FDA invites sponsors to seek early RWE advice through Advancing Real-World Evidence Program

The US Food and Drug Administration has set September 30, 2026 as the next submission deadline for its Advancing Real-World Evidence Program, which allows selected sponsors to discuss pivotal studies using real-world data with the agency before study designs are finalized.

The Baseline

• The FDA has set September 30, 2026 as the next submission deadline for its Advancing Real-World Evidence Program.
• The program provides selected sponsors with early regulatory engagement on pivotal studies using real-world data.
• Eligible studies must be intended to support regulatory decision-making, with the final submission deadline scheduled for March 31, 2027.

The FDA has announced September 30, 2026, as the next submission deadline for its Advancing RWE Program, which allows selected sponsors to discuss the use of real-world data (RWD) and real-world evidence (RWE) in medical product development with the agency before study designs are finalized.

Established in 2022 under the Prescription Drug User Fee Act (PDUFA) VII, the program is intended to inform the development of RWE approaches used to support regulatory decisions. These may include new indications, patient populations, or dosing information for approved products, as well as post-approval study requirements.

Selected sponsors can meet with multidisciplinary teams from FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER), with the Oncology Center of Excellence participating in relevant oncology applications. FDA conducts an initial meeting for accepted requests, with sponsors able to request up to three follow-up meetings.

The program’s focus on discussions before study designs are finalized is consistent with broader FDA recommendations concerning RWE studies and the use of external controls. FDA officials have repeatedly urged sponsors, including during public speaking events, to engage with the agency early while study designs are still being developed. Speaking at ISPE 2026 about FDA’s 2023 draft guidance on externally controlled trials, Motiur Rahman (FDA) emphasized regulatory engagement during study development, the use of fit-for-purpose and comparable data, and prespecified approaches to external control selection and analysis. “Come and talk to us, please,” he said. 

FDA has previously cited regulatory decisions in which RWE contributed to the evidence considered. Examples include a new lung transplant indication for Prograf (tacrolimus), supported in part by a non-interventional registry study; Voxzogo (vosoritide) for achondroplasia, where registry data were used to construct external controls; and a new indication for Orencia (abatacept) to prevent acute graft-versus-host disease, which was also supported by registry data.

To participate in the Advancing RWE Program, sponsors must have an Investigational New Drug (IND) or pre-IND number and propose a pivotal study intended to meet regulatory requirements. Studies focused solely on methods development or replicating pivotal studies are not eligible.

FDA will select between one and four primary requests and up to four alternates per submission cycle through fiscal year 2027. Selection considers fit-for-use data, adequate study design, and appropriate regulatory conduct, as well as diversity in data sources, analytical methodologies, study designs, diseases, and regulatory indications. Initial requests are limited to 12 pages and should describe the rationale for using RWE, proposed study design, data sources, and analytical approach, including how potential confounding and missing or misclassified data will be addressed.

Study designs considered through the program may subsequently be discussed in FDA guidance, workshops, or other public forums, with information for disclosure agreed with sponsors in advance. Sponsor identities, treatments of interest, complete eligibility criteria, and patient-level data are excluded. The September 30 deadline will be followed by the program’s final scheduled submission deadline on March 31, 2027.