At the end of 2021, the United States Food and Drug Administration (FDA) issued two draft guidance documents that outline the agency’s recommendations for using real-world data.
Assessing Registries
The first draft guidance, Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products Guidance for Industry, outlines the FDA’s recommendations for using real-world data (RWD) from registries and curating RWD in registries to support regulatory decision making.
FDA issued this draft guidance as part of a series of guidance documents to satisfy the Cures Act mandate and the PDUFA VI commitment.
In the guidance, FDA covers a registry’s fitness-for-use for regulatory decision-making, focusing on attributes of a registry that support the collection of relevant and reliable data; considerations when linking a registry to another data source, such as data from medical claims, electronic health records, digital health technologies, or another registry; and considerations for supporting FDA review of submissions, including registry data.
Using Registry Data to Support Regulatory Decisions
One of the topics covered in the draft guidance include using registry data to support regulatory decisions. In the document, FDA notes that sponsors should consider the strength and limitations of using registries as a source of data to generate evidence for regulatory decision making, and that before using any RWD for regulatory decision-making, sponsors should consider whether the data are fit-for-use by assessing the data’s relevance and reliability.
Relevance of Registry Data
In the draft guidance, FDA notes that the assessment of the data’s relevance is context-dependent. In particular, the inclusion and exclusion criteria used to enter patients into a registry may result in the patient population in a registry study differing from the target population for the sponsor’s drug development program.
Reliability of Registry Data
With respect to reliability of registry data, FDA notes that it considers whether the registry personnel and processes in place during data collection and analysis provide adequate assurance that errors are minimized and that data integrity is sufficient. Sponsors should address whether the registry has privacy and security controls in place to ensure that the confidentiality and security of data are preserved.
The draft guidance, when finalized, will represent the current thinking of FDA on “Real-World-Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products.” It does not establish any rights for any person and is not binding on FDA or the public. Stakeholders will still be able to use an alternative approach, provided it satisfies the requirements of the applicable statutes and regulations.
Comments
FDA asks that stakeholders submit any relevant comments in response to the Guidance by February 28, 2022.
Considerations for Use of RWD and RWE
The second draft guidance was issued on December 9, 2021. The document, Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products, discusses the applicability of FDA’s investigational new drug application (IND) regulations to various clinical study designs that utilize real-world data (RWD), and clarifies the Agency’s expectations regarding clinical studies using RWD submitted to FDA in support of a regulatory decision regarding the effectiveness or safety of a drug ( e.g., as part of a new drug application or a biologics license application) that are not subject to the IND regulations.
More specifically, the draft guidance discusses the following major topics: (1) Applicability of 21 CFR part 312 to studies using RWD and (2) regulatory considerations for non-interventional (observational) studies using RWD. Topics covered under regulatory considerations include the following: (1) Transparency for data collection and analysis, (2) data access, (3) study monitoring, (4) safety reporting, and (5) sponsor responsibilities.
Comments
Stakeholders may submit comments online or via USPS. FDA recommends all comments be submitted by March 8, 2022.