Real World Evidence In Drug Development And Evaluation

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Real-World Evidence in Drug Development and Evaluation

Harry Yang,Binbing Yu
Real World Evidence in Drug Development and Evaluation Book PDF

Author : Harry Yang,Binbing Yu
Publisher : CRC Press
Page : 191 pages
File Size : 51,7 Mb
Release : 2021-01-11
Category : Mathematics
ISBN : 9780429676826

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Real-World Evidence in Drug Development and Evaluation by Harry Yang,Binbing Yu Pdf

Real-world evidence (RWE) has been at the forefront of pharmaceutical innovations. It plays an important role in transforming drug development from a process aimed at meeting regulatory expectations to an operating model that leverages data from disparate sources to aid business, regulatory, and healthcare decision making. Despite its many benefits, there is no single book systematically covering the latest development in the field. Written specifically for pharmaceutical practitioners, Real-World Evidence in Drug Development and Evaluation, presents a wide range of RWE applications throughout the lifecycle of drug product development. With contributions from experienced researchers in the pharmaceutical industry, the book discusses at length RWE opportunities, challenges, and solutions. Features Provides the first book and a single source of information on RWE in drug development Covers a broad array of topics on outcomes- and value-based RWE assessments Demonstrates proper Bayesian application and causal inference for real-world data (RWD) Presents real-world use cases to illustrate the use of advanced analytics and statistical methods to generate insights Offers a balanced discussion of practical RWE issues at hand and technical solutions suitable for practitioners with limited data science expertise

Real-World Evidence in Drug Development and Evaluation

Harry Yang,Binbing Yu
Real World Evidence in Drug Development and Evaluation Book PDF

Author : Harry Yang,Binbing Yu
Publisher : CRC Press
Page : 177 pages
File Size : 48,8 Mb
Release : 2021-01-11
Category : Mathematics
ISBN : 9780429676819

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Real-World Evidence in Drug Development and Evaluation by Harry Yang,Binbing Yu Pdf

Real-world evidence (RWE) has been at the forefront of pharmaceutical innovations. It plays an important role in transforming drug development from a process aimed at meeting regulatory expectations to an operating model that leverages data from disparate sources to aid business, regulatory, and healthcare decision making. Despite its many benefits, there is no single book systematically covering the latest development in the field. Written specifically for pharmaceutical practitioners, Real-World Evidence in Drug Development and Evaluation, presents a wide range of RWE applications throughout the lifecycle of drug product development. With contributions from experienced researchers in the pharmaceutical industry, the book discusses at length RWE opportunities, challenges, and solutions. Features Provides the first book and a single source of information on RWE in drug development Covers a broad array of topics on outcomes- and value-based RWE assessments Demonstrates proper Bayesian application and causal inference for real-world data (RWD) Presents real-world use cases to illustrate the use of advanced analytics and statistical methods to generate insights Offers a balanced discussion of practical RWE issues at hand and technical solutions suitable for practitioners with limited data science expertise

Real-World Evidence in Drug Development and Evaluation

HARRY. YANG
Real World Evidence in Drug Development and Evaluation Book PDF

Author : HARRY. YANG
Publisher : Chapman & Hall/CRC Biostatistics Series
Page : 0 pages
File Size : 45,7 Mb
Release : 2022-08-29
Category : Drug development
ISBN : 0367637014

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Real-World Evidence in Drug Development and Evaluation by HARRY. YANG Pdf

This book concerns use of real world data (RWD) and real world evidence (RWE) to aid drug development across product cycle. RWD are healthcare data that are collected outside the constraints of conventual controlled randomized trials (CRTs); whereas RWE is the knowledge derived from aggregation and analysis of RWD.

Real-World Evidence Generation and Evaluation of Therapeutics

National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation
Real World Evidence Generation and Evaluation of Therapeutics Book PDF

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation
Publisher : National Academies Press
Page : 103 pages
File Size : 45,7 Mb
Release : 2017-07-05
Category : Medical
ISBN : 9780309455657

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Real-World Evidence Generation and Evaluation of Therapeutics by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation Pdf

The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Potential effects on medical product development in the context of this wealth of real-world data could be numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to the ability to assess how therapeutics affect patients' lives through measurement of lifestyle changes. In October 2016, the National Academies of Sciences, Engineering, and Medicine held a workshop to facilitate dialogue among stakeholders about the opportunities and challenges for incorporating real-world evidence into all stages in the process for the generation and evaluation of therapeutics. Participants explored unmet stakeholder needs and opportunities to generate new kinds of evidence that meet those needs. This publication summarizes the presentations and discussions from the workshop.

Real-World Evidence Generation and Evaluation of Therapeutics

National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation
Real World Evidence Generation and Evaluation of Therapeutics Book PDF

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation
Publisher : National Academies Press
Page : 103 pages
File Size : 49,8 Mb
Release : 2017-08-05
Category : Medical
ISBN : 9780309455626

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Real-World Evidence Generation and Evaluation of Therapeutics by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation Pdf

The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Potential effects on medical product development in the context of this wealth of real-world data could be numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to the ability to assess how therapeutics affect patients' lives through measurement of lifestyle changes. In October 2016, the National Academies of Sciences, Engineering, and Medicine held a workshop to facilitate dialogue among stakeholders about the opportunities and challenges for incorporating real-world evidence into all stages in the process for the generation and evaluation of therapeutics. Participants explored unmet stakeholder needs and opportunities to generate new kinds of evidence that meet those needs. This publication summarizes the presentations and discussions from the workshop.

Examining the Impact of Real-World Evidence on Medical Product Development

National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation
Examining the Impact of Real World Evidence on Medical Product Development Book PDF

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation
Publisher : National Academies Press
Page : 231 pages
File Size : 48,9 Mb
Release : 2019-04-05
Category : Medical
ISBN : 9780309488327

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Examining the Impact of Real-World Evidence on Medical Product Development by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation Pdf

Randomized controlled trials (RCTs) have traditionally served as the gold standard for generating evidence about medical interventions. However, RCTs have inherent limitations and may not reflect the use of medical products in the real world. Additionally, RCTs are expensive, time consuming, and cannot answer all questions about a product or intervention. Evidence generated from real-world use, such as real-world evidence (RWE) may provide valuable information, alongside RCTs, to inform medical product decision making. To explore the potential for using RWE in medical product decision making, the National Academies of Sciences, Engineering, and Medicine planned a three-part workshop series. The series was designed to examine the current system of evidence generation and its limitations, to identify when and why RWE may be an appropriate type of evidence on which to base decisions, to learn from successful initiatives that have incorporated RWE, and to describe barriers that prevent RWE from being used to its full potential. This publication summarizes the discussions from the entire workshop series.

The Drug Development Paradigm in Oncology

National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Care Services,National Cancer Policy Forum
The Drug Development Paradigm in Oncology Book PDF

Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Care Services,National Cancer Policy Forum
Publisher : National Academies Press
Page : 145 pages
File Size : 46,5 Mb
Release : 2018-02-12
Category : Medical
ISBN : 9780309457972

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The Drug Development Paradigm in Oncology by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Care Services,National Cancer Policy Forum Pdf

Advances in cancer research have led to an improved understanding of the molecular mechanisms underpinning the development of cancer and how the immune system responds to cancer. This influx of research has led to an increasing number and variety of therapies in the drug development pipeline, including targeted therapies and associated biomarker tests that can select which patients are most likely to respond, and immunotherapies that harness the body's immune system to destroy cancer cells. Compared with standard chemotherapies, these new cancer therapies may demonstrate evidence of benefit and clearer distinctions between efficacy and toxicity at an earlier stage of development. However, there is a concern that the traditional processes for cancer drug development, evaluation, and regulatory approval could impede or delay the use of these promising cancer treatments in clinical practice. This has led to a number of effortsâ€"by patient advocates, the pharmaceutical industry, and the Food and Drug Administration (FDA)â€"to accelerate the review of promising new cancer therapies, especially for cancers that currently lack effective treatments. However, generating the necessary data to confirm safety and efficacy during expedited drug development programs can present a unique set of challenges and opportunities. To explore this new landscape in cancer drug development, the National Academies of Sciences, Engineering, and Medicine developed a workshop held in December 2016. This workshop convened cancer researchers, patient advocates, and representatives from industry, academia, and government to discuss challenges with traditional approaches to drug development, opportunities to improve the efficiency of drug development, and strategies to enhance the information available about a cancer therapy throughout its life cycle in order to improve its use in clinical practice. This publication summarizes the presentations and discussions from the workshop.

Improving and Accelerating Therapeutic Development for Nervous System Disorders

Institute of Medicine,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
Improving and Accelerating Therapeutic Development for Nervous System Disorders Book PDF

Author : Institute of Medicine,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
Publisher : National Academies Press
Page : 118 pages
File Size : 54,9 Mb
Release : 2014-02-06
Category : Medical
ISBN : 9780309292498

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Improving and Accelerating Therapeutic Development for Nervous System Disorders by Institute of Medicine,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders Pdf

Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.

Real-World Evidence in Medical Product Development

Weili He,Yixin Fang,Hongwei Wang
Real World Evidence in Medical Product Development Book PDF

Author : Weili He,Yixin Fang,Hongwei Wang
Publisher : Springer Nature
Page : 431 pages
File Size : 43,6 Mb
Release : 2023-05-11
Category : Mathematics
ISBN : 9783031263286

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Real-World Evidence in Medical Product Development by Weili He,Yixin Fang,Hongwei Wang Pdf

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.

Modern Methods of Clinical Investigation

Institute of Medicine,Committee on Technological Innovation in Medicine
Modern Methods of Clinical Investigation Book PDF

Author : Institute of Medicine,Committee on Technological Innovation in Medicine
Publisher : National Academies Press
Page : 241 pages
File Size : 49,8 Mb
Release : 1990-02-01
Category : Medical
ISBN : 9780309042864

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Modern Methods of Clinical Investigation by Institute of Medicine,Committee on Technological Innovation in Medicine Pdf

The very rapid pace of advances in biomedical research promises us a wide range of new drugs, medical devices, and clinical procedures. The extent to which these discoveries will benefit the public, however, depends in large part on the methods we choose for developing and testing them. Modern Methods of Clinical Investigation focuses on strategies for clinical evaluation and their role in uncovering the actual benefits and risks of medical innovation. Essays explore differences in our current systems for evaluating drugs, medical devices, and clinical procedures; health insurance databases as a tool for assessing treatment outcomes; the role of the medical profession, the Food and Drug Administration, and industry in stimulating the use of evaluative methods; and more. This book will be of special interest to policymakers, regulators, executives in the medical industry, clinical researchers, and physicians.

Registries for Evaluating Patient Outcomes

Agency for Healthcare Research and Quality/AHRQ
Registries for Evaluating Patient Outcomes Book PDF

Author : Agency for Healthcare Research and Quality/AHRQ
Publisher : Government Printing Office
Page : 396 pages
File Size : 52,5 Mb
Release : 2014-04-01
Category : Medical
ISBN : 9781587634338

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Registries for Evaluating Patient Outcomes by Agency for Healthcare Research and Quality/AHRQ Pdf

This User’s Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase understanding of patient outcomes. For the purposes of this guide, a patient registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes. A registry database is a file (or files) derived from the registry. Although registries can serve many purposes, this guide focuses on registries created for one or more of the following purposes: to describe the natural history of disease, to determine clinical effectiveness or cost-effectiveness of health care products and services, to measure or monitor safety and harm, and/or to measure quality of care. Registries are classified according to how their populations are defined. For example, product registries include patients who have been exposed to biopharmaceutical products or medical devices. Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization. Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews.

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 : 286 pages
File Size : 53,7 Mb
Release : 2020-11-11
Category : Mathematics
ISBN : 9781000208337

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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.

Economic Evaluation of Cancer Drugs

Iftekhar Khan,Ralph Crott,Zahid Bashir
Economic Evaluation of Cancer Drugs Book PDF

Author : Iftekhar Khan,Ralph Crott,Zahid Bashir
Publisher : CRC Press
Page : 416 pages
File Size : 43,7 Mb
Release : 2019-06-14
Category : Mathematics
ISBN : 9781498761314

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Economic Evaluation of Cancer Drugs by Iftekhar Khan,Ralph Crott,Zahid Bashir Pdf

Cancer is a major healthcare burden across the world and impacts not only the people diagnosed with various cancers but also their families, carers, and healthcare systems. With advances in the diagnosis and treatment, more people are diagnosed early and receive treatments for a disease where few treatments options were previously available. As a result, the survival of patients with cancer has steadily improved and, in most cases, patients who are not cured may receive multiple lines of treatment, often with financial consequences for the patients, insurers and healthcare systems. Although many books exist that address economic evaluation, Economic Evaluation of Cancer Drugs using Clinical Trial and Real World Data is the first unified text that specifically addresses the economic evaluation of cancer drugs. The authors discuss how to perform cost-effectiveness analyses while emphasising the strategic importance of designing cost-effectiveness into cancer trials and building robust economic evaluation models that have a higher chance of reimbursement if truly cost-effective. They cover the use of real-world data using cancer registries and discuss how such data can support or complement clinical trials with limited follow up. Lessons learned from failed reimbursement attempts, factors predictive of successful reimbursement and the different payer requirements across major countries including US, Australia, Canada, UK, Germany, France and Italy are also discussed. The book includes many detailed practical examples, case studies and thought-provoking exercises for use in classroom and seminar discussions. Iftekhar Khan is a medical statistician and health economist and a lead statistician at Oxford Unviersity’s Center for Statistics in Medicine. Professor Khan is also a Senior Research Fellow in Health Economics at University of Warwick and is a Senior Statistical Assessor within the Licensing Division of the UK Medicine and Health Regulation Agency. Ralph Crott is a former professor in Pharmacoeconomics at the University of Montreal in Quebec, Canada and former head of the EORTC Health Economics Unit and former senior health economist at the Belgian HTA organization. Zahid Bashir has over twelve years experience working in the pharmaceutical industry in medical affairs and oncology drug development where he is involved in the design and execution of oncology clinical trials and development of reimbursement dossiers for HTA submission.

Rare Diseases and Orphan Products

Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development
Rare Diseases and Orphan Products Book PDF

Author : Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development
Publisher : National Academies Press
Page : 442 pages
File Size : 47,9 Mb
Release : 2011-04-03
Category : Medical
ISBN : 9780309158060

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Rare Diseases and Orphan Products by Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development Pdf

Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

Developing a Protocol for Observational Comparative Effectiveness Research: A User's Guide

Agency for Health Care Research and Quality (U.S.)
Developing a Protocol for Observational Comparative Effectiveness Research A User s Guide Book PDF

Author : Agency for Health Care Research and Quality (U.S.)
Publisher : Government Printing Office
Page : 204 pages
File Size : 48,9 Mb
Release : 2013-02-21
Category : Medical
ISBN : 9781587634239

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Developing a Protocol for Observational Comparative Effectiveness Research: A User's Guide by Agency for Health Care Research and Quality (U.S.) Pdf

This User’s Guide is a resource for investigators and stakeholders who develop and review observational comparative effectiveness research protocols. It explains how to (1) identify key considerations and best practices for research design; (2) build a protocol based on these standards and best practices; and (3) judge the adequacy and completeness of a protocol. Eleven chapters cover all aspects of research design, including: developing study objectives, defining and refining study questions, addressing the heterogeneity of treatment effect, characterizing exposure, selecting a comparator, defining and measuring outcomes, and identifying optimal data sources. Checklists of guidance and key considerations for protocols are provided at the end of each chapter. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews. More more information, please consult the Agency website: www.effectivehealthcare.ahrq.gov)