Randomized controlled trials (RCTs) are traditionally used by the US Food and Drug Administration (FDA) to evaluate a product’s safety and effectiveness. While they are the gold-standard from a research perspective, RCTs are resource-intensive and require investing significant financial, time, and human capital. Coupled with unmet clinical needs and drug-access challenges, the demands of RCTs have fueled a call to action from patients and legislators. The regulatory system needs new ways to optimize the drug development process, so the delivery of effective drugs to the marketplace accelerates—while public safety remains ensured.

A large amount of patient information is captured at the point of care. Health care stakeholders are enthusiastic to explore whether that information can inform and hasten new drug development. Called real-world data (RWD), analyzing this burgeoning data source has the potential to provide real-world evidence (RWE). In turn, that RWE can tell us about the use of drugs, drug outcomes, and it can help us understand whether drugs are performing as intended in routine care.

The 21st Century Cures Act and the sixth Prescription Drug User Fee Act (PDUFA VI) set milestones for FDA to explore the use of RWE in regulatory decision-making. To inform FDA as it works to meet these milestones, the Duke-Margolis Center for Health Policy RWE Collaborative engages stakeholders to guide high-priority efforts aimed at improving the development and use of RWE. The Collaborative also strives to drive progress in the use of real-world data and evidence to improve patient treatment options and outcomes more broadly. Guided by an advisory group consisting of leaders representing medical product developers, payers, research groups, providers, patient networks, and regulators, the RWE Collaborative is interested in the following 2022 priorities:

Regulatory Acceptability of Real-World Data and Real-World Evidence...New methods of collecting data and evaluating evidence necessitates new rules for the road. This workstream looks at where regulatory policy is and where it needs to go in order to make RWD and RWE work for all stakeholders.

Learning Health Care Systems: RWD for Evidence-based Decision-making and Shared Learning...the Agency for Healthcare Research and Quality defines "learning health system" as "a health system in which internal data and experience are systematically integrated with external evidence, and that knowledge is put into practice." This workstream is interested in the current state of play for LHS implementation in the United States and how RWD/E is being leveraged to advance learning health systems.

Master RWE Protocols...Traditionally, master protocols have helped clinical trials achieve greater efficiency. This workstream asks how we can bring those lessons learned to studies that use real-world data.

Real-World Efficacy: Patient Subgroups...One of the greatest advantages of RWD is that it creates evidence about groups that are traditionally excluded from clinical trials. This workstream looks at cases where real-world data and real-world evidence can advance knowledge for the benefit of specific groups of patients.

The Real-World Evidence Collaborative is led by Research Director, Rachele Hendricks-Sturrup, DHSc, MSc, MA. To learn more about the group's efforts, please contact her at rachele.hendricks.sturrup@duke.edu