scholarly journals The Expanding Role of Real-World Evidence Trials in Health Care Decision Making

2019 ◽  
Vol 14 (1) ◽  
pp. 174-179 ◽  
Author(s):  
David C. Klonoff
Keyword(s):  
Real World ◽  
Clinical Evidence ◽  
Real World Data ◽  
Medical Products ◽  
Advantages And Disadvantages ◽  
Randomized Controlled ◽  
Real World Evidence ◽  
Care Decision

Real-world evidence (RWE) is the clinical evidence about benefits or risks of medical products derived from analyzing real world data (RWD), which are data collected through routine clinical practice. This article discusses the advantages and disadvantages of RWE studies, how these studies differ from randomized controlled trials (RCTs), how to overcome barriers to current skepticism about RWE, how FDA is using RWE, how to improve the quality of RWE, and finally the future of RWE trials.

scholarly journals Role of real-world evidence in informing cancer care: lessons from colorectal cancer

2019 ◽  
Vol 26 (1) ◽  
Author(s):  
A. Batra ◽  
W. Y. Cheung
Keyword(s):  
Colorectal Cancer ◽  
Real World ◽  
Cancer Care ◽  
Clinical Care ◽  
Real World Data ◽  
Cancer Management ◽  
Randomized Controlled ◽  
Real World Evidence ◽  
Research Questions

The term “real-world evidence” (rwe) describes the analysis of data that are collected beyond the context of clinical trials. The use and application of rwe have become increasingly common and relevant, especially in oncology, because there is growing recognition that randomized controlled trials (rcts) might not be sufficiently representative of the entire patient population that is affected by cancer, and that specific clinical research questions might be best addressed by real-world data. In this brief review, our main aim is to highlight the role of rwe in informing cancer care, particularly focusing on specific examples from colorectal cancer. Our hope is to illustrate the ways in which rwe can complement rcts in improving the understanding of cancer management and how rwe can facilitate cancer treatment decisions for real-world patients encountered in routine clinical care.

scholarly journals Role of real-world evidence studies in cardiology

2021 ◽  
Vol 26 (3S) ◽  
pp. 4636
Author(s):  
T. V. Pavlova
Keyword(s):  
Real World ◽  
Observational Studies ◽  
Safety Data ◽  
Randomized Controlled ◽  
Medication Effects ◽  
Real World Evidence ◽  
Methodological Problems ◽  
Based Medicine

The introduction of evidence-based medicine into practical healthcare provides physicians with the opportunity to use treatment approaches, which have been proven to be effective in randomized controlled trials. In this case, it is necessary to consider using the medication in a particular patient in accordance with the design of clinical trials and strictly follow the instructions. Drugs should be critically selected with focus on quality of available efficacy and safety data obtained in the population closest to a patient. In addition, clinicians should regularly review the available data with particular regard to its quality. The advantages, disadvantages, limitations and methodological problems of observational studies should be carefully considered during the interpretation of results. At the same time, the compliance of the results of real-world evidence studies with registration trial data indicates a high reproducibility of medication effects.

scholarly journals Increasing Trust in Real-World Evidence Through Evaluation of Observational Data Quality

2021 ◽  
Author(s):  
Clair Blacketer ◽  
Frank J Defalco ◽  
Patrick B Ryan ◽  
Peter R Rijnbeek
Keyword(s):  
Data Quality ◽  
Real World ◽  
R Package ◽  
Observational Research ◽  
Real World Data ◽  
Quality Reporting ◽  
Healthcare Data ◽  
Real World Evidence ◽  
Quality Checks

Advances in standardization of observational healthcare data have enabled methodological breakthroughs, rapid global collaboration, and generation of real-world evidence to improve patient outcomes. Standardizations in data structure, such as use of Common Data Models (CDM), need to be coupled with standardized approaches for data quality assessment. To ensure confidence in real-world evidence generated from the analysis of real-world data, one must first have confidence in the data itself. The Data Quality Dashboard is an open-source R package that reports potential quality issues in an OMOP CDM instance through the systematic execution and summarization of over 3,300 configurable data quality checks. We describe the implementation of check types across a data quality framework of conformance, completeness, plausibility, with both verification and validation. We illustrate how data quality checks, paired with decision thresholds, can be configured to customize data quality reporting across a range of observational health data sources. We discuss how data quality reporting can become part of the overall real-world evidence generation and dissemination process to promote transparency and build confidence in the resulting output. Transparently communicating how well CDM standardized databases adhere to a set of quality measures adds a crucial piece that is currently missing from observational research. Assessing and improving the quality of our data will inherently improve the quality of the evidence we generate.

scholarly journals Race and Ethnicity in Real-World Data Sources: Considerations for Medical Device Regulatory Efforts

2021 ◽  
Vol 12 ◽  
pp. 215013272199404
Author(s):  
Michelle E. Tarver
Keyword(s):  
Medical Device ◽  
Real World ◽  
Race And Ethnicity ◽  
Data Sources ◽  
Real World Data ◽  
Daily Lives ◽  
World Data ◽  
Medical Products ◽  
Real World Evidence ◽  
Ethnic Subgroups

Real-world evidence is increasingly being collected in our interconnected world. This information can provide insights on how medical products are impacting the daily lives of patients, translating into public health consequences. Understanding the demographic characteristics of patients contributing health information to real-world data sources is critical to determining the safety and effectiveness of medical products for specific populations. Race and ethnicity are recorded inconsistently in different real-world data sources, with many lacking reliable information on these variables. To better leverage all forms of real-world data to inform medical device evaluation, accurate and consistent collection of information from different racial and ethnic subgroups is necessary.

Contributions of Real-World Evidence and Real-World Data to Decision-Making in the Management of Soft Tissue Sarcomas

Oncology ◽  
2021 ◽  
Vol 99 (Suppl. 1) ◽  
pp. 3-7
Author(s):  
George D. Demetri ◽  
Silvia Stacchiotti
Keyword(s):  
Decision Making ◽  
Soft Tissue ◽  
Real World ◽  
Soft Tissue Sarcomas ◽  
Clinical Settings ◽  
Disease Entity ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence

Real-world data are defined as data relating to any aspect of a patient’s health status collected in the context of routine health surveillance and medical care delivery. Sources range from insurance billing claims through to electronic surveillance data (e.g., activity trackers). Real-world data derive from large populations in diverse clinical settings and thus can be extrapolated more readily than clinical trial data to patients in different clinical settings or with a variety of comorbidities. Real-world data are used to generate real-world evidence, which might be regarded as a “meta-analysis” of accumulated real-world data. Increasingly, regulatory authorities are recognizing the value of real-world data and real-world evidence, especially for rare diseases where it may be practically unfeasible to conduct randomized controlled trials. However, the quality of real-world evidence depends on the quality of the data collected which, in turn, depends on a correct pathological diagnosis and the homogeneous behaviour of a reliably defined and consistent disease entity. As each of the more than 80 varieties of soft tissue sarcoma (STS) types represents a distinct disease entity, the situation is exceedingly complicated. Discordant diagnoses, which affect data quality, present a major challenge for use of real-world data. As real-world data are difficult to collect, collaboration across sarcoma reference institutions and sophisticated information technology solutions are required before the potential of real-world evidence to inform decision-making in the management of STS can be fully exploited.

scholarly journals Regulatory-grade clinical trial design using real-world data

2020 ◽  
Vol 17 (4) ◽  
pp. 377-382
Author(s):  
Mark S Levenson
Keyword(s):  
Real World ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence ◽  
Study Results ◽  
Potential Use ◽  
The U.S

Real-world data and evidence provide the potential to address the effectiveness and safety of drugs. The U.S. Food & Drug Administration has initiated a program to evaluate the potential use of real-world evidence for regulatory uses. Whether a study is designed for regulatory purposes or for other purposes, existing regulation and guidance provide a reference for high-quality studies. Clarifying the study objectives and the role of real-world data in the study are important considerations. Robustness and transparency of the analysis allow for greater understanding and acceptance of the study results.

scholarly journals Allergen immunotherapy: the growing role of observational and randomised trial “real-world evidence”

2021 ◽  
Author(s):  
Giovanni Paoletti ◽  
Danilo Di Bona ◽  
Derek Chu ◽  
Davide Firinu ◽  
Enrico Heffler ◽  
...  
Keyword(s):  
Real World ◽  
Allergen Immunotherapy ◽  
Clinical Decision Making ◽  
Randomised Trial ◽  
Unmet Need ◽  
Real Life ◽  
Real World Data ◽  
Level Of Evidence ◽  
Real World Evidence

Although there is a considerable body of knowledge about allergen immunotherapy (AIT), there is a lack of data on the reliability of real-world evidence (RWE) in AIT and consequently, a lack of information on how AIT effectively works in real life. To address the current unmet need for an appraisal of the quality of RWE in AIT, the European Academy of Allergy and Clinical Immunology Methodology Committee recently initiated a systematic review of observational studies of AIT, which will use the RELEVANT tool and the Grading of Recommendations Assessment, Development and Evaluation approach (GRADE) to rate the quality of the evidence base as a whole. The next step will be to develop a broadly applicable, pragmatic “real-world” database using systematic data collection. Based on the current RWE base, and perspectives and recommendations of authorities and scientific societies, a hierarchy of RWE in AIT is proposed, which places pragmatic trials and registry data at the positions of highest level of evidence. There is a need to establish more AIT registries that collect data in a cohesive way, using standardised protocols. This will provide an essential source of real-world data that can be easily shared, promoting evidence-based research and quality improvement in study design and clinical decision-making.

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