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 Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER) 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	FY 2024	FY 2025
Protocol^a	4	15	8
New drug application (NDA)/biologics license application (BLA)^b	0^d	2	0
Final study report to satisfy a postmarketing requirement (PMR) or postmarketing commitment (PMC)^c	0^e	5	1

^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 NDA and BLA submissions are reported in the fiscal year in which FDA took regulatory action.
^c Final study reports to satisfy a PMR or PMC are reported in the fiscal year in which FDA determines that the PMR or PMC was satisfied.
^d There were no eligible NDAs or BLAs for which CBER took regulatory action in FY 2023 and FY 2025.
^e 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.

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	FY 2024	FY 2025
Effectiveness	0	3	4
Safety	4	12	4

Intended Regulatory Purpose^b	FY 2023	FY 2024	FY 2025
To support the demonstration of safety and/or effectiveness for a product not previously approved by FDA	0	0	2
To support labeling changes for an approved product, including:	0	1	2
Add or modify an indication	0	1	1
Change dose, dose regimen, or route of administration	0	0	0
Expand the labeled indication of the product to a new population	0	0	1
Add comparative effectiveness information	0	0	0
Add or modify safety information	0	0	0
To satisfy a PMR	2	10	4
To satisfy a PMC	2	4	2

	FY 2023	FY 2024	FY 2025
Electronic health records	0	8	4
Medical claims	2	5	3
Product, disease, or other registry^c	2	7	4
Digital health technologies in non-research settings	0	0	0
Other^d	0	2	2

Study Design	FY 2023	FY 2024	FY 2025
Randomized controlled trial	0	0	0
Externally controlled trial	0	0	0
Non-interventional (observational) study	4	15	7
Other	0	0	5

^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.
^d Includes patient-level data from publications and national death records.

NDAs/BLAs

The table below describes the characteristics of studies containing RWD to generate RWE submitted to CBER in an original or supplemental NDA or BLA. An NDA or BLA may include more than one relevant study. Each NDA and BLA is reported in the fiscal year during which a regulatory action is taken. There were no NDAs or BLAs containing RWD/RWE for which CBER took regulatory action in FY 2023 and FY 2025, so that column is omitted from the table below.

Primary Focus^a	FY 2024
Effectiveness	2
Safety	0

Intended Regulatory Purpose^b	FY 2024
To support the demonstration of safety and/or effectiveness for a product not previously approved by FDA	2
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

Data Source^b	FY 2024
Electronic health records	0
Medical claims	1
Product, disease, or other registry^c	1
Digital health technologies in non-research settings	0
Other^d	0

Study Design	FY 2024
Randomized controlled trial	0
Externally controlled trial	1
Non-interventional (observational) study	1
Other	0

NDA/BLA Approvals

The table below includes information on NDA/BLA approvals based, at least in part, on RWE. For additional information, see Drugs@FDA.

Approval Date	Drug Name	Company
March 18, 2024	LENMELDY	Orchard Therapeutics (Europe) Limited

Final Study Reports to Satisfy a PMR or PMC

The table below describes the characteristics of studies containing RWD to generate RWE submitted to CBER as final study reports to satisfy a PMR or PMC. Final study reports to satisfy a PMR or PMC are reported in the fiscal year during which FDA determines that the PMR or PMC was satisfied. There were no final study reports containing RWD/RWE to satisfy a PMR or PMC for which FDA took regulatory action in FY 2023, so that column is omitted from the table below.

Primary Focus^a	FY 2024	FY 2025
Effectiveness	2	0
Safety	3	1

Intended Regulatory Purpose^b	FY 2024	FY 2025
To support the demonstration of safety and/or effectiveness for a product not previously approved by FDA	0	0
To support labeling changes for an approved product, including:	0	0
Add or modify an indication	0	0
Change dose, dose regimen, or route of administration	0	0
Expand the labeled indication of the product to a new population	0	0
Add comparative effectiveness information	0	0
Add or modify safety information	0	0
To satisfy a PMR	1	0
To satisfy a PMC	4	1

Data Source^b	FY 2024	FY 2025
Electronic health records	2	0
Medical claims	2	1
Product, disease, or other registry^c	1	0
Digital health technologies in non-research settings	0	0
Other^d	1	0

Study Design	FY 2024	FY 2025
Randomized controlled trial	0	0
Externally controlled trial	0	0
Non-interventional (observational) study	5	1
Other	0	0