The Robert J. Margolis Center for Health Policy at Duke University, under a cooperative agreement with the U.S. Food and Drug Administration (FDA), is exploring the potential for broader use of the Sentinel System’s data infrastructure and partnerships. The Sentinel System is designed to monitor the safety of FDA-regulated medical products and has the potential for its data infrastructure to be used for quick and efficient development of real-world evidence. The Sentinel System’s data infrastructure includes data on 170 million covered lives, with more than 50 million considered highly curated and ready for rapid analysis. Through exploratory discussions with stakeholders, Duke-Margolis has identified the cholesterol-lowering anti-PCSK9 monoclonal antibodies, or PCSK9 inhibitors, as a promising use case to explore the feasibility of generating real-world evidence using the Sentinel System data infrastructure.
PCSK9 inhibitors are a relatively new class of drugs, with two products approved in 2015 and additional products in late-stage clinical development. Given the new nature of this class, there remain unanswered questions where real-world evidence is needed. The Duke-Margolis Center convened a workshop with patient representatives, payers, integrated health systems, non-profit research institutions, cardiovascular specialists, and data analytics professionals to understand the challenges and opportunities to developing a Sentinel System study for PCSK9 inhibitors. The first day of the two-day workshop included a larger group assessing the feasibility of exploring high-priority PCSK9 inhibitor research using the Sentinel System infrastructure and a smaller group on the second day exploring the detailed practical considerations including key operational issues, administrative needs, and the value proposition for data partners to participate. Following on this project, the Duke-Margolis will engage with stakeholders to explore a pilot project that could generate needed evidence about this new class of drugs.