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.

FDA-NIH Clinical Research Glossary

In May 2024, FDA and NIH published a request for information on the “Resource on Terminology for Clinical Research,” a glossary to promote consistency in the use of clinical research terms related to innovative clinical study designs, including studies involving RWD.

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FDA & Duke-Margolis Innovative Clinical Trials Workshop

In October 2023, FDA published a request for comments on its public workshop, “Enhancing Adoption of Innovative Clinical Trial Approaches,” co-sponsored by the Duke-Margolis Center for Health Policy.

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FDA's Cross-Center Paper on AI

In March 2024, FDA published “Artificial Intelligence & Medical Products: How CBER, CDER, CDRH, and OCP are Working Together,” describing the Agency’s cross-center approach and four shared areas of focus regarding the development and use of AI in medical product development and commercialization: “Foster Collaboration to Safeguard Health;” “Advance the Development of Regulatory Approaches that Support Innovation;” “Promote the Development of Harmonized Standards, Guidelines, Best Practices, and Tools;” and “Support Research Related to the Evaluation and Monitoring of AI Performance.”

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NIH RFI on RWD

In September 2023, the National Institutes of Health (NIH) published a request for information “Inviting Comments and Suggestions on Opportunities and Challenges for the Collection, Use, and Sharing of Real-World Data (RWD) including Electronic Health Records, for NIH Supported Biomedical and Behavioral Research.”

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FDA's IT Strategy for FY24-27

In September 2023, FDA published its “Information Technology (IT) Strategy for Fiscal Years 2024 to 2027.”
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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.

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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.

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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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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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