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Examining the Recent Duke-Margolis White Paper: A Roadmap for Developing Study Endpoints in Real-World Settings

By Rachel Sobel, DrPH, FISPE Vice President, Epidemiology at UBC

Illustration of real world dataIn August, the Duke Margolis Center for Health Policy released a paper exploring research considerations for developing real world evidence endpoints. As the International Society for Pharmacoepidemiology’s (ISPE) representative on the project, I participated in the Working Group tasked with providing feedback to the Duke-Margolis authors. The Working Group was comprised of individuals with diverse expertise and affiliations across academia, industry, government, and scientific societies, which brought a breadth of expertise and perspectives.

I am an active member and fellow of ISPE, which has been involved in real world evidence (RWE) for decades, and ISPE is playing a strategic role internally and externally in the use of RWE for regulatory decision-making.  UBC epidemiologists, clinicians, and biostatisticians similarly have been involved in the application of real world data (RWD) and RWE for over 25 years and have been generous in supporting my involvement in these kinds of important scientific society and policy initiatives.

Creating real world evidence endpoints is not easy, and it’s often even more complicated in the context of RWE for regulatory decision-making where data are often perceived as “messy” and skepticism of real world studies is generally higher compared to traditional randomized clinical trials.

Real world evidence endpoint development needs many of the same considerations in RWE and clinical trials:

  • The concept of interest must be appropriately articulated and tailored to the scientific question to be addressed
  • The outcome and endpoint hierarchy needs to be carefully considered
  • The real world evidence endpoint needs to be conceptually, if not formally, validated to be potentially acceptable for regulatory decision-making.

UBC takes the same methodical approach in selecting and/or developing endpoints for our clients’ projects. We carefully consider the data source that is best suited to address the research question and then using a variety of techniques – from evaluating the literature for existing endpoint definitions, to qualitative research to inform our thinking, to creating novel endpoints across multiple disparate data sources that are specific to the scientific objective using advanced analytics. The majority of our studies are submitted to regulators to fulfill post-approval commitments and are thus “regulatory grade.”

To hear more about our RWE experience and how it can be applied to your programs, please get in touch with us today.

The white paper published by Duke-Margolis Center for Health Policy and Research can be accessed here.