Estimands Estimators And Sensitivity Analysis In Clinical Trials
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Estimands Estimators And Sensitivity Analysis In Clinical Trials
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Author : Craig Mallinckrodt
language : en
Publisher: CRC Press
Release Date : 2019-12-23
Estimands Estimators And Sensitivity Analysis In Clinical Trials written by Craig Mallinckrodt and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019-12-23 with Medical categories.
The concepts of estimands, analyses (estimators), and sensitivity are interrelated. Therefore, great need exists for an integrated approach to these topics. This book acts as a practical guide to developing and implementing statistical analysis plans by explaining fundamental concepts using accessible language, providing technical details, real-world examples, and SAS and R code to implement analyses. The updated ICH guideline raises new analytic and cross-functional challenges for statisticians. Gaps between different communities have come to surface, such as between causal inference and clinical trialists, as well as among clinicians, statisticians, and regulators when it comes to communicating decision-making objectives, assumptions, and interpretations of evidence. This book lays out a path toward bridging some of these gaps. It offers A common language and unifying framework along with the technical details and practical guidance to help statisticians meet the challenges A thorough treatment of intercurrent events (ICEs), i.e., postrandomization events that confound interpretation of outcomes and five strategies for ICEs in ICH E9 (R1) Details on how estimands, integrated into a principled study development process, lay a foundation for coherent specification of trial design, conduct, and analysis needed to overcome the issues caused by ICEs: A perspective on the role of the intention-to-treat principle Examples and case studies from various areas Example code in SAS and R A connection with causal inference Implications and methods for analysis of longitudinal trials with missing data Together, the authors have offered the readers their ample expertise in clinical trial design and analysis, from an industrial and academic perspective.
Statistical Thinking For Non Statisticians In Drug Regulation
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Author : Richard Kay
language : en
Publisher: John Wiley & Sons
Release Date : 2022-11-29
Statistical Thinking For Non Statisticians In Drug Regulation written by Richard Kay and has been published by John Wiley & Sons this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-11-29 with Medical categories.
STATISTICAL THINKING FOR NON-STATISTICIANS IN DRUG REGULATION Statistical methods in the pharmaceutical industry are accepted as a key element in the design and analysis of clinical studies. Increasingly, the medical and scientific community are aligning with the regulatory authorities and recognizing that correct statistical methodology is essential as the basis for valid conclusions. In order for those correct and robust methods to be successfully employed there needs to be effective communication across disciplines at all stages of the planning, conducting, analyzing and reporting of clinical studies associated with the development and evaluation of new drugs and devices. Statistical Thinking for Non-Statisticians in Drug Regulation provides a comprehensive in-depth guide to statistical methodology for pharmaceutical industry professionals, including physicians, investigators, medical science liaisons, clinical research scientists, medical writers, regulatory personnel, statistical programmers, senior data managers and those working in pharmacovigilance. The author’s years of experience and up-to-date familiarity with pharmaceutical regulations and statistical practice within the wider clinical community make this an essential guide for the those working in and with the industry. The third edition of Statistical Thinking for Non-Statisticians in Drug Regulation includes: A detailed new chapter on Estimands in line with the 2019 Addendum to ICH E9 Major new sections on topics including Combining Hierarchical Testing and Alpha Adjustment, Biosimilars, Restricted Mean Survival Time, Composite Endpoints and Cumulative Incidence Functions, Adjusting for Cross-Over in Oncology, Inverse Propensity Score Weighting, and Network Meta-Analysis Updated coverage of many existing topics to reflect new and revised guidance from regulatory authorities and author experience Statistical Thinking for Non-Statisticians in Drug Regulation is a valuable guide for pharmaceutical and medical device industry professionals, as well as statisticians joining the pharmaceutical industry and students and teachers of drug development.
Causal Inference In Pharmaceutical Statistics
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Author : Yixin Fang
language : en
Publisher: CRC Press
Release Date : 2024-06-24
Causal Inference In Pharmaceutical Statistics written by Yixin Fang and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2024-06-24 with Mathematics categories.
Causal Inference in Pharmaceutical Statistics introduces the basic concepts and fundamental methods of causal inference relevant to pharmaceutical statistics. This book covers causal thinking for different types of commonly used study designs in the pharmaceutical industry, including but not limited to randomized controlled clinical trials, longitudinal studies, singlearm clinical trials with external controls, and real-world evidence studies. The book starts with the central questions in drug development and licensing, takes the reader through the basic concepts and methods via different study types and through different stages, and concludes with a roadmap to conduct causal inference in clinical studies. The book is intended for clinical statisticians and epidemiologists working in the pharmaceutical industry. It will also be useful to graduate students in statistics, biostatistics, and data science looking to pursue a career in the pharmaceutical industry. Key Features: Causal inference book for clinical statisticians in the pharmaceutical industry Introductory level on the most important concepts and methods Align with FDA and ICH guidance documents Across different stages of clinical studies: plan, design, conduct, analysis, and interpretation Cover a variety of commonly used study designs
Digital Therapeutics
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Author : Oleksandr Sverdlov
language : en
Publisher: CRC Press
Release Date : 2022-12-06
Digital Therapeutics written by Oleksandr Sverdlov and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-12-06 with Mathematics categories.
One of the hallmarks of the 21st century medicine is the emergence of digital therapeutics (DTx)—evidence-based, clinically validated digital technologies to prevent, diagnose, treat, and manage various diseases and medical conditions. DTx solutions have been gaining interest from patients, investors, healthcare providers, health authorities, and other stakeholders because of the potential of DTx to deliver equitable, massively scalable, personalized and transformative treatments for different unmet medical needs. Digital Therapeutics: Scientific, Statistical, Clinical, and Regulatory Aspects is an unparalleled summary of the current scientific, statistical, developmental, and regulatory aspects of DTx which is poised to become the fastest growing area of the biopharmaceutical and digital medicine product development. This edited volume intends to provide a systematic exposition to digital therapeutics through 19 peer-reviewed chapters written by subject matter experts in this emerging field. This edited volume is an invaluable resource for business leaders and researchers working in public health, healthcare, digital health, information technology, and biopharmaceutical industries. It will be also useful for regulatory scientists involved in the review of DTx products, and for faculty and students involved in an interdisciplinary research on digital health and digital medicine. Key Features: Provides the taxonomy of the concepts and a navigation tool for the field of DTx. Covers important strategic aspects of the DTx industry, thereby helping investors, developers, and regulators gain a better appreciation of the potential value of DTx. Expounds on many existing and emerging state-of-the art scientific and technological tools, as well as data privacy, ethical and regulatory considerations for DTx product development. Presents several case studies of successful development of some of the most remarkable DTx products. Provides some perspectives and forward-looking statements on the future of digital medicine.
Report Of The Technical Consultation On Innovative Clinical Trial Designs For Development Of New Tb Treatments
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Author :
language : en
Publisher: World Health Organization
Release Date : 2021-07-28
Report Of The Technical Consultation On Innovative Clinical Trial Designs For Development Of New Tb Treatments written by and has been published by World Health Organization this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-07-28 with Medical categories.
The Global Tuberculosis Programme of the World Health Organization (WHO) convened a virtual technical consultation on “Innovative Clinical Trial Designs for Development of New TB Treatments” on 5 days between 20 August and 13 October 2021. The main objective of the meeting was to establish evidence-based approaches to trial designs and use of data to inform policy guidance of new regimens for the treatment of TB with the view to accelerate future regimen development. The consultation brought together researchers, academics, technical partners, TB drugs and regimens developers including pharmaceutical industry, trialists, regulators, guideline developers, programme managers, nongovernmental organizations and civil society.
Multiple Imputation And Its Application
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Author : James R. Carpenter
language : en
Publisher: John Wiley & Sons
Release Date : 2023-07-20
Multiple Imputation And Its Application written by James R. Carpenter and has been published by John Wiley & Sons this book supported file pdf, txt, epub, kindle and other format this book has been release on 2023-07-20 with Medical categories.
Multiple Imputation and its Application The most up-to-date edition of a bestselling guide to analyzing partially observed data In this comprehensively revised Second Edition of Multiple Imputation and its Application, a team of distinguished statisticians delivers an overview of the issues raised by missing data, the rationale for multiple imputation as a solution, and the practicalities of applying it in a multitude of settings. With an accessible and carefully structured presentation aimed at quantitative researchers, Multiple Imputation and its Application is illustrated with a range of examples and offers key mathematical details. The book includes a wide range of theoretical and computer-based exercises, tested in the classroom, which are especially useful for users of R or Stata. Readers will find: A comprehensive overview of one of the most effective and popular methodologies for dealing with incomplete data sets Careful discussion of key concepts A range of examples illustrating the key ideas Practical advice on using multiple imputation Exercises and examples designed for use in the classroom and/or private study Written for applied researchers looking to use multiple imputation with confidence, and for methods researchers seeking an accessible overview of the topic, Multiple Imputation and its Application will also earn a place in the libraries of graduate students undertaking quantitative analyses.
Statistical Design Monitoring And Analysis Of Clinical Trials
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Author : Weichung Joe Shih
language : en
Publisher: CRC Press
Release Date : 2021-10-26
Statistical Design Monitoring And Analysis Of Clinical Trials written by Weichung Joe Shih and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-10-26 with Medical categories.
Statistical Design, Monitoring, and Analysis of Clinical Trials, Second Edition concentrates on the biostatistics component of clinical trials. This new edition is updated throughout and includes five new chapters. Developed from the authors’ courses taught to public health and medical students, residents, and fellows during the past 20 years, the text shows how biostatistics in clinical trials is an integration of many fundamental scientific principles and statistical methods. The book begins with ethical and safety principles, core trial design concepts, the principles and methods of sample size and power calculation, and analysis of covariance and stratified analysis. It then focuses on sequential designs and methods for two-stage Phase II cancer trials to Phase III group sequential trials, covering monitoring safety, futility, and efficacy. The authors also discuss the development of sample size reestimation and adaptive group sequential procedures, phase 2/3 seamless design and trials with predictive biomarkers, exploit multiple testing procedures, and explain the concept of estimand, intercurrent events, and different missing data processes, and describe how to analyze incomplete data by proper multiple imputations. This text reflects the academic research, commercial development, and public health aspects of clinical trials. It gives students and practitioners a multidisciplinary understanding of the concepts and techniques involved in designing, monitoring, and analyzing various types of trials. The book’s balanced set of homework assignments and in-class exercises are appropriate for students and researchers in (bio)statistics, epidemiology, medicine, pharmacy, and public health.
Innovative Statistics In Regulatory Science
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Author : Shein-Chung Chow
language : en
Publisher: CRC Press
Release Date : 2019-11-14
Innovative Statistics In Regulatory Science written by Shein-Chung Chow and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019-11-14 with Mathematics categories.
Statistical methods that are commonly used in the review and approval process of regulatory submissions are usually referred to as statistics in regulatory science or regulatory statistics. In a broader sense, statistics in regulatory science can be defined as valid statistics that are employed in the review and approval process of regulatory submissions of pharmaceutical products. In addition, statistics in regulatory science are involved with the development of regulatory policy, guidance, and regulatory critical clinical initiatives related research. This book is devoted to the discussion of statistics in regulatory science for pharmaceutical development. It covers practical issues that are commonly encountered in regulatory science of pharmaceutical research and development including topics related to research activities, review of regulatory submissions, recent critical clinical initiatives, and policy/guidance development in regulatory science. Devoted entirely to discussing statistics in regulatory science for pharmaceutical development. Reviews critical issues (e.g., endpoint/margin selection and complex innovative design such as adaptive trial design) in the pharmaceutical development and regulatory approval process. Clarifies controversial statistical issues (e.g., hypothesis testing versus confidence interval approach, missing data/estimands, multiplicity, and Bayesian design and approach) in review/approval of regulatory submissions. Proposes innovative thinking regarding study designs and statistical methods (e.g., n-of-1 trial design, adaptive trial design, and probability monitoring procedure for sample size) for rare disease drug development. Provides insight regarding current regulatory clinical initiatives (e.g., precision/personalized medicine, biomarker-driven target clinical trials, model informed drug development, big data analytics, and real world data/evidence). This book provides key statistical concepts, innovative designs, and analysis methods that are useful in regulatory science. Also included are some practical, challenging, and controversial issues that are commonly seen in the review and approval process of regulatory submissions. About the author Shein-Chung Chow, Ph.D. is currently a Professor at Duke University School of Medicine, Durham, NC. He was previously the Associate Director at the Office of Biostatistics, Center for Drug Evaluation and Research, United States Food and Drug Administration (FDA). Dr. Chow has also held various positions in the pharmaceutical industry such as Vice President at Millennium, Cambridge, MA, Executive Director at Covance, Princeton, NJ, and Director and Department Head at Bristol-Myers Squibb, Plainsboro, NJ. He was elected Fellow of the American Statistical Association and an elected member of the ISI (International Statistical Institute). Dr. Chow is Editor-in-Chief of the Journal of Biopharmaceutical Statistics and Biostatistics Book Series, Chapman and Hall/CRC Press, Taylor & Francis, New York. Dr. Chow is the author or co-author of over 300 methodology papers and 30 books.
Handbook Of Quality Of Life In Cancer
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Author : Angelos P. Kassianos
language : en
Publisher: Springer Nature
Release Date : 2022-03-25
Handbook Of Quality Of Life In Cancer written by Angelos P. Kassianos and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-03-25 with Social Science categories.
Quality of life is an important outcome when treating a cancer patient. Research is vast on the role of quality of life on patients' general wellbeing, responsiveness to treatment, and even mortality. On the other hand, there are several methodological considerations when planning to measure and assess quality of life in cancer patients. This handbook – with authorship that is diverse in terms of perspectives, countries, and fields – aims to fill a gap in the available literature and responds to a number of questions in its 26 chapters: What is quality of life and health-related quality of life and why are they important? How is quality of life assessed? What are the theoretical and methodological considerations when using quality of life outcomes in cancer research? How is quality of life useful in routine clinical care? How is quality of life impacting different cancer populations in terms of site of the cancer, age, gender, and context? Handbook of Quality of Life in Cancer is a learning and consulting tool that can be used by a diverse audience. It is an essential resource for researchers who wish to use quality of life assessment tools in clinical trials or other types of studies; clinicians who want to develop their understanding of how they can utilize quality of life and how it is important for the patients they care for; and commissioners who wish to see why quality of life may impact population health and health system costs. Students in diverse fields of study (medicine, nursing, psychology, social work, medical sociology, population health, epidemiology, and medical statistics, among others) also would benefit from using the handbook for their studies and for their continuing professional development.
Real World Evidence In Medical Product Development
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Author : Weili He
language : en
Publisher: Springer Nature
Release Date : 2023-05-11
Real World Evidence In Medical Product Development written by Weili He and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2023-05-11 with Mathematics categories.
This book provides state-of-art statistical methodologies, practical considerations from regulators and sponsors, logistics, and real use cases for practitioners for the uptake of RWE/D. Randomized clinical trials have been the gold standard for the evaluation of efficacy and safety of medical products. However, the cost, duration, practicality, and limited generalizability have incentivized many to look for alternative ways to optimize drug development. This book provides a comprehensive list of topics together to include all aspects with the uptake of RWE/D, including, but not limited to, applications in regulatory and non-regulatory settings, causal inference methodologies, organization and infrastructure considerations, logistic challenges, and practical use cases.