Q: According to the program’s eligibility criteria, proposed RWE studies should be 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. Does this mean participation is limited to proposals for products that the Agency has previously approved?
A: Given the data required to support an initial approval of a new product as well as the lack of U.S. real-world data for a product not previously approved in the U.S., we anticipate that initial meeting requests selected to participate in the Advancing RWE Program will generally be those intended to support labeling for effectiveness of previously approved products or to meet post-approval study requirements.
Q: Are proposals for natural history studies eligible to participate in the Advancing RWE Program?
A: To be eligible for the Advancing RWE Program, the proposed RWE must be intended to meet regulatory requirements in support of labeling for effectiveness or for meeting post-approval study requirements. Stand-alone natural history studies are intended to track the course of the disease and therefore proposals for such studies are not eligible to participate in the Advancing RWE Program. Sponsors interested in discussing the role of natural history study data in the evaluation of potential benefits or risks of a medical product (e.g., in an externally controlled trial) should include in their Advancing RWE Program meeting request information on both the natural history study and the proposed comparative study intended to meet regulatory requirements in support of labeling for effectiveness or for meeting post-approval study requirements.
Q: Are RWE proposals intended to support a 351(k) application for a biosimilar product eligible to participate in the Advancing RWE Program?
A: The Advancing RWE Program fulfills a commitment under the seventh reauthorization of the Prescription Drug User Fee Act (PDUFA VII) and is open to products covered under PDUFA. As such, biosimilar products are not included in the program.
Q: Are RWE proposals intended to support the approval of a medical device eligible to participate in the Advancing RWE Program?
A: The Advancing Real-World Evidence program fulfills a commitment under PDUFA VII and is open to products covered under PDUFA. As such, medical devices are not included in the program.
Q: If a sponsor’s initial meeting request is selected to participate in the Advancing RWE Program, does the sponsor need to provide additional information to the Agency before the initial program meeting?
A: Information provided in the initial meeting request will serve as the basis for discussion at the initial Advancing RWE Program meeting. The Agency will provide the sponsor with preliminary comments approximately five days before the meeting, and the preliminary comments will guide the meeting discussion. The comments will focus on the general areas of data sources, study design, analytic plan, and study conduct.
Q: We do not have an active IND for our product. Is an IND required to submit an initial meeting request?
A: According to the eligibility criteria for the program, sponsors must have an IND or pre-IND number for the medical product in the Advancing RWE Program meeting request. If a sponsor does not have an active IND or a pre-IND for the product, the sponsor should request a pre-IND number. To obtain a pre-assigned number, see CDER instructions or CBER SOPP 8117.
Q: According to the Advancing RWE Program website, initial meeting requests to the program are limited to 12 pages. Does this include administrative elements, such as a submission cover page, table of contents, and references?
A: The 12-page limit described under “Content and Format of the Initial Meeting Request” applies to the 12 items required for an initial meeting request, with a two-page limit for items #1-8 and a 10-page limit for items #9-12. It is acceptable to include additional pages for a cover page, table of contents, and/or references.