Real World Data and the Regulatory Process

1. Prescription Drug User Fee Act (PDUFA).…/UCM511438.pdf
2. Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices.…/ucm513027.pdf
3. Real-World Evidence — What Is It and What Can It Tell Us? Sherman RE, Anderson SA, Dal Pan GJ, Gray GW, Gross T, Hunter NL, et al. N Engl J Med2016; 375:2293-2297 DOI: 10.1056/NEJMsb1609216


Real world evidence (RWE) is continuing to gain traction—we now see its potential to become an integral part of the drug approval process. In the PDUFA VI proposed commitment letter, which will cover fiscal years (FY) 2018-2022, FDA has committed to explore the use of real world evidence for use in regulatory decision-making:

“As we participate in the current data revolution, it is important that FDA consider the possibilities of using so-called “real world” data as an important tool in evaluating not only the safety of medications but also their effectiveness. To accomplish this will require an understanding of what questions to ask, including how such data can be generated and used appropriately in product evaluation, what the challenges are to appropriate generation and use of these data, and how to address such challenges.”1

Activities planned by FDA to accomplish their goals call for holding public workshops with key stakeholders, including representatives of the biopharmaceutical industry by the end of FY 2018, and launch of appropriate activities such as pilot studies to address key outstanding questions. They have also committed to issuing draft guidance on the use of RWE for regulatory decision making by end of FY 2021. Similar language appears in the FDA draft guidance entitled Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices.2

A December 2016 publication in NEJM by Sherman and colleagues provides an interesting commentary on the importance of creating a standardized definition of RWE, and cautions on some of the key factors for consideration to ensure that data are collected and analyzed using rigorous methodologies.3

Note: PDUFA V, which was reauthorized in 2012, expires at the end of September 2017.

To access the FDA documents, please visit the links below: [mepr-show if=”rule: 4078″]

PDUFA VI FINAL Committment Letter
FDA draft guidance RWE and devices
Sherman et al can be found at:


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