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In December 2021, FDA issued draft guidance titled “Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products.” This draft guidance outlines various regulatory considerations for submissions containing RWD/E. The RWE Alliance commented on the draft guidance in March 2022, and FDA issued final guidance in August 2023.
In March 2023, FDA issued draft guidance titled “Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics.” This draft guidance provides recommendations to sponsors of oncology products on considerations for designing trials intended to support accelerated approval.
In February 2023, FDA issued draft guidance titled “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products.” This draft guidance provides recommendations for using externally controlled trials to demonstrate the safety and effectiveness of drugs.
In November 2021, FDA issued draft guidance titled “Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products Guidance for Industry.” This draft guidance provides recommendations for using RWD derived from data registries to support regulatory submissions.
In October 2021, FDA issued draft guidance titled “Data Standards for Drug and Biological Product Submissions Containing Real-World Data.” This draft guidance provides recommendations for applying FDA’s currently supported data standards to submissions containing RWD.
In September 2021, FDA issued draft guidance titled “Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products.” This draft guidance provides recommendations for using real-world evidence derived from electronic health records and medical claims data for regulatory decisions across therapeutic areas.
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.