Real-World Evidence – Why it is important for patient communities

Karen Facey
Karen Facey
  • 30 minute duration


Real-World Data (RWD) arises from a wide variety of situations in healthcare that give us an insight into normal clinical practice. In recent years there has been growing interest in understanding whether these data can be harnessed to develop robust Real-World Evidence that can inform decisions made by regulators, HTA bodies, Payers, clinicians and patients. When traditional research evidence from controlled clinical trials is limited, as is often the case in rare diseases, the potential for use of RWE seems particularly important, but its use is not commonplace.

This session will explore the opportunities and challenges in using RWD and reflect on actions that all stakeholders need to take to ensure that the most robust RWE is generated to inform all decisions. Particular focus will be placed on actions recommended for patient groups.

Learning outcomes

  1. The importance of RWE in the field of rare diseases
  2. Appreciation of the challenges in developing RWE
  3. Specific actions that patient groups could take in collaboration with others.
Karen Facey
Karen Facey
Karen Facey worked as a senior statistician in pharma and medicines regulation before going to Scotland to setup the first national health technology assessment (HTA) Agency. Since 2003 she has been an independent consultant on HTA and patient involvement. In 2005, she founded the HTA International Interest Group for Patient/Citizen Involvement and was lead editor on the definitive book on patient involvement in HTA published in 2017. Currently, she has a part-time post at the University of Edinburgh researching appraisal of rare disease treatments. As part of this research she has been involved in a number of multi-stakeholder initiatives exploring the potential of real-world evidence to inform decision makers about the value of new treatments for rare diseases.