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Real-World Evidence Submissions to the Center for Drug 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 CDER containing RWE that meet reporting criteria. This report is not intended to include all submissions to CDER 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 CDER containing RWE by category. A study that generates RWE may be reflected in more than one category depending on the status of the study.
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 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.
The table below describes the characteristics of studies containing RWD to generate RWE submitted to CDER 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. Counts for FY 2025 include submissions approved by FDA even when RWE was included but did not contribute to FDA's decision. The ten submissions for FY 2025 include (a) four approvals where RWE contributed to FDA's decision (ACETADOTE (acetylcysteine), ONAPGO (apomorphine), ISTURISA (osilodrostat), and GAMIFANT (emapalumab-lzsg)), (b) one approval where RWE did not contribute to FDA's decision (VYKAT XR (diazoxide choline)), (c) four complete responses, and (d) one refuse to file.
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
December 21, 2022
Actemra (tocilizumab)
Genentech
April 28, 2023
Vimpat (lacosamide)
UCB Inc.
February 13, 2024
Aurlumyn (iloprost)
Eicos Sciences, Inc.
November 26, 2024
Acetadote (acetylcysteine)
Cumberland
February 3, 2025
Onapgo (apomorphine)
MDD US Operations
April 15, 2025
Isturisa (osilodrostat)
Recordati
June 27, 2025
Gamifant (emapalumab-lzsg)
Swedish Orphan Biovitrum
Final Study Reports to Satisfy a PMR or PMC
The table below describes the characteristics of studies containing RWD to generate RWE submitted to CDER 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 to satisfy a PMR or PMC submitted in FY 2023 for which FDA took regulatory action in FY 2023, so that column is omitted from the table below.
aStudies 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.” bA study may have more than one regulatory purpose or data source and therefore may be included in more than one category. cThe 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. dIncludes patient-level data from publications and national death records.
