Analysis of Real-World Evidence Use Across FDA Submissions in 2022 and 2023
Author(s)
Elisabet Viayna, PhD1, Edward J. CaJacob III, PharmD2, M. Chris Runken, PharmD2;
1Grifols, Global HEOR & RWE, Sant Cugat del Valles, Spain, 2Grifols, Global HEOR & RWE, Durham, NC, USA
1Grifols, Global HEOR & RWE, Sant Cugat del Valles, Spain, 2Grifols, Global HEOR & RWE, Durham, NC, USA
Presentation Documents
OBJECTIVES: The 21st Century Cures Act, aimed to help accelerate medical product development and access to innovation, included the use of Real-World Evidence (RWE) to support medical products development and approval by the US Food and Drug Administration (FDA). The present study aims to describe the use of RWE among applications submitted to the FDA.
METHODS: A primary structured review of FDA submissions was performed through the FDA website and complemented with a secondary targeted review of other sources to confirm findings and complete the data extraction. Submissions including RWE during 2022 and 2023 were identified and the following data was extracted: year of approval, therapeutic area, type of submission [Biologics License Application (BLA), supplemental BLA (sBLA), New Drug Application (NDA) and supplemental NDA (sNDA)], clinical studies and RWE (historical/external control, natural history, effectiveness, safety) included in the submission and Real-World Data (RWD) source. Type of application and RWE included among submissions was descriptively analyzed.
RESULTS: Overall, 15 submissions were identified that included a RWE component during 2022-2023. Most common therapeutic areas were neurology, (33.3%, N=5), oncology (20.0%, N=3) and immunology (20.0%, N=3). Most submissions were either NDA (53.3%, N=8) or BLA (20.0%, N=3), whereas only 2 were sNDA (13.3%) and 3 were sBLA (13.3%). Most submissions presented evidence from either randomized clinical trials (73.3%, N=11) or single arm trials (13.3%, N=2), whereas only 2 (13.3%) relied on effectiveness from RWE sources only. Most common type of RWD came from prospective non-interventional studies (73.3%, N=11), followed by registries (26.7%, N=4) and electronic health records (20.0%, N=3). Effectiveness was the most common outcome assessed within the RWE component (73.3%, N=11).
CONCLUSIONS: The present study shows how RWE was used among FDA submissions over 2022-2023, mainly to support effectiveness claims in addition to efficacy and safety data coming from clinical studies.
METHODS: A primary structured review of FDA submissions was performed through the FDA website and complemented with a secondary targeted review of other sources to confirm findings and complete the data extraction. Submissions including RWE during 2022 and 2023 were identified and the following data was extracted: year of approval, therapeutic area, type of submission [Biologics License Application (BLA), supplemental BLA (sBLA), New Drug Application (NDA) and supplemental NDA (sNDA)], clinical studies and RWE (historical/external control, natural history, effectiveness, safety) included in the submission and Real-World Data (RWD) source. Type of application and RWE included among submissions was descriptively analyzed.
RESULTS: Overall, 15 submissions were identified that included a RWE component during 2022-2023. Most common therapeutic areas were neurology, (33.3%, N=5), oncology (20.0%, N=3) and immunology (20.0%, N=3). Most submissions were either NDA (53.3%, N=8) or BLA (20.0%, N=3), whereas only 2 were sNDA (13.3%) and 3 were sBLA (13.3%). Most submissions presented evidence from either randomized clinical trials (73.3%, N=11) or single arm trials (13.3%, N=2), whereas only 2 (13.3%) relied on effectiveness from RWE sources only. Most common type of RWD came from prospective non-interventional studies (73.3%, N=11), followed by registries (26.7%, N=4) and electronic health records (20.0%, N=3). Effectiveness was the most common outcome assessed within the RWE component (73.3%, N=11).
CONCLUSIONS: The present study shows how RWE was used among FDA submissions over 2022-2023, mainly to support effectiveness claims in addition to efficacy and safety data coming from clinical studies.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR77
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas