Got a question about real-world data and evidence, health economics and outcomes research, or decision-making? Read our frequently asked questions below!

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What is real-world evidence?
Real-world evidence is evidence obtained from real-world data. These are observational data, generated during routine clinical practice, obtained outside the context of tightly controlled randomized controlled trials. Real-world evidence is generated by analyzing data which are stored in electronic health records, electronic medical records, medical claims or billing activities databases, registries, patient-generated data and mobile devices, amongst other sources.
Where do real-world data come from?
Real-world data are derived from a number of sources associated with outcomes in heterogeneous patient populations in real-world settings. Examples of real-world data sources include claims data, observational cohort studies, electronic and medical health records and national registries. Real-world data can also be generated from patient-reported outcomes and app-based questionnaires, such as through analysis of data obtained from wearables and patient surveys.
What is real-world evidence used for?
Real-world evidence and real-world data are being increasingly employed by various healthcare stakeholders.

Physicians and providers are interested in the use of real-world evidence for the assessment of patients’ treatment adherences, monitoring the safety of newly approved interventions and assessing adverse events associated with these. This real-world evidence can also inform stakeholders as to whether certain interventions are more effective in specific patient subgroups.

Payers employ real-world evidence, in the form of claims and billing data, to evaluate the cost–effectiveness of interventions, to help make drug coverage decisions and improve the affordability of healthcare.

Real-world evidence is also employed in regulatory decision-making, the post-marketing surveillance of drugs and medical devices, and in the development of guidelines and decision support tools for clinical practice.

How do real-world data compare with randomized controlled trial data obtained from traditional clinical trials?
Randomized controlled trials are considered the gold standard for determining whether treatments or interventions actually work. Relatively small patient populations – that represent only a fraction of a disease population – are recruited to participate in randomized controlled trials and the data derived from these clinical trials are often used to acquire regulatory approval for new drugs or interventions and make these readily available for patients.

By contrast, real-world data depict patients’ experiences in their real-world environments, outside of tightly controlled, unnatural clinical trial settings. Real-world evidence studies provide insights concerning whether a treatment does work, for real people in real-world environments, accounting for their other indications and medications, and the results pertain to a much larger, heterogenous patient population.

Can real-world data replicate clinical trial data?
The ‘RCT DUPLICATE‘ study aims to use real-world evidence to attempt to replicate the results of 30 completed randomized controlled trials. In April 2019, it was announced that this project was expanded to employ real-world evidence to predict the results of seven ongoing Phase IV trials, which represents the first time researchers will attempt to predict the results of trials that have not reached conclusion.
What is the Cures Act?
In the USA in 2016, the 21st Century Cures Act – often simply referred to as the Cures Act – was passed. This act intended to promote more rapid development of drugs and biologics and enhances the US FDA’s ability to modernize clinical trial designs, including the use of real-world evidence for example.
What are electronic health records?
Electronic health records are systematized collections of patients’ and populations’ electronically stored health information, in a digital format. Electronic health records are often used as sources of real-world data.
What is the difference between an electronic health record and an electronic medical record?
This distinction is very nuanced: an electronic health record comprises all of a patients’ health record, across all medical service-proving institutions. By contrast, an electronic medical record is the single database of an individual’s information within one institution.
What is comparative effectiveness research?
Comparative effectiveness research concerns the conduct, and/or synthesis, of research directly comparing different benefits and harms of alternative interventions or strategies to prevent, diagnose, treat and monitor a specified health condition in routine clinical practice (i.e. the real-world setting). Comparative effectiveness research includes both primary data collection and secondary analyses – such as systematic literature reviews, meta-analyses and economic evaluations.