SUCCESSFUL USES OF REAL-WORLD EVIDENCE IN FDA LABEL UPDATES: A DESCRIPTIVE ANALYSIS OF REGULATORY TRENDS
Author(s)
Navneet Kumar, PhD1, Rachel Gamburg, BSc2, Harshit Dixit, MBA1, Jared Miller, MSc3, Coby Martin, MSc4, Jatinder Kumar, MPharm1.
1Axtria India Pvt Ltd, Gurugram, India, 2Axtria Inc, Boston, MA, USA, 3Axtria Inc, Berkeley Heights, NJ, USA, 4Axtria Inc, Toronto, ON, Canada.
1Axtria India Pvt Ltd, Gurugram, India, 2Axtria Inc, Boston, MA, USA, 3Axtria Inc, Berkeley Heights, NJ, USA, 4Axtria Inc, Toronto, ON, Canada.
OBJECTIVES: Regulatory use of real-world evidence (RWE) by the U.S. Food and Drug Administration (FDA) has increasingly been utilized in drug approvals and label updates, although many manufacturers face difficulties. Furthermore, the role of RWE in successful label updates has not been characterized. This study describes successful RWE studies that have been positively viewed by the FDA.
METHODS: A descriptive analysis was conducted using FDA regulatory decisions supported by RWE. Regulatory descriptions were systematically abstracted into standardized variables, and analyzed to capture therapeutic modality, therapeutic area, real-world data (RWD) source, study type, and regulatory role of RWE.
RESULTS: The analysis included 35 (10 biologics, 19 small molecules, 6 vaccines) instances where RWE was used in label updates, out of 57 total successful cases. Therapeutic area varied with infectious diseases representing the most successful cases of label updates (23%), followed by cardiology (14%) and hematology (14%). Study designs included retrospective cohort (37%), prospective registry (23%), and descriptive (20%) studies, and RWD sources included program based RWD (40%), registries (23%), and administrative / surveillance data (17%). Within retrospective cohort studies, program-based RWD (46%) was the most common data source followed by administrative / surveillance data (31%). Specifically, data types comprise Medicare claims, medical and pharmacy claims, Sentinel System, and electronic medical records. In two cases specifically for biologic gene therapies, an external control arm was developed using medical records, prompting the FDA to claim substantial evidence of effectiveness was shown. In addition, a slow increase in acceptance of RWE for label updates was observed initiating in 2017.
CONCLUSIONS: Label updates have specifically proven to be challenging with RWE. This analysis represents successful cases positively viewed by the FDA, establishing a preferred benchmark, and providing strategies for manufacturers in implementing RWD successfully.
METHODS: A descriptive analysis was conducted using FDA regulatory decisions supported by RWE. Regulatory descriptions were systematically abstracted into standardized variables, and analyzed to capture therapeutic modality, therapeutic area, real-world data (RWD) source, study type, and regulatory role of RWE.
RESULTS: The analysis included 35 (10 biologics, 19 small molecules, 6 vaccines) instances where RWE was used in label updates, out of 57 total successful cases. Therapeutic area varied with infectious diseases representing the most successful cases of label updates (23%), followed by cardiology (14%) and hematology (14%). Study designs included retrospective cohort (37%), prospective registry (23%), and descriptive (20%) studies, and RWD sources included program based RWD (40%), registries (23%), and administrative / surveillance data (17%). Within retrospective cohort studies, program-based RWD (46%) was the most common data source followed by administrative / surveillance data (31%). Specifically, data types comprise Medicare claims, medical and pharmacy claims, Sentinel System, and electronic medical records. In two cases specifically for biologic gene therapies, an external control arm was developed using medical records, prompting the FDA to claim substantial evidence of effectiveness was shown. In addition, a slow increase in acceptance of RWE for label updates was observed initiating in 2017.
CONCLUSIONS: Label updates have specifically proven to be challenging with RWE. This analysis represents successful cases positively viewed by the FDA, establishing a preferred benchmark, and providing strategies for manufacturers in implementing RWD successfully.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR122
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas