As defined by FDA, real-world data (RWD) are the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources and real-world evidence (RWE) is the clinical evidence regarding a medical product’s use and potential benefits or risks derived from analysis of RWD.
Collection and analysis of RWD continue to expand in the biomedical community, accompanied by a growing understanding of RWD’s strengths and limitations. Increased availability of reliable and relevant RWD is leading to the generation of RWE that can potentially inform regulatory decisions regarding the safety and effectiveness of medical therapies. Such regulatory decisions include whether to approve a drug, what that drug’s indication(s) should be, and what safety protocols (if any) are necessary to ensure safe use of the product.
To address congressional mandates and commitments, FDA issued a series of draft RWE guidances in late 2021. There are plans for FDA to publish more in the future.
PDUFA VII commitments encourage the FDA to advance the use of RWE for use in regulatory decision-making. In particular, the Advancing RWE Program seeks to improve the quality and acceptability of RWE-based approaches in support of new intended labeling claims, including approval of new indications of approved medical products or to satisfy post-approval study requirements.
The RWE Subcommittee (SC) of CDER’s Medical Policy and Program Review Council was established to serve as a cross-cutting forum for RWE issues and focuses on the evaluation of RWE and guides policy development. This multi-discipline subcommittee includes leadership and representatives from across CDER Offices, CBER, CDRH, and OCE.
Staff from the CDER, CBER, and OCE can consult with the RWE SC when evaluating the use of RWD and RWE. The RWE SC provides advisory recommendations as needed on whether underlying data, methods, and other study design elements are appropriate to provide support for a regulatory decision. OMP’s RWE Analytics Staff serve as primary clinical advisors for RWE submissions to facilitate consistency across CDER and CBER.
To promote shared learning and understanding with external stakeholders, FDA continues to oversee and support numerous demonstration (i.e., research) projects through multiple venues and programs including the broad agency announcements, funding opportunity announcements, CERSI grants, and inter-Agency agreements.
A list of FDA-funded RWD/RWE projects can be found on our demonstration projects.
- Framework for FDA’s Real-World Evidence Program (2018)
- Use of Electronic Health Records in Clinical Investigations (2018)
- Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products (2021)
- Data Standards for Drug and Biological Product Submissions Containing Real-World Data (2021)
- Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products (2021)
- Digital Health Technologies for Remote Data Acquisition in Clinical Investigations (2022)
- Submitting Documents Utilizing Real-World Data and Real-World Evidence to FDA for Drugs and Biologics (2022)
- Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products (2023)
- Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products (2023)
- July 2021: FDA approves new use of transplant drug based on RWE
- April 25, 2023: Emulation of Randomized Clinical Trials with Nonrandomized Database Analyses
- May 5, 2022: Real-World Evidence- Where Are We Now?
- Sep 2020: Randomized, observational, interventional, and real-world-What’s in a name?
- Oct 2019: CTTI Recommendations: Use of Real-World Data to Plan Eligibility Criteria and Enhance Recruitment
- March 2019: The US FDA’s Real-World Evidence Framework: A Commitment for Engagement and Transparency on Real-World Evidence
- Sep 2018: Real-World Evidence and Real-World Data for Evaluating Drug Safety and Effectiveness
The table lists public training resources, including webinars, presentation, and workshops.
For more information on RWE, please visit the individual Center pages or contact CDERMedicalPolicy-RealWorldEvidence@fda.hhs.gov.