The volume and diversity of real-world data (RWD) have been growing exponentially as technology and integrated electronic medical records have made this information increasingly accessible and useful for outcomes research and regulatory purposes. While clinical trial evidence remains the gold standard for evaluation of treatment efficacy, there is increasing interest and potential for converting RWD into real-world evidence (RWE) that, through careful analysis and interpretation, can be used to inform healthcare decision making.

RWD offers many benefits over randomized controlled trials that are particularly useful for research that can be applied to healthcare decision making. Advantages include the availability of timely data at reasonable cost, large sample sizes that enable analysis of subpopulations and less common effects, and the representativeness of real-world practice and behaviors.

While RWE offers tremendous potential, it also presents very real concerns that continue to cast shadows on its credibility, such as biases due to lack of randomization, data quality, and the potential for spurious results due to data mining. Despite having at least 2 decades of significant data capabilities in healthcare, progress has lagged primarily due to these concerns.

For these reasons, ISPOR is actively working to improve standards and practice for the collection and analysis of RWD. The Society views this initiative as a critical aspect of ISPOR’s mission to promote health economics and outcomes research excellence to improve decision making for health globally.

Since its founding, ISPOR has:

  • Convened several RWD-related task forces and issued a number of Good Practices Reports on this issue
  • Assembled an International Digest of Databases with an associated special interest group
  • Collaborated with the Academy of Managed Care Pharmacy and the National Pharmaceutical Council to create a tool and related training for decision makers in evaluating comparative-effectiveness research using RWD and other sources.
  • Partnered with the International Society for Pharmacoepidemiology (ISPE) to form a joint Special Task Force on Real-World Evidence in Healthcare Decision Making. The Special Task Force produced and published 2 papers and an editorial in this area (see call-out below).

For additional information on the initiative, please contact us.