Real-world evidence can enhance clinical decision-making

Wednesday, 28 Jun 2017

Real-world data generated using rigorous scientific methods can complement randomised controlled trials by providing unique insights for practicing evidence-based medicine, delegates attending this year’s EULAR congress in Madrid have been told.

Speaking during an AbbVie sponsored symposium titled rheum for improvement: the increasing role of real-world evidence in decision making Associate Professor Deborah Marshall from the University of Calgary in Canada said randomised controlled trials (RCTs) were a powerful tool for safety and efficacy in medicine and would always be considered the gold standard for clinical research.

However, she said RCTS had some inherent limitations in that they may not truly represent patient outcomes in the real world.

This was because these trials often involved highly selected participants who were studied for a limited duration under controlled conditions.

In contrast, real world evidence (RWE) involved a heterogenous population in routine practice conditions that reflected diverse patient behaviours.

“The primary attribute that distinguishes RWE from RCTs is the context in which the evidence is gathered,” she explained.

“Through RWE we are able to reflect different patient behaviours and different preferences and choices so that we can think about and really understand adherence and compliance to treatments,” she told delegates.

She said RWE could inform healthcare decision making in four key ways: by identifying the burden of illness, assessing treatment patterns, examining patient behaviours and comparing treatment outcomes.

For example, a real-world study in Canada demonstrated the impact of achieving sustained remission in RA on the reduction of healthcare costs in Canada1.

Another real-world study reported that a delay in the diagnosis of psoriatic arthritis by more than one year was associated with worse clinical outcomes2.

Professor Marshall said the value that RWE could add to evidence based medicine was gaining traction, particularly in regulatory circles.

 “There’s a lot of increasing interest to expand and integrate this kind of research [RWE] and this is largely in response to a need for better generalisability because of the limitations of RCTs and also the intense and costly efforts required to conduct RCTs,” she said.

She noted that the European Medicines Agency had recognised that RWE had the ability to “significantly contribute to the way the benefit-risk balance of medicines is assessed over their entire life cycle”3.

The FDA described the key to understanding the usefulness of RWE as “an appreciation of its potential for complementing the knowledge gained from traditional clinical trials”4.

However Professor Marshall cautioned that RWE posed challenges both in its design and analysis because there was a need to think about how to assess causality and how to control for confounding factors.

“Real world evidence is not a panacea and we need to be cautious with respect to robust methods we need to analyse and interpret and design RWE studies,” she said.

This article has been commissioned and sponsored by AbbVie Pty ltd. The content and views within the article are based on published studies and the speaker’s opinions. The views within the article do not necessarily reflect those of AbbVie Pty Ltd.


  1. Barnabe, C et al. Ann Rheum Dis 2013; 72:1664-1668.
  2. Harron M, et al. Ann Rheum Dis 2015; 74: 1045-1050.
  3. European Medicines Agency Annual Report 2016
  4. Sherman R et al. N Engl J Med 2016;375:2293-2297.


Already a member?

Login to keep reading.

Email me a login link

© 2022 the limbic