Real-World Evidence Submissions to the Center for Biologics Evaluation and Research

As part of the reauthorization of the Prescription Drug User Fee Act (PDUFA VII), FDA committed to reporting aggregate and anonymized information on submissions to the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) that contain real-world evidence (RWE).

The tables below describe submissions to CBER containing RWE that meet reporting criteria. This report is not intended to include all submissions to CBER containing analyses of real-world data (RWD). Columns will be added annually to represent submissions by fiscal year (FY) from FYs 2023 through 2027.

Overview

The table below provides an overview of submissions to CBER containing RWE by category. A study that generates RWE may be reflected in more than one category depending on the status of the study.

Category	FY 2023
Protocol^a	4
New drug application (NDA)/biologics license application (BLA)	0^b
Final study report to satisfy a postmarketing requirement (PMR) or postmarketing commitment (PMC)	0^c
^a Submission of an interventional study protocol to an IND or submission of a non-interventional study protocol to an existing IND or to a pre-IND. ^b There were no NDAs or BLAs for which CBER took regulatory action in FY 2023. ^c Does not include submissions before FY 2023. There were no final study reports to satisfy a PMR or PMC submitted in FY 2023 for which FDA took regulatory action in FY 2023. Final study reports to satisfy a PMR or PMC submitted in FY 2023 will be reported in the fiscal year in which FDA determines that the PMR or PMC was satisfied.

Protocols

The table below describes the characteristics of new protocols containing RWD as well as protocol amendments that added RWD to a study that did not previously include RWD. The numbers include submissions of interventional study protocols to an IND and submissions of non-interventional study protocols to an existing IND or to a pre-IND. The numbers do not include protocols or protocol amendments submitted only as part of a background package for a meeting with FDA. Protocols are reported in the fiscal year during which they are submitted.

Primary Focus^a	FY 2023
Effectiveness	0
Safety	4

Intended Regulatory Purpose^b	FY 2023
To support the demonstration of safety and/or effectiveness for a product not previously approved by FDA	0
To support labeling changes for an approved product, including:	0
Add or modify an indication	0
Change dose, dose regimen, or route of administration	0
Expand the labeled indication of the product to a new population	0
Add comparative effectiveness information	0
Add or modify safety information	0
To satisfy a PMR	2
To satisfy a PMC	2

Data Source^b	FY 2023
Electronic health records	0
Medical claims	2
Product, disease, or other registry^c	2
Digital health technologies in non-research settings	0
Other	0

Study Design	FY 2023
Randomized controlled trial	0
Externally controlled trial	0
Non-interventional (observational) study	4
Other	0

^a Studies often provide information on both effectiveness and safety. For this report, a study was classified as “safety” if it was conducted primarily to assess a known or potential safety risk. All other studies were classified as “effectiveness.”
^b A study may have more than one regulatory purpose or data source and therefore may be included in more than one category.
^c The term “registry” is sometimes used to refer to a non-interventional cohort study that is intended to address a specific regulatory question in a targeted population. For such studies, this report provides the original source(s) of study data.