Real-World Evidence — What Is It and What Can It Tell Us?

• Published in: The New England journal of medicine Vol. 375; no. 23; pp. 2293 - 2297
• Main Authors: Sherman, Rachel E, Anderson, Steven A, Dal Pan, Gerald J, Gray, Gerry W, Gross, Thomas, Hunter, Nina L, LaVange, Lisa, Marinac-Dabic, Danica, Marks, Peter W, Robb, Melissa A, Shuren, Jeffrey, Temple, Robert, Woodcock, Janet, Yue, Lilly Q, Califf, Robert M
• Format: Journal Article
• Language: English
• Published: United States Massachusetts Medical Society 08-12-2016
• Subjects: Clinical Trials as Topic - methods; Effectiveness; Electronic health records; Electronic medical records; Evidence-based medicine; Evidence-Based Practice; Government Regulation; Health care; Humans; Information; Observational Studies as Topic - methods; Patient safety; Prescription Fees - legislation & jurisprudence; Research Design; United States; United States Food and Drug Administration
• ISSN: 0028-4793; 1533-4406
• DOI: 10.1056/NEJMsb1609216

The FDA is developing guidance on the use of “real-world evidence” — health care information from atypical sources, including electronic health records, billing databases, and product and disease registries — to assess the safety and effectiveness of drugs and devices.
The term “real-world evidence” is widely used by those who develop medical products or who study, deliver, or pay for health care, but its specific meaning is elusive. We believe it refers to information on health care that is derived from multiple sources outside typical clinical research settings, including electronic health records (EHRs), claims and billing data, product and disease registries, and data gathered through personal devices and health applications.
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Key to understanding the usefulness of real-world evidence is an appreciation of its potential for complementing the knowledge gained from traditional clinical trials, whose well-known limitations make it difficult . . .