RARE Daily

FDA Issues Draft Guidance on Use of Registries to Support Regulatory Decision-Making

November 30, 2021

The U.S. Food and Drug Administration has released a draft guidance for industry on the use of registries to support its decision-making on drugs and biologics as part of its real-world evidence program.

The agency said registries have the potential to support development of therapies, and registry data can inform the design and support the clinical trials and observational studies. This can include such things as characterizing the natural history of a disease, providing information that can help define endpoints for clinical studies, identifying biomarkers, and serving as an external control arm.

The guidance considers the suitability of registry data for use in regulatory decision-making. It said sponsors should consider the regulatory purpose of the data, the patients enrolled, and how the data is collected, maintained, and curated. Before using any real-world data for regulatory decision-making, the agency said sponsors should consider whether the data are fit-for-use by assessing the data’s relevance and reliability.

When considering using an existing registry or establishing a new registry, sponsors should ensure there are processes and procedures to govern registry operation, education and training of registry staff, resource planning, and general practices that help ensure the quality of the registry data. Such governance attributes help ensure that the registry can achieve its objectives and ensure the consistency, accuracy, and completeness of data.

When registries don’t capture all of the data to answer a specific question of interest, the agency said sponsors may consider seeking supplemental information from another source such as another registry, an electronic health record, medical claims database, or through the use of digital health technologies.

Before using a specific registry as part of a study to support regulatory decision-making, the agency said sponsors should meet with the FDA.

Anyone wishing to submit comments to the agency on the draft guidance should do so by February 28, 2022. Instructions for submitting comments can be found in the Federal Register notice from the FDA.  

Author: Rare Daily Staff

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