The US Food and Drug Administration (FDA) on Thursday finalized two guidances that detail how sponsors can leverage and submit real-world data (RWD) in their new drug and biological product submissions.
One guidance sets out the agency’s criteria for converting data derived from RWD sources such as electronic health records (EHRs) to FDA-supported data standards for new drug applications (NDAs) and biologics license applications (BLAs), while the other addresses the use of registries as a source of RWD in regulatory submissions.
Both guidance documents were issued as part of the agency’s larger real-world evidence (RWE) program and to fulfill a requirement outlined in the 21st Century Cures Act and Prescription Drug User Fee Act which requires FDA to evaluate how RWD and RWE can be leveraged as the basis for regulatory approvals in submissions.
FDA Commissioner Robert Califf on Thursday wrote that “in this rapidly evolving world with a plethora of digital data, the FDA intends to help develop the best methods for RWD and RWE in the domains of data quality, study design and analysis.” The announcement detailed the agency’s progress in implementing the RWD sections of the Cures Act.
Data standards for submissions containing RWD
The final guidance on data standards for RWD submissions addresses considerations for mapping RWD to FDA supported data standards. Such RWD can include electronic health records (EHRs), medical claims data, data from product and disease registries, patient-generated data and data gathered from mobile devices.
The final guidance contains minor revisions from a draft version issued in October 2021.
The guidance addresses some of the challenges in standardizing RWD, the processes for processes for managing RWD and considerations for mapping RWD to FDA’s data standards. These standards include the Clinical Data Interchange Standards Consortium (CDISC’s) Study Data Tabulation Model (SDTM) and the Analysis Data Model (ADaM).
An annex offers some examples of how to map health care data to the Clinical Data Interchange Standards Consortium (CDISC) terminologies.
FDA said it considered public comments into account in its finalized version.
The finalized version cites additional factors that challenge the standardization of RWD. Such challenges include the potential of using more than one RWD source in a study, such as combining electronic health records and claim data while another obstacle is that certain information, such as physician notes, only exist in non-structured documentation. The other challenge is that sponsors have varying levels of access to data sources used in the study.
Another revision adds the Study Data Technical Conformance Guidance (TCG) to supplement the Study Data Guidance as an additional resource in converting RWD into FDA data standards.
Assessing registries
FDA also finalized its guidance on the use of registries to support regulatory decisions about the safety and effectiveness of drugs or biologics. The guidance addresses a registry’s fitness-for-use and considerations when linking a registry to another data source, such as medical claims, EHRs and digital health technologies.
Changes from the 2021 draft guidance include new text stating that sponsors proposing to use registry data to support regulatory decision-making are responsible for ensuring that attributes of the registry support the collection of relevant and reliable data, including situations where the registry data are not managed by the sponsor.
The finalized version also adds text telling sponsors to have a plan to address missing assessments. As an example, “a registry should have plans for outreach to patients who do not maintain contact with the registry or have difficulty doing so (e.g., text messages, phone calls, emails, offers of transportation assistance).”
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