FDA releases new guidance for drug and biological product data

The US Food and Drug Administration (FDA) is announcing the availability of a draft guidance for industry entitled “Data Standards for Drug and Biological Product Submissions Containing Real-World Data.”

The 21st Century Cures Act was signed in 2016 and is intended to accelerate medical product development and bring innovations faster and more efficiently to the patients who need them. Among other provisions, the 21st Century Cures Act added section 505F to the Federal Food, Drug, and Cosmetic Act (FD&C Act).

This guidance provides recommendations to sponsors to help support compliance with the FD&C Act when submitting study data derived from real-world data (RWD) sources in applicable regulatory submissions using standards specified in the Data Standards Catalog (Catalog). FDA is publishing this draft guidance as part of a series of guidance documents under its program to evaluate the use of real-world evidence (RWE) in regulatory decision making.

The guidance addresses considerations for the use of data standards currently supported by FDA in applicable drug submissions containing study data derived from RWD sources. Examples of RWD  include data from electronic health records (EHRs); medical claims data, data from product and disease registries; patient-generated data, including data from in-home-use settings; and data gathered from other sources that can inform on health status, such as mobile devices.

One of the recommendations includes the documentation of data. The FDA state that, during data curation and data transformation, adequate processes should be in place to increase confidence in the resultant data. Documentation of these processes may include but are not limited to electronic documentation (such as metadata-driven audit trails, quality control procedures, etc.) of data additions, deletions, or alterations from the source data system to the final study analytic data set(s). 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 FDA have also opened submission of comments before it begins work on the final version of the guidance. The comment period will end on 21 December 2021.