Real-World Data Guidance on Using Registries
FDA has issued a draft guidance on Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products. The document provides sponsors with considerations about designing a registry or proposing to use an existing registry to support a regulatory decision about a drug’s effectiveness or safety.
The guidance addresses a registry’s “fitness-for-use” in regulatory decision-making, and focuses on registry attributes that support the collection of relevant and reliable data. It also discusses considerations when linking a registry to another data source for supplemental information (e.g., medical claims data, electronic health records (EHR), digital health technologies, or another registry).
According to the guidance, sponsors should consider both the strength and limitations of using registries as a source of data to generate evidence for regulatory decision-making. “Registries may have advantages over other RWD [real-world data] sources, given that registries collect structured and predetermined data elements and can offer longitudinal, curated data about a defined population of patients and their corresponding disease course, complications, and medical care,” the document says. “In addition, registries can systematically collect patient-reported data that medical claims datasets or EHR datasets may lack.”
Additionally, the guidance cautions that registries can have limitations for use in a regulatory context. “For example,” it says, “existing registries may focus on one disease, with limited information on comorbid conditions, even after linkage to other data sources. In addition, the enrolled patients may not be representative of the target population of interest due to challenges related to patient recruitment and retention. For example, patients with more severe disease may be more likely to be enrolled in a registry compared to patients with milder disease; or enrolled patients might have different self-care practices, socioeconomic backgrounds, or levels of supportive care versus the entire population of interest. These issues can potentially introduce bias into analyses that make use of registry data.”
Other potential limitations of registries involve issues with data heterogeneity (e.g., different clinical characteristics across various populations) and variation in approaches used to address data quality, according to the guidance. FDA says that in general, registries are “better suited as a data source for regulatory purposes when sponsors aim to capture objective endpoints, such as death or hospitalization. Subjective endpoints, such as pain, can be collected in a registry, but additional challenges are involved to standardize such measurements. In addition, a registry that is designed to collect data to answer a specific research question can have advantages over an existing registry designed for another purpose, which is subsequently repurposed for that same question.”