P341: Characterization of Submissions with Real-World Evidence to FDA’s Center for Drug Evaluation and Research, Fiscal Year 2025
Poster Presenter
Motiur Rahman
Senior Epidemiologist & Policy Advisor, Real World Evidence Analytics, OMP, CDER
FDA United States
Objectives
To provide interim reporting and characterization of submissions to FDA’s Center for Drug Evaluation and Research (CDER) that contain real-world evidence (RWE) in fiscal year 2025 (FY25) in accordance with the seventh reauthorization of the Prescription Drug User Fee Act (PDUFA VII)
Method
To identify studies for public reporting, we screened FY25 submissions for studies that contained real-world data (RWD). Studies were then compared with PDUFA criteria for reporting RWE and verified as being eligible. Lastly, studies were classified according to categories developed for reporting.
Results
A total of 731 submissions have been screened and 344 unique studies were identified as potentially containing RWD to generate RWE. Of these, 130 studies (37.8%) were deemed ineligible for public reporting because studies were not confirmed to include an effectiveness or safety endpoint (n=7; 5.4%) or studies did not contain RWD that repurposes existing data to address a question about the effectiveness or safety of a therapeutic product (n=123; 94.6%). Of the 214 studies (62.2%) confirmed to contain RWD and potentially eligible for public reporting, 34 (15.9%) were associated with an original or supplemental New Drug Application (NDA) or Biologics License Application (BLA). Submissions containing RWD to satisfy a Postmarketing Requirement (PMR) or Postmarketing Commitment (PMC) (n=112; 52.3%) were further analyzed and 47 (22.0%) found to be pregnancy-related. In addition, of the 68 (31.8%) studies that were not associated with an NDA, BLA, PMR, or PMC, 42 (19.6%) addressed effectiveness and 26 (12.1%) addressed safety.
The PDUFA VII criteria for public reporting will be applied to studies containing RWD that generate RWE to identify studies for inclusion in the FY25 report. Two subject-matter experts (SMEs) will review each candidate study and apply the PDUFA VII criteria. Preliminary results indicate that submissions to CDER containing RWE in FY25 include approximately 31 study protocols, 10 studies that are intended to or do provide substantial evidence of effectiveness or safety information to support approval of an NDA or BLA, and six final study reports that satisfy a PMR or PMC. Studies in each of these 3 categories will be further sub-categorized by primary focus, intended regulatory purpose, data source, and study design. In addition, there were at least five NDA/BLA approvals in FY25 based, at least in part, on RWE, compared with two in FY23 and one in FY24.
Conclusion
FDA is committed to reporting aggregate and anonymized information describing submissions containing RWE to the Agency. When completed, the report will describe submissions containing RWD submitted to CDER in FY25. The studies will be characterized by categorizing each by primary focus, intended regulatory purpose, data source, and study design. The FY25 report will provide a comparison point to reported data released in FY23 and FY24. Preliminary results currently indicate an increase in submissions containing RWE to CDER in FY25 compared to both FY23 and FY24. The reasons for this possible increase may be due to multiple factors including increased sophistication in regulatory science and acceleration in the collection and analyses of RWD. The potential increase in studies using RWD submitted to CDER in FY25 may also be due to sponsors’ increased aptitude and enthusiasm for the use of RWD to generate RWE. Further research and data from additional years is necessary to identify trends and determine potential causes of any patterns observed. Characterization of submissions containing RWD over a period of multiple years may help improve understanding of the types of submissions and their regulatory impact.
Public reporting of these data reflects FDA’s commitment to transparency and continued public engagement. The Agency is also committed to realizing the full potential of fit-for-use RWD to generate RWE that will advance the development of therapeutic products and strengthen regulatory oversight of drugs and biologics across their lifecycle. Together with other efforts by FDA, these data contribute to a comprehensive landscape analysis to assess the scope and frequency of RWE in regulatory determinations across the Agency.
