Real-world evidence (RWE) in medicine is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of
real-world data (RWD). RWE can be generated by different study designs or analyses, including but not limited to, randomized trials, including large simple trials, pragmatic trials, and retrospective or prospective
observational studies
In fields such as epidemiology, social sciences, psychology and statistics, an observational study draws inferences from a sample (statistics), sample to a statistical population, population where the dependent and independent variables, independ ...
.
In the USA the
21st Century Cures Act required the
FDA
The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food ...
to expand the role of real world evidence.
[Division A—21st Century Cures]
/ref>
Real-world evidence comes into play when clinical trials cannot really account for the entire patient population of a particular disease. Patients with comorbidities or belonging to a distant geographic region or age limit who did not participate in any clinical trial may not respond to the treatment in question as expected. RWE provides answers to these problems and also to analyze effects of drugs over a longer period of time. Pharmaceutical companies and health insurance payers study RWE to understand patient pathways to deliver appropriate care for appropriate individuals and to minimize their own financial risk by investing on drugs that work for patients.
Data quality
In order to use real-world data to generate evidence, data must be of sufficient quality. Kahn et al. define data quality as consisting of three components: (1) conformance (do data values adhere to do specified standard and formats?; subtypes: value, relational and computational conformance); (2) completeness (are data values present?); and (3) plausibility (are data values believable?; subtypes uniqueness, atemporal; temporal).
Fitness for purpose
Similarly to having sufficient data quality, the real-world data must be fit for purpose. An RWD resource can be fit for addressing some questions, but not others. For example, a dataset that lacks mother-to-baby links may not be appropriate to address drug risk for fetus but can be used for questions for drug safety in patients taking epilepsy treatment (limited to the patient; not including safety for fetus). Since data quality can be evaluated outside a particular purpose (on a general level), fitness for purpose is evaluated separate from data quality and is not included in the concept of data quality.
See also
* Correlation does not imply causation
The phrase "correlation does not imply causation" refers to the inability to legitimately deduce a cause-and-effect relationship between two events or variables solely on the basis of an observed association or correlation between them. The id ...
* Evidence-based medicine
Evidence-based medicine (EBM) is "the conscientious, explicit and judicious use of current best evidence in making decisions about the care of individual patients". The aim of EBM is to integrate the experience of the clinician, the values of t ...
* Levels of evidence
A hierarchy of evidence (or levels of evidence) is a heuristic used to rank the relative strength of results obtained from scientific research. There is broad agreement on the relative strength of large-scale, epidemiological studies. More than 8 ...
* Pragmatic clinical trial
A pragmatic clinical trial (PCT), sometimes called a practical clinical trial (PCT), is a clinical trial that focuses on correlation between treatments and outcomes in real-world health system practice rather than focusing on proving causativ ...
* Qualitative research
Qualitative research is a type of research that aims to gather and analyse non-numerical (descriptive) data in order to gain an understanding of individuals' social reality, including understanding their attitudes, beliefs, and motivation. This ...
* Quantitative research
Quantitative research is a research strategy that focuses on quantifying the collection and analysis of data. It is formed from a deductive approach where emphasis is placed on the testing of theory, shaped by empiricist and positivist philosop ...
References
* Real-World Evidence — What Is It and What Can It Tell Us? ''The New England Journal of Medicine
''The New England Journal of Medicine'' (''NEJM'') is a weekly medical journal published by the Massachusetts Medical Society. It is among the most prestigious peer-reviewed medical journals as well as the oldest continuously published one.
His ...
'', December 6, 2016
Real World Evidence
FDA, June 21, 2018.
* Mahajan, Rajiv. “Real World Data: Additional Source for Making Clinical Decisions.” '' International Journal of Applied and Basic Medical Research'' 5.2 (2015): 82. PMC. Web. 5 May 2018.
* Berger, Marc L. et al. “Good Practices for Real‐world Data Studies of Treatment And/or Comparative Effectiveness: Recommendations from the Joint ISPOR‐ISPE Special Task Force on Real‐world Evidence in Health Care Decision Making.” '' Pharmacoepidemiology and Drug Safety'' 26.9 (2017): 1033–1039. PMC. Web. 5 May 2018.
External links
"Real World Evidence"
at FDA
The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food ...
"21st Century Cures Act"
"Use of Real World Data in Development Programmes"
at EMA
"Observational Health Data Sciences and Informatics"
"Need for Real World Evidence"
"Real-world evidence: From activity to impact in healthcare decision making"
{{DEFAULTSORT:Real world evidence
Evidence
Health informatics
Clinical research