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 important healthcare questions. Regulators and policymakers recognize the power of policy to realize the full potential of data to improve patient care and outcomes.

Major RWE Policy Developments

FDA's AI/ML Discussion Paper

In May 2023, FDA published a discussion paper titled “Using Artificial Intelligence & Machine Learning in the Development of Drug and Biological Products” to facilitate a discussion with stakeholders on the use of artificial intelligence (AI) and machine learning (ML) in drug development and to help inform future regulatory action in this space.

LEARN MORE ABOUT THE FDA DISCUSSION PAPER SEE THE RWE ALLIANCE COMMENTS

FDA’s PDUFA VII Commitment Letter

In September 2021, FDA published the proposed commitment letter for the next reauthorization of the Prescription Drug User Fee Act, which is known as “PDUFA VII.” This letter outlines specific goals, enhancements, and other commitments for FDA’s human drug review program, including commitments for exploring the use of RWE in regulatory decision making.

LEARN MORE ABOUT PDUFA VII SEE THE RWE ALLIANCE COMMENTS

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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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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