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.
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.
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.
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.”
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.”
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.
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.
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.
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:
FDA has indicated that more specific guidance is forthcoming.