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Real World Evidence

Focus Area: Data Transparency

Real World Evidence

The project entitled Observational Patient Evidence for Regulatory Science And uNderstanding Disease (OPERAND) in partnership with OptumLabs is designed to better inform the use of Real World Evidence (RWE) from retrospective observational studies in regulatory decision-making and medicine. The strategy of the OPERAND program is to convene key stakeholders, including industry, academia, and regulators, to consider the principles behind, the methodology to draw upon, and the appropriate utilization of observational data in regulatory review and approval and comparative effectiveness
research. This is a program that will first use retrospective observational data to determine if such data can confirm previously published randomized clinical trial (RCT) results and, if successful, extend the use of the data for other potential applications. We aim to develop and utilize methodology that is compelling to regulatory approvers, payers, and policy makers.

While there are examples of individual RCTs that have been replicated using observational database analyses, there has never been an effort to replicate a large number of trials across several different therapeutic areas simultaneously. Such an effort is needed to shed light on a number of critical questions particularly relevant in retrospective analyses, including quality of data sources, bias (and ways to mitigate it), and variability in observational database studies. One important issue is the potential of data linkage (e.g., claims data linked with electronic medical records) to control for confounders that is missing in analyses based on claims data alone. The MRCT Center in collaboration with OptumLabs is leading the development of an empirical study by bringing together a panel of technical experts from various organizations to address these questions.

Current Status: Active Project

Impact: Utilizing RWE can expand the population of an approved product to a broader population within the same indication or encompass a new indication (label expansion).


  • To investigate the reliability of evidence from retrospective database studies of medical claims and electronic medical records by engaging diverse stakeholders to determine study design.
  • To implement a series of pilot studies to determine feasibility and scale the approach to different diseases and trials.

Key Milestones:

  • March 2017: Initial scoping and concept sheet completed; initial discussions among strategic partners: Margolis Center for Health Policy (Duke) and OptumLabs
  • September 2017: Launched Project OPERAND (Observational Patient Evidence for Regulatory Science And uNderstanding Disease) in partnership with OptumLabs. Convened key stakeholders from industry, academia, and regulatory agencies to discuss appropriate utilization of observational data in comparative effective research and regulatory review/approvals

Project Coordinator: Hayat Ahmed