March 15, 2019: Leveraging RWE to Support Regulatory Decisions–An Update on Efforts to Inform Policy (Gregory W. Daniel, PhD, MPH, RPh)


Gregory W. Daniel, PhD, MPH, RPh
Deputy Center Director, Duke-Margolis Center for Health Policy
Clinical Professor, Fuqua School of Business
Duke University


Leveraging RWE to Support Regulatory Decisions–An Update on Efforts to Inform Policy


Real world evidence; Health care system; Regulatory; Data curation; Policy making; FDA

Key Points

  • Real world data and real world evidence are not the same thing. Real world evidence includes observational and randomized designs while real world data does not.
  • While real world data and real world evidence can support important health care system questions, it does not yet inform regulatory decisions.
  • The U.S. Food and Drug Administration (FDA) real world evidence program aims to evaluate potential uses of real world evidence to support drug label changes and product effectiveness.
  • Several planned guidances on real world data and real world evidence released by the FDA are used to support regulatory decision making.

Discussion Themes

Data curation is often complex and hard to explain.

Two key components of dependable data are adequacy and control. Adequacy of data can easily be determined, but within a real world evidence system, determining if data is well controlled can be more challenging. A better understanding of what study designs will help in arriving at the most trustworthy data is paramount in effectively leveraging real world evidence.

Immediate next steps for applying FDA RWE guidance includes determining what the best practices for commonalities in data curation are. It’s also important to explore what has been done in the past and determine where problems lie and what can be done to solve them.  

Research design and methods play a key role in determining the best way of determining the reliability of data and how it can be leveraged in a clinical practice setting.


#realworldevidence, #pctGR, #healthcaresystem, #regulatory, @gregwdaniel, @dukemargolis, @collaboratory1

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