The highlights the aspects to be taken into consideration in order to ensure the real-world data used to support regulatory submissions is relevant and reliable.
Table of content
The Food and Drug Administration (FDA or the Agency), the US regulating authority in the sphere of healthcare products, has published a draft guidance document dedicated to the use of real-world evidence (RWE) and real-world data (RWD) to support regulatory decision-making for medical devices.
The document provides an overview of the applicable regulatory requirements, as well as additional clarifications and recommendations to be considered to ensure compliance with them.
At the same time, provisions of the guidance are non-binding in their legal nature, nor are they intended to introduce new rules or impose new obligations.
Moreover, the authority explicitly states that an alternative approach could be applied, provided such an approach is in line with the existing legal framework. It has been agreed with the authority in advance.
Relevance and Reliability: Key Points
The scope of the guidance covers, among other things, the aspects related to data relevance and reliability. In particular, the document outlines several key considerations, including, among other things, the following:
- Relevance of RWD
The FDA emphasizes the need for RWD to be timely, available, and generalisable.
To determine the relevance of RWD for generating RWE, factors like data availability, use of the device, outcomes of interest, and covariates impacting these outcomes are considered.
The data must capture detailed information relevant to the study’s target population. Moreover, the duration and continuity of data collection are critical to capture all necessary exposures and outcomes.
If the primary RWD source lacks certain information, sponsors are advised to consider integrating supplemental data sources. - Data Linkages
In cases where multiple data sources are used, the FDA requires an assessment of how these sources can be integrated, considering the heterogeneity in patient populations, clinical practices, and coding systems.
Linkage methodologies should be predefined, scientifically valid, and privacy-compliant.
They must ensure logical consistency and accuracy in data matching, and strategies must be in place to address redundant data and resolve inconsistencies. - Timeliness of Data
The timeliness of data collection and release is crucial.
The FDA stresses that RWD should reflect the current clinical environment, considering changes in clinical practice and guidelines.
Sponsors must update the RWD promptly during the study and define reporting schedules in their regulatory submissions. - Generalizability of Data
The FDA requires that the study sample be representative of the population eligible for using the device and generalizable to the larger target population.
If the study sample does not represent a subset of the target population, additional analyses are necessary to assess the generalizability of the study findings. - Reliability of RWD
This aspect covers the consistency and systematic nature of data collection, processing, and accrual.
The FDA considers various factors for ensuring reliability, such as using recognized coding systems, data capture methods (structured or unstructured), and the adequacy of data source descriptions.
Reliability also depends on the completeness of data fields, latency, version control, and technical and privacy-related aspects. - Data Quality and Integrity
Quality control processes and the integrity of data are crucial.
The FDA looks for completeness, accuracy, and consistency in data across sites and over time. Missing data should be minimal, and sponsors are expected to assess the impact of any lost data.
Consistency in data capture across different sites and over time is vital, and any changes in diagnostic criteria or clinical practices should be documented and assessed for their impact on the study. - Patient Protections and Previous Demonstrations of RWE
Ensuring patient privacy and adhering to ethical standards is mandatory. Sponsors should provide documentation of any previous uses of the RWD source for similar populations and any peer-reviewed literature demonstrating RWE generation from the data source.
Conclusion
The present FDA document outlines the guidelines for using RWD in regulatory submissions. It highlights the importance of ensuring that the data is relevant, reliable, timely, and generalisable, emphasizing data quality, integrity, and adherence to privacy and ethical standards.
This comprehensive approach ensures that RWD is appropriately used for regulatory decision-making, particularly in the context of medical devices and treatments.
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