Creating Evidence from Real World Patient Digital Data

Creating Evidence from Real World Patient Digital Data
Author: Jane Nikles
Publisher: Frontiers Media SA
Total Pages: 135
Release: 2021-04-07
Genre: Science
ISBN: 2889666719

Topic Editor Dr. Eric Daza is Senior Statistician at Clarify Health Solutions. All other Topic Editors declare no competing interests with regards to the Research Topic subject.

Real-World Evidence in a Patient-Centric Digital Era

Real-World Evidence in a Patient-Centric Digital Era
Author: Kelly H. Zou
Publisher: CRC Press
Total Pages: 155
Release: 2022-08-03
Genre: Mathematics
ISBN: 1000613453

Real-world evidence is defined as evidence generated from real-world data outside randomized controlled trials. As scientific discoveries and methodologies continue to advance, real-world data and their companion technologies offer powerful new tools for evidence generation. Real-World Evidence in a Patient-Centric Digital Era provides perspectives, examples, and insights on the innovative application of real-world evidence to meet patient needs and improve healthcare, with a focus on the pharmaceutical industry. This book presents an overview of key analytical issues and best practices. Special attention is paid to the development, methodologies, and other salient features of the statistical and data science techniques that are customarily used to generate real-world evidence. It provides a review of key topics and emerging trends in cutting-edge data science and health innovation. Features: Provides an overview of statistical and analytic methodologies in real-world evidence to generate insights on healthcare, with a special focus on the pharmaceutical industry Examines timely topics of high relevance to industry such as bioethical considerations, regulatory standards, and compliance requirements Highlights emerging and current trends, and provides guidelines for best practices Illustrates methods through examples and use-case studies to demonstrate impact Provides guidance on software choices and digital applications for successful analytics Real-World Evidence in a Patient-Centric Digital Era will be a vital reference for medical researchers, health technology innovators, data scientists, epidemiologists, population health analysts, health economists, outcomes researchers, policymakers, and analysts in the healthcare industry.

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

Examining the Impact of Real-World Evidence on Medical Product Development
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
Total Pages: 231
Release: 2019-04-05
Genre: Medical
ISBN: 030948832X

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.

Using Patient Reported Outcomes to Improve Health Care

Using Patient Reported Outcomes to Improve Health Care
Author: John Appleby
Publisher: John Wiley & Sons
Total Pages: 120
Release: 2016-01-19
Genre: Medical
ISBN: 1118948602

A practical, introductory guide to the best use of Patient Reported Outcomes (PROs) to improve the quality of health care and patient health. Only title to exclusively introduce, explain and show how PROs can be best used to improve healthcare and patient outcomes Includes real life examples and case studies of PROs in practice Assesses the growing evidence base for PROs in practice Editor team from Office of Health Economics (OHE), The King's Fund and King’s College London with contributions from practising clinicians, GPs and other healthcare professionals

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

Examining the Impact of Real-World Evidence on Medical Product Development
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
Total Pages: 231
Release: 2019-05-05
Genre: Medical
ISBN: 030948829X

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.

Patient Reported Outcomes

Patient Reported Outcomes
Author: Annabel Nixon
Publisher: SEEd
Total Pages: 43
Release: 2015-07-01
Genre: Medical
ISBN: 8897419607

Patient reported outcomes (PROs) are a measurement based on a report that comes directly from the patient about the status of their health condition without amendment or interpretation of the patient’s response by a clinician or anyone else. Valid and reliable PRO instruments are able to provide a standardized, quantifiable measure of treatment benefit, upon which the outcomes of interventions and treatment effect from the patient’s perspective can be judged. In some instances, PROs provide the best evidence of a treatment’s effectiveness, for example when evaluating treatments for pain, gastrointestinal and urological symptoms, or psychological well-being. This book aims to provide an overview of PRO applications, methodology and validation in order to help reader approach this measurement that are playing an increasingly central role in drug development decision making.

Maximising the Impact of Patient-reported Outcome Trial Results to Benefit Patients and Society

Maximising the Impact of Patient-reported Outcome Trial Results to Benefit Patients and Society
Author: Samantha Cruz Rivera
Publisher:
Total Pages: 0
Release: 2019
Genre:
ISBN:

There is an increasing need to demonstrate the return on medical research investment through benefit to patients, society and the economy. Patient-reported outcomes (PROs), such as quality of life and symptom data, are increasingly collected in clinical trials and may provide evidence, which can lead to a range of impacts. However it is unclear whether PRO impact is realised in practice. In addition, the different types of impact associated with PRO trial results, their barriers and facilitators, and appropriate impact metrics are not well defined. The doctoral research constituting this thesis adopted a mixed-methods approach with the aim of: a) synthesising existing methodological frameworks for healthcare research impact; b) determining the range of potential impacts associated with PRO data collected in trials, identifying potential PRO impact metrics and barriers and defining common facilitators to maximise PRO impact and; c) examining real-world evidence of PRO trial data impact and highlight optimal pathways to such impact. A number of studies were undertaken to address the aims. First, a systematic review of the literature identified 24 existing non-PRO-specific frameworks and over 80 impact metrics, which were then synthesised into a novel impact matrix and a simplified consolidated methodological framework for use by researchers and other stakeholders to help maximise the impact of healthcare research. Second, informed by this framework, an additional systematic review sought to determine the potential impact of PRO data collected in clinical trials and examined real world evidence of PRO trial data impact based on Research Excellence Framework (REF). This systemic review suggested that PRO trial data has the potential to inform clinical practice, clinical guidelines and, health policy; support drug approval, pricing and reimbursement decisions and; inform clinical and shared decision-making and consent for treatment. Furthermore, this second systematic review highlighted perceived methodological problems regarding the design, conduct and analysis and reporting of PRO data from clinical trials; which may hinder the impact of PRO data from clinical findings. Potential facilitators aimed at maximising PRO trial impact were also identified. The review identified 12 (n=69, 17%) REF 2014 impact case studies, which reported impact directly attributable to PRO findings. Including changes to international clinical guidelines and national guidelines, influencing cost-effectiveness analysis and drug approvals. Finally, in order to gain deeper understanding about the topics identified in the second systematic review, 24 semi-structured qualitative interviews were conducted with international stakeholders. Interviewees suggested PRO trial findings could lead to impact in the five impact categories identified in the aforementioned 'pathways to research impact' methodological framework. However, it was suggested that broader international stakeholder collaboration is required to tackle existing barriers and maximise the realisation of PRO trial impact on patients and society. In conclusion, this thesis has identified a range of potential impacts from PRO data which may benefit patients and society. However, a number of barriers need to be addressed to fully realise these benefits. This research highlights that the measurement of research impact, and specifically PRO research impact, is an essential exercise to better allocate limited funding, provide accountability and minimise research waste. Nonetheless, determining and implementing impact metrics is a complex task and will require greater stakeholder collaboration and engagement throughout the research pathway.

Registries for Evaluating Patient Outcomes

Registries for Evaluating Patient Outcomes
Author: Agency for Healthcare Research and Quality/AHRQ
Publisher: Government Printing Office
Total Pages: 385
Release: 2014-04-01
Genre: Medical
ISBN: 1587634333

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.

Virtual Clinical Trials

Virtual Clinical Trials
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
Total Pages: 127
Release: 2019-11-16
Genre: Medical
ISBN: 0309494885

Successful drug development relies on accurate and efficient clinical trials to deliver the best and most effective pharmaceuticals and clinical care to patients. However, the current model for clinical trials is outdated, inefficient and costly. Clinical trials are limited by small sample sizes that do not reflect variations among patients in the real world, financial burdens on participants, and slow processes, and these factors contribute to the disconnect between clinical research and clinical practice. On November 28-29, the National Academies of Sciences, Engineering, and Medicine convened a workshop to investigate the current clinical trials system and explore the potential benefits and challenges of implementing virtual clinical trials as an enhanced alternative for the future. This publication summarizes the presentations and discussions from the workshop.