FDA User Fee Reauthorization and the Value of Real-World Evidence

FDA User Fee Reauthorization and the Value of Real-World Evidence Cover

Issue Brief

FDA User Fee Reauthorization and the Value of Real-World Evidence

Published date

September 6, 2022

Increasing use of real-world evidence (RWE) in regulatory decision-making has the potential to provide advantages, including more efficient drug development and the ability to collect more robust information about the benefits and risks of new therapies. The result is potentially increased uses of RWE among key stakeholders, including regulators, providers, product developers, health systems, and patients/patient advocates, who are key to facilitating and broadening access to innovative, safe, and effective medical products for patients.

The United States Food and Drug Administration (FDA) utilized RWE in several cases to inform regulatory reviews of medical products, and the Prescription Drug User Fee Act (PDUFA) has provided critical support to advance the use of RWE. The foundation of the user fee programs is the understanding that collaboration and partnerships are essential to improve regulatory science and unlock innovation. User fees have provided resources for the FDA to engage in several multi-stakeholder discussions, including those convened by the Duke-Margolis Center for Health Policy, to identify key RWE policy considerations and gain experience working with this evidence as part of pilot studies and demonstration projects that inform regulatory reviews of medical products.

Congress is currently considering a reauthorization of FDA’s user fee authorities that will continue to drive exploration into the use of RWE for regulatory decision-making. The Duke-Margolis Real-World Evidence Collaborative has synthesized input from a range of stakeholders to recommend important opportunities for the use of RWE amid Congress’ consideration of reauthorizing FDA user fees. This blog considers this stakeholder input and the Center’s ongoing work to highlight current opportunities for advancing RWE implementation in support of regulatory decision-making and more.

Duke-Margolis Authors

Nora Emmott Headshot

Nora Emmott, MPH

Senior Policy Analyst

Matthew Matt D'Ambrosio Headshot Photo

Matt D'Ambrosio

Policy Analyst

Rachele Hendricks-Sturrup headshot

Rachele Hendricks-Sturrup, DHSc, MSc, MA

Research Director
Senior Team Member