FDA invites sponsors to participate in next cycle of Advancing Real-World Evidence Program

The US Food and Drug Administration (FDA) is inviting sponsors to submit proposals to its Advancing Real-World Evidence (RWE) Program, its initiative designed to support the development of regulatory-grade evidence using real-world data (RWD).

Established in October 2022 as part of the agency’s commitments under the Prescription Drug User Fee Act (PDUFA) VII, the program aims to improve the quality and acceptability of RWE approaches used in regulatory submissions. In particular, it focuses on studies that could support new intended labeling claims, including additional therapeutic indications for approved products, expanded patient populations, or dosing modifications. RWE approaches may also be used to fulfill post-approval study requirements.

Through the program, selected sponsors can request a meeting with multidisciplinary FDA teams from the Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), and the Oncology Center of Excellence (OCE). These discussions take place early in the development process, before study protocols are finalized or clinical studies begin, allowing companies to obtain feedback on their proposed use of RWD and study design.

Speaking recently about the program in a recent webinar, Marie Bradley, Senior Advisor for Real-World Evidence Analytics at FDA, encouraged sponsors to engage with the agency early in the evidence development process. Discussions, she noted, should occur “as early as possible, before any sort of solid decisions are made,” helping sponsors identify potential data or study design challenges before significant resources are committed.

The FDA expects RWE generated through the program to provide evidence capable of supporting regulatory decision-making. Potential applications include demonstrating effectiveness in new therapeutic indications, evaluating outcomes in broader patient populations, informing label expansions, or addressing regulatory commitments following product approval.

Meeting requests will be selected based on several factors, including whether the proposed data sources are fit-for-purpose, the adequacy of the study design, and the overall regulatory relevance of the proposed evidence-generation approach. While the FDA will consider a wide range of submissions, it has indicated that priority may be given to proposals that help expand diversity in data sources, study designs, analytical methodologies, diseases studied, and FDA centers involved.

One of the mains goals of the initiative is to encourage shared learning between regulators and industry regarding the use of RWE in regulatory contexts. To support this objective, the FDA may discuss study designs emerging from the program in public forums such as workshops or future guidance documents, helping to illustrate characteristics of RWE that may support regulatory decisions.

Sponsors interested in participating must hold an Investigational New Drug (IND) or pre-IND number for a medical product and submit proposals describing a pivotal RWE study intended to support a regulatory decision. Proposals focused solely on methods development or replication studies are outside the scope of the program.

The Advancing RWE Program accepts meeting requests twice a year, with submission deadlines on March 31 and September 30. The program remains open to submissions on a rolling basis through March 2027, providing sponsors with ongoing opportunities to engage with the FDA on how RWE can support product development and regulatory decision-making.