Drugs

FDA Releases 4 Guides on Real-World Data/Evidence

FDA has released four draft guidances on the use of real-world data (RWD) and real-world evidence (RWE) in regulatory decision-making, specifically covering topics related to data sources, data standards, and regulatory considerations.

The draft guidance on Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products, discusses considerations for using RWD from electronic health records (EHRs) and medical claims data. FDA notes that while these specific data have been used in safety studies for some time, they are still seen as having certain limitations. “These same limitations apply, and often have a greater impact, when using RWD in effectiveness studies,” according to an agency notice. (XXISNERT LINKXX0. “Cognizant of the inherent challenges of using such data in research, investigators have been developing improved approaches to generate valid evidence.” The guidance provides recommendations on how to select relevant data sources as well as to define and validate study variables, including population eligibility criteria, treatments, and outcomes.

The draft guidance Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products addresses data obtained from health care providers or patients and how they are linked to other data sources, such as EHRs or medical claims data. It provides recommendations on designing a registry or using a pre-existing one to support an application. It also discusses challenges when linking a registry to another data source for supplemental information, including resolving inconsistencies, protecting patient privacy, and reducing redundancy.

The draft guidance Data Standards for Drug and Biological Product Submissions Containing Real-World Data discusses considerations for complying with relevant legal requirements by using FDA-supported data standards in applicable drug submissions that contain study data derived from RWD. “When standardizing variables in RWD, the guidance advises sponsors to document their rationale and to note the challenges they faced and changes they made to the data to conform to FDA-supported data standards,” the agency says. The guidance also recommends creating a “data dictionary” with data element definitions.

And the draft guidance Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drugs and Biological Products examines regulatory considerations for observational studies. The document discusses issues such as finalizing a study protocol and analysis plan before conducting the study, maintaining study records, and making sure FDA can access and verify those records.

Additionally, FDA says more guidance is expected to be released such as on designing studies that use RWD, specifically externally controlled trials and randomized controlled trials conducted in clinical practice settings.

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