RWE Policy Developments

A proliferation of new data sources and sophisticated analytic methods are helping researchers transform real-world data (RWD) into real-world evidence (RWE) to address critical healthcare questions. Regulators and policymakers recognize the need for new RWE policies to realize the full potential of data to improve patient care and outcomes.

Major RWE Policy Developments

FDA's RWD/E Draft Submissions Guidance

In May 2019, FDA issued draft guidance entitled “Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics.” This draft guidance outlines recommendations for how a sponsor can identify a regulatory submission to FDA as including RWE.

LEARN MORE ABOUT THE 2019 FDA DRAFT GUIDANCE SEE THE RWE ALLIANCE COMMENTS


Cures 2.0 Discussion Draft

In June 2021, Representatives Diana DeGette (D-CO) and Fred Upton (R-MI) released draft follow-up legislation to the 21st Century Cures Act. The “Cures 2.0 Act” discussion draft includes several provisions related to the use of RWE in regulatory decision making, including provisions to establish a Real-World Evidence Task Force and issue guidance on the use of RWE in evaluating safety and effectiveness in various FDA approval pathways, among other policies.

LEARN MORE SEE THE RWE ALLIANCE COMMENTS


21st Century Cures Act

PUBLIC LAW 114-255
The 21st Century Cures Act (Cures Act), enacted in December 2016, directs FDA to establish a program to evaluate the potential use of RWE to help support the approval of a new indication for an already-approved drug or satisfy drug post-approval study requirements. The Cures Act provided FDA two years to develop a draft framework to implement this program.

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Framework for FDA’s RWE Program

In December 2018, FDA released its Framework for using RWD and RWE in regulatory decision making for drugs and biological products. The Framework defines RWD and RWE and establishes a “three-part approach” for incorporating this information into regulatory decision making for drugs and biological products:

  • Whether the RWD are “fit for use” in regulatory decision making;
  • Whether the methodologies used to generate RWE can provide “adequate scientific evidence” to address the regulatory questions presented; and
  • Whether the approach used in a particular case meets FDA’s regulatory requirements, such as established standards for data collection and study monitoring.

FDA has indicated that more specific guidance is forthcoming.

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Guidance on the Use of RWE to Support Regulatory Decision Making for Medical Devices

In August 2017, FDA issued guidance to clarify how it will treat RWD to determine whether they are sufficient for generating RWE that can be used to support regulatory decision making for medical devices.

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Examples of RWE Used in Medical Device Regulatory Decisions

In March 2021, FDA released a summary document containing 90 examples of submissions in which RWE was used to support regulatory decision making for medical devices.

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