is an increasing need for educational resources for statisticians and investigators. Reflecting this, the goal of this book is to provide readers with a sound foundation in the statistical design, conduct, and analysis of clinical trials. Furthermore, it is intended as a guide for statisticians and investigators with minimal clinical trial experience who are interested in pursuing a career in this area. The advancement in genetic and molecular technologies have revolutionized drug development. In recent years, clinical trials have become increasingly sophisticated as they incorporate genomic studies, and efficient designs (such as basket and umbrella trials) have permeated the field. This book offers the requisite background and expert guidance for the innovative statistical design and analysis of clinical trials in oncology. Key Features: Cutting-edge topics with appropriate technical background Built around case studies which give the work a "hands-on" approach Real examples of flaws in previously reported clinical trials and how to avoid them Access to statistical code on the book’s website Chapters written by internationally recognized statisticians from academia and pharmaceutical companies Carefully edited to ensure consistency in style, level, and approach Topics covered include innovating phase I and II designs, trials in immune-oncology and rare diseases, among many others

Access to Non-Summary Clinical Trial Data for Research Purposes Under EU Law

Daria Kim
Access to Non Summary Clinical Trial Data for Research Purposes Under EU Law Book PDF

Author : Daria Kim
Publisher : Springer Nature
Page : 310 pages
File Size : 47,6 Mb
Release : 2021-10-19
Category : Law
ISBN : 9783030867782

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Access to Non-Summary Clinical Trial Data for Research Purposes Under EU Law by Daria Kim Pdf

This book draws a unique perspective on the regulation of access to clinical trial data as a case on research and knowledge externalities. Notwithstanding numerous potential benefits for medical research and public health, many jurisdictions have struggled to ensure access to clinical trial data, even at the level of the trial results. Pro-access policy initiatives have been strongly opposed by research-based drug companies arguing that mandatory data disclosure impedes their innovation incentives. Conventionally, access to test data has been approached from the perspective of transparency and research ethics. The book offers a complementary view and considers access to individual patient-level trial data for exploratory analysis as a matter of research and innovation policy. Such approach appears to be especially relevant in the data-driven economy where digital data constitutes a valuable economic resource. The study seeks to define how the rules of access to clinical trial data should be designed to reconcile the policy objectives of leveraging the research potential of data through secondary analysis, on the one hand, and protecting economic incentives of research-based drug companies, on the other hand. Overall, it is argued that the mainstream innovation-based justification for exclusive control over the outcomes of research and development can hardly rationalise trial sponsors’ control over primary data from trials. Instead, access to such data and its robust analysis should be prioritised.