Summary of Evolving Role and Impact of Real World Evidence (RWE) in US FDA Regulatory Approvals (2020-2024)
Author(s)
Sanjeev Sachdeva, MCA1, Jaideep K. Kaneria, MBA2, Rita Shah, Post Doctoral Fellowship, PhD1, Vikal Tripathy, PhD1, Kapil Naudiyal, BE, MBA1;
1Tata Consultancy Services, Noida, India, 2Tata Consultancy Services, Head Real World Evidence, Mumbai, India
1Tata Consultancy Services, Noida, India, 2Tata Consultancy Services, Head Real World Evidence, Mumbai, India
OBJECTIVES: 1) Analyze role of Real World Evidence in US FDA Regulatory Approvals from 2020- 2024. 2) Key approvals leveraging real world evidence 3) Summarize trends and patterns of use of RWE in drug approvals
METHODS: All novel drug approvals including NDA (New Drug Application) and BLA (Biological License Application)submitted from 2020-2024 , were analysed from Center for Drug Evaluation and Research(CDER) and Drugs@FDA. The submission packags were categorized in two mutually non-exclusive groups: a. RWE used to support approvals from therapeutic context b. RWE used to support safety and/or effectiveness.
RESULTS: 1) The percentage of approvals involving RWE grew from 5-10% in 2020 to 45-50% by 2024. 2) Therapeutic areas most included oncology, rare diseases, and neurology. 3) RWE transitioned from supplementary to a critical data source for regulatory decision-making.
CONCLUSIONS: 1) 2020, RWE was primarily used in label expansions and post-marketing safety evaluations, particularly in oncology and rare diseases. Notable approvals included Ibrance (palbociclib) for male breast cancer, based on real-world data from EHRs.2) 2021, during COVID-19 pandemic, RWE played a key role in Emergency Use Authorizations (EUAs) and full approvals of treatments like Veklury (remdesivir). The % of approvals involving RWE increased to 15-20%, with RWE supporting label expansions for drugs like Keytruda (pembrolizumab).
3)2022, RWE contributed to 25-30% of approvals, including initial NDAs and BLAs. Notable cases like Rozlytrek (entrectinib) for rare cancers leveraged synthetic control arms, Zolgensma (onasemnogene abeparvovec-xioi) used RWE for expanded indications. 3) 2023, 35-40% of approvals included RWE, integral to accelerated pathways. Examples include Enhertu (fam-trastuzumab deruxtecan-nxki) for HER2-low breast cancer and Evrysdi (risdiplam) for spinal muscular atrophy, supported by longitudinal real-world data. 4) 2024, RWE was central to nearly half of all approvals. Digital health tools and AI/ML enhanced data quality, supporting approvals like Leqembi (lecanemab-irmb) for Alzheimer’s disease and Opdivo (nivolumab) for advanced cancers.
METHODS: All novel drug approvals including NDA (New Drug Application) and BLA (Biological License Application)submitted from 2020-2024 , were analysed from Center for Drug Evaluation and Research(CDER) and Drugs@FDA. The submission packags were categorized in two mutually non-exclusive groups: a. RWE used to support approvals from therapeutic context b. RWE used to support safety and/or effectiveness.
RESULTS: 1) The percentage of approvals involving RWE grew from 5-10% in 2020 to 45-50% by 2024. 2) Therapeutic areas most included oncology, rare diseases, and neurology. 3) RWE transitioned from supplementary to a critical data source for regulatory decision-making.
CONCLUSIONS: 1) 2020, RWE was primarily used in label expansions and post-marketing safety evaluations, particularly in oncology and rare diseases. Notable approvals included Ibrance (palbociclib) for male breast cancer, based on real-world data from EHRs.2) 2021, during COVID-19 pandemic, RWE played a key role in Emergency Use Authorizations (EUAs) and full approvals of treatments like Veklury (remdesivir). The % of approvals involving RWE increased to 15-20%, with RWE supporting label expansions for drugs like Keytruda (pembrolizumab).
3)2022, RWE contributed to 25-30% of approvals, including initial NDAs and BLAs. Notable cases like Rozlytrek (entrectinib) for rare cancers leveraged synthetic control arms, Zolgensma (onasemnogene abeparvovec-xioi) used RWE for expanded indications. 3) 2023, 35-40% of approvals included RWE, integral to accelerated pathways. Examples include Enhertu (fam-trastuzumab deruxtecan-nxki) for HER2-low breast cancer and Evrysdi (risdiplam) for spinal muscular atrophy, supported by longitudinal real-world data. 4) 2024, RWE was central to nearly half of all approvals. Digital health tools and AI/ML enhanced data quality, supporting approvals like Leqembi (lecanemab-irmb) for Alzheimer’s disease and Opdivo (nivolumab) for advanced cancers.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR81
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Infectious Disease (non-vaccine), SDC: Neurological Disorders, SDC: Oncology, SDC: Rare & Orphan Diseases