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 for Outcomes Research 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. ISPOR and ISPE held a Summit in October 2017 that provided a forum to review these task force reports, consider the perspectives of key stakeholders, and discuss future steps that could help enable these recommendations. The presentation from this summit is available here. The Special Task Force produced and published 2 papers and an editorial in this area (see call-out below).
- Leading a collaborative initiative regarding RWE transparency with approximately 35 different stakeholder organizations represented, related to a priori registration of hypothesis testing treatment effect evaluating studies including a Summit in October 2019 with an update on this project.
For additional information on the initiative, please contact us.
Real-World Evidence Transparency Initiative
The Real-World Evidence Transparency Initiative Partnership is a collaborative effort between ISPOR, the International Society for Pharmacoepidemiology, the Duke-Margolis Center for Health Policy, and the National Pharmaceutical Council. The objective of this initiative is to establish a culture of transparency for study analysis and reporting of hypothesis evaluating real-world evidence studies on treatment effects. Learn more about the initiative here.
On September 18, 2019 the steering committee of the Real-World Evidence Transparency Initiative released a draft white paper for public comment. The white paper focuses on the need for study registration as a necessary first step towards improving transparency and trust in non-interventional research for hypothesis testing. Following comments from membership and other stakeholders, the white paper was finalized and is pending publication in Value in Health as well as Pharmacoepidemiology and Drug Safety. Publication is expected in the third quarter of 2020.
AHA-ISPOR Joint Session on Real World Evidence
Adrian Hernandez, MD and Richard Willke, PhD, chief science officer of the International Society for Pharmacoeconomics and Outcomes Research, discuss the topics they presented during QCOR 2020: real world evidence and the standards evolving for its proper use in clinical trials.
Joint ISPE-ISPOR Special Task Force Reports:
- Good Practices for Real‐World Data Studies of Treatment and/or Comparative Effectiveness: Recommendations from the Joint ISPOR‐ISPE Special Task Force on Real‐World Evidence in Healthcare Decision Making
- Reporting to Improve Reproducibility and Facilitate Validity Assessment for Healthcare Database Studies V1.0
- Making Real-World Evidence More Useful for Decision Making (editorial)