FDA Use of Real-World Evidence in Regulatory Decision-Making
The FDA uses real-world data (RWD) and real-world evidence (RWE) to support regulatory decision-making across the lifecycle of medical products, including pre- and post-market evaluation of effectiveness and safety. The FDA is committed to realizing the full potential of RWD to generate RWE that can advance the development of medical products. The studies presented in the links below exemplify instances in which the Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), and the Center for Devices and Radiological Health (CDRH) applied RWE in regulatory decision-making processes since 2011. These include, but are not limited to, product approvals/authorizations, labeling changes, and assessments that determined no regulatory action was warranted. This compilation forms part of a comprehensive landscape analysis to assess the scope and frequency of RWE use in regulatory determinations across the Agency.
CDER Drug Approvals that Used Real-World Evidence
CDER Postmarketing Studies that Used Real-World Evidence
CDRH Premarket Authorizations that Used Real-World Evidence
CBER Biological Product Approvals that Used Real-World Evidence
510(k) - 510(k) premarket notification
ACE - Angiotensin-converting enzyme
AEMS – Adverse Event Monitoring System
ARB - Angiotensin receptor blocker
BLA - Biologics License Application
CBER - Center for Biologics Evaluation and Research
CDER - Center for Drug Evaluation and Research
CDRH - Center for Devices and Radiological Health
CMS - Center for Medicare and Medicaid Services
DSC - Drug Safety Communication
EHR- Electronic health records
EMR - Electronic medical records
ESA - Erythropoietin stimulating agent
GBCA - Gadolinium-based contrast agent
GLP-1 RA - Glucagon-like peptide-1 receptor agonists
HCTZ - Hydrochlorothiazide
HDE - Humanitarian device exemption
IAA - Inter-Agency Agreement
IDE - Investigational Device Exemption
MACE - Major adverse cardiac event
NDA - New Drug Application
OSE - Office of Surveillance and Epidemiology
OUS - Outside the U.S.
PLLR - Pregnancy and Lactation Labeling Rule
PMA - Premarket approval application
PPI - Proton pump inhibitor
REMS - Risk Evaluation and Mitigation Strategy
RWD - Real-World Data
RWE - Real-World Evidence
SEE – Substantial Evidence of Effectiveness
U.S. - United States
VHA - Veterans Health Administration
VTE - Venous thromboembolism