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 critical healthcare questions. Regulators and policymakers recognize the need for new RWE policies to realize the full potential of data to improve patient care and outcomes.
In February 2023, FDA issued draft guidance entitled “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products.” This draft guidance provides recommendations for using externally controlled trials to demonstrate the safety and effectiveness of drugs.
In May 2019, FDA issued draft guidance entitled “Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics.” This draft guidance outlined recommendations for how a sponsor can identify a regulatory submission to FDA as including RWE. The RWE Alliance commented on the guidance in September 2021, and FDA issued final guidance in September 2022 incorporating several of the RWE Alliance’s suggestions.
In December 2021, FDA issued draft guidance entitled “Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products.” This draft guidance outlines various regulatory considerations for submissions containing RWD/E.
In November 2021, FDA issued draft guidance entitled “Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products Guidance for Industry.” This draft guidance provides recommendations for using RWD derived from data registries to support regulatory submissions.
In October 2021, FDA issued draft guidance entitled “Data Standards for Drug and Biological Product Submissions Containing Real-World Data.” This draft guidance provides recommendations for applying FDA’s currently supported data standards to submissions containing RWD.
In September 2021, FDA issued draft guidance entitled “Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products.” This draft guidance provides recommendations for using real-world evidence derived from electronic health records and medical claims data for regulatory decisions across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 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 June 2021, Representatives Diana DeGette (D-CO) and Fred Upton (R-MI) released draft follow-up legislation to the 21st Century Cures Act. The “Cures 2.0 Act” discussion draft includes several provisions related to the use of RWE in regulatory decision making, including provisions to establish a Real-World Evidence Task Force and issue guidance on the use of RWE in evaluating safety and effectiveness in various FDA approval pathways, among other policies.
PUBLIC LAW 114-255
The 21st Century Cures Act (Cures Act), enacted in December 2016, directs FDA to establish a program to evaluate the potential use of RWE to help support the approval of a new indication for an already-approved drug or satisfy drug post-approval study requirements. The Cures Act provided FDA two years to develop a draft framework to implement this 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:
FDA has indicated that more specific guidance is forthcoming.
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.
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.