Real-World Evidence Usage in Regulatory Approvals from USFDA and EMA


Shaikh J1, Samnaliev M2
1Axtria India Pvt Ltd, Hyderabad, AP, India, 2Axtria, Inc., Needham, MA, USA

Presentation Documents

OBJECTIVES: Randomized, blinded, and controlled trials are gold standard for establishing medical product effectiveness. However, conducting interventional studies may not always be feasible. In those circumstances, real-world data (RWD) can be used to support regulatory approval. This review identified and compared inclusion of real-world evidence (RWE) as part of an evidence package to support approvals from USFDA & EMA.

METHODS: USFDA &EMA submissions between 2017 and 2022 were screened to identify the usage of RWE. Details of name of drug, disease area, approval year, & manufacturer were analyzed and categorized using a pre-specified data extraction form.

RESULTS: More than 400 regulatory approvals were screened of which RWE was utilized in >50% of cases. RWE usage increased significantly from year 2017 onwards suggesting the increasing acceptance of RWE by regulatory bodies. RWE was used at a higher rate in EMA (>70% of all submissions) compared to USFDA (>25% of all submissions). RWE was mainly used as clinical evidence to evaluate safety and efficacy, additional supportive evidence, and as a future commitment for post approval studies. RWE serving as an external control arm was observed to be higher in rare diseases and oncology drugs. It was also submitted to inform epidemiological information in EMA approvals other areas of RWE usage included, leveraging RWE to better explain current treatment landscape, or evidence of drug being used off-label. Most common sources of RWE cited included claims data, registry data, medical chart review, medical records, surveys, observational studies, and published literature.

CONCLUSIONS: RWE studies are becoming more commonly accepted when ethics, orphan diseases, or enrolment challenges limit the conduct of RCTs. Robust and appropriate RWE have the potential to accelerate patient access to new technologies in diseases with a high level of unmet need.

Conference/Value in Health Info

2023-05, ISPOR 2023, Boston, MA, USA

Value in Health, Volume 26, Issue 6, S2 (June 2023)




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