FDA drafts data standards guidance for RWD

Regulatory NewsRegulatory News | 22 October 2021 |  By 

The US Food and Drug Administration (FDA) has released draft guidance for industry that outlines the agency’s thinking on how a sponsor should submit drug and biological product study data from real-world data (RWD) sources.
FDA acknowledged that standardizing data for use as RWD is fraught with such challenges as inconsistent formats and sources, different source data captured by region, differences in terminology and exchange, a wide range of methods to build datasets for aggregation, and variations in overall data quality. However, real-world evidence (RWE) is still subject to the Federal Food, Drug, and Cosmetic Act (FD&C Act), FDA said.
The guidance offers several examples of how to map health care data to the Clinical Data Interchange Standards Consortium (CDISC) Study Data Tabulation Model (SDTM), and specifies that RWD submissions must still use data standards set forth in FDA’s Data Standards Catalog.
Because of some of the challenges that can arise in aggregating and reporting disparate data when using RWD in support of a submission, FDA encourages sponsors to contact the FDA review division that will be handling the submission early on in their process. Sponsors should be ready to give a detailed description of their proposed data transformation approach.
According to the guidance, industry should have “adequate processes” to increase confidence in RWD in effect during the data curation and data transformation stage, and these processes should be documented electronically. “Sponsors should also document in their applicable drug submission changes to data to conform to the current FDA-supported data standards, and the potential impacts of these changes,” the agency said.
Concerning disparities in language and terminology between datasets, FDA said these differences should also be documented by industry.
“Documentation of the sponsor’s rationale for choosing particular CDISC data elements for RWD and documentation of the differences between the two is critical. The sponsor should provide a description of the general approach and anticipated impact of data mapping as a part of or in an appendix to the Study Data Reviewer’s Guide to highlight the domains involved,” FDA wrote. “Furthermore, the sponsor should include a data dictionary that documents the definition of every data element used and all relevant information about the element, such as its relationships to other data, origin, usage, and format.”
The agency’s guidance is the latest in FDA’s Real-World Evidence Program, which meets the requirements outlined in the 21st Century Cures Act and Prescription Drug User Fee Act. In September, FDA released draft guidance on how to use RWD drawn from electronic health records (EHRs) and medical claims data to facilitate regulatory decision-making. (RELATED: FDA issues draft guidance on RWD sourced from EHRs, claims data, Regulatory Focus 29 September 2021)
Previously, FDA demonstrated a commitment to using RWE under the last reauthorization of the Prescription Drug User Fee Act (PDUFA VI); the recently released PDUFA VII commitment letter has signaled the agency’s intent to further expand opportunities to employ RWE in support of regulatory actions. (RELATED: FDA expands RWE demonstration project, Regulatory Focus 10 April 2019; PDUFA VII commitment letter: RWE, rare diseases see renewed attention, Regulatory Focus 23 August 2021)
“FDA plans to issue further guidance and/or to update the Catalog with standards for study data that are derived from RWD sources,” the agency wrote in the latest guidance. Comments on the draft guidance are requested by 21 December.
Data Standards for Drug and Biological Product Submissions Containing Real-World Data: Guidance for Industry
Federal Register notice


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