As announced in the Federal Register notice published on October 20, 2022, FDA is conducting an Advancing Real-World Evidence (RWE) Program, which seeks to improve the quality and acceptability of RWE-based approaches in support of new intended labeling claims, including approval of new indications of approved medical products or to satisfy post-approval study requirements. The Advancing RWE Program fulfills an FDA commitment under PDUFA VII, incorporated as part of the FDA User Fee Reauthorization Act of 2022.
The Advancing RWE Program provides sponsors who are selected into the Program the opportunity to meet with Agency staff—before protocol development or study initiation—to discuss the use of RWE in medical product development. The Advancing RWE Program is an optional pathway for sponsors submitting RWE proposals; established procedures to engage with the Agency will continue to be available.
Meetings will be conducted by FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) during fiscal years 2023 to 2027. Oncology applications will include participation from the Oncology Center of Excellence. To promote awareness of characteristics of RWE that can support regulatory decisions, study designs discussed through the program may be presented by FDA in a public forum (e.g., in a guidance or public workshop).
Goals of the Advancing Real-World Evidence Program
The Advancing RWE Program is designed to:
- identify approaches for generating RWE that meet regulatory requirements in support of labeling for effectiveness (e.g., new indications, populations, dosing information) or for meeting post-approval study requirements;
- develop agency processes that promote consistent decision-making and shared learning regarding RWE; and
- promote awareness of characteristics of RWE that can support regulatory decisions by allowing FDA to discuss study designs considered in the Advancing RWE Program in a public forum.
- The sponsor has an Investigational New Drug (IND) or pre-IND number for the medical product in the Advancing RWE Program meeting request.
- The proposed RWE is intended to meet regulatory requirements in support of labeling for effectiveness (e.g., new indications, populations, dosing information) or for meeting post-approval study requirements.
- The sponsor and FDA reach agreement on the study design information to be publicly disclosed.
Selection for Participation
FDA welcomes submissions related to any eligible RWE proposal. Given the limited number of requests accepted per submission cycle, however, FDA will select requests based on their potential regarding fit-for-use data, adequate study design, and appropriate regulatory conduct. Consideration will also be given to promoting diversity of data sources, study designs, analytical methodologies, and regulatory indications, as well as to diversity of diseases under study and FDA Centers and Offices involved.