Alexandra Reid

5 Key Points in FDA’s Real-World Evidence Draft Guidance

FDA Real-World Evidence Draft GuidanceThe FDA is making significant strides in integrating real-world data (RWD) into the regulatory framework for medical devices. A recent 40 page draft guidance, “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices” issued by the FDA elaborates on how real-world evidence (RWE) can be effectively used in regulatory decision-making for medical devices.

There is more recent interest in using RWE for regulatory decision-making in the pre-market setting, including to support a determination regarding a medical product’s effectiveness and inform benefit-risk analysis of devices for new intended uses,” notes Dr. Robert M. Callif, Commissioner of Food and Drugs, in a recent blog. “Realizing the full potential of RWE in regulatory decision-making presents several challenges, and work is underway at the FDA to better understand this area.”

Now is an ideal opportunity to familiarize yourself with this draft guidance. This blog outlines five key points to help understand its potential impact.

Quality and Relevance of Real Word Data

The FDA’s draft guidance places a strong emphasis on the quality and relevance of real-world data (RWD) in regulatory decision-making. RWD encompasses a wide array of data sources like electronic health records, medical claims, postmarket surveillance, and digital health technologies. It provides a more comprehensive view of a medical device’s performance in real-world settings. The FDA underscores the importance of ensuring that RWD is accurate, complete, and systematically collected data.

This approach acknowledges the diverse nature of RWD sources and the potential they hold for improving regulatory processes. By leveraging high-quality RWD (e.g., data collected and processed in a consistent and methodical manner), the FDA aims to enhance its understanding of medical device safety and efficacy, leading to more informed regulatory decisions that reflect the nuances of everyday healthcare practices.

Fit-for-Purpose Assessment

The concept of ‘fit-for-purpose’ is central to the FDA’s guidance, serving as a benchmark to evaluate the suitability of RWD for regulatory decisions. This assessment involves a thorough evaluation of the relevance of submitted RWD to the specific regulatory context and its reliability. The FDA looks for data that is not only relevant to the health status of patients and the delivery of healthcare but also reliable enough to generate credible real-world evidence (RWE).

The manner of collection may differ for newly developed RWD sources which are actively collecting data (e.g., data dictionary to provide a common definitional framework in a registry), using nationally or internationally recognized coding systems (e.g., International Classification of Diseases, Tenth Revision, Clinical Modification (ICD-10-CM), Logical Observation Identifiers, Names, and Codes (LOINC), UDI, Current Procedural Terminology (CPT) in EHR or Claims), custom-designed structured data capture (e.g., data capture within a device), or unstructured data capture (e.g., narrative portion of clinical notes).

This assessment includes examining methods of data collection, consistency and completeness of data, and the data alignment with the regulatory objectives. A rigorous fit-for-purpose assessment ensures that RWE derived from RWD is robust and capable of supporting regulatory submissions, thus enhancing the quality of submissions and decision-making processes.

Study Design Using Real Word Data

The FDA’s guidance acknowledges the versatility of RWD in supporting various study designs ranging from randomized controlled trials to observational studies. This flexibility allows design of studies that are most appropriate for the specific questions at hand. The guidance encourages sponsors to consider innovative study designs that leverage the strengths of RWD, such as the study’s ability to capture data across diverse patient populations and clinical settings. The guidance also emphasizes the need for careful planning to minimize biases inherent in RWD, such as those related to data collection methods or patient selection. By promoting thoughtful and well-designed studies that use RWD, the FDA fosters a more nuanced and comprehensive evaluation of medical devices.

International Alignment

The FDA’s approach to RWD aligns with international regulatory trends and standards, particularly those set forth by the International Medical Devices Regulators Forum (IMDRF). This alignment is crucial in an increasingly globalized medical device market, where consistent regulatory standards are essential for ensuring patient safety and efficient market access. By aligning its guidance with international standards, the FDA not only fosters consistency in regulatory practices but also facilitates a more collaborative and harmonized regulatory environment. This alignment benefits device sponsors by reducing regulatory discrepancies and streamlining the approval process for devices marketed in multiple jurisdictions.

Documentation and Reporting

In its guidance, the FDA underscores the importance of comprehensive documentation and reporting in regulatory submissions that include RWD. This emphasis ensures transparency and accountability in how RWD is used to support regulatory decisions. Sponsors are expected to provide detailed documentation on data sources, methods of data collection, data quality controls, and the rationale behind the choice of data. This requirement for thorough documentation not only ensures the integrity of the regulatory process but also aids in the reproducibility and verification of the results. By mandating detailed documentation, the FDA is enhancing the credibility and reliability of the RWE used in regulatory decisions.

What does this mean for Medical Device Sponsors?

The integration of RWD into regulatory processes is a game-changer for medical device manufacturers. It offers a more streamlined approach to demonstrating the safety and effectiveness of new devices or expanding indications for existing ones. By leveraging RWD, sponsors can potentially expedite the regulatory review process and reduce the time and resources required for traditional clinical trials. There are several examples in Appendix B of the guidance that describe some situations where RWE may be used to support regulatory decisions, with additional examples listed in Examples of Real-World Evidence (RWE) Used in Medical Device Regulatory Decisions.

While the FDA’s draft guidance is a significant step, it is part of a broader shift towards evidence-based regulation using real-world data. Future pilot programs and updates to the guidance will likely refine the use of RWD, enhancing the efficiency and effectiveness of medical device regulation. As the medical device industry continues to evolve, this guidance will likely play a key role in shaping the future of timely medical device approvals and successful market introductions.

Alexandra (Sandy) Reid is a StarFish Medical QA/RA specialist. She brings years of experience working in the industry. Sandy supports the QA/RA team in helping clients develop and deliver FDA Approved or Cleared Medical Devices.


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