08/02/2024
On July 25, 2024 the US Food and Drug Agency (FDA) published a final guidance for industry “Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products.” The 21st Century Cures Act (Cures Act) of 2016 amended the Federal Food, Drug, and Cosmetic Act (FD&C Act) which required the FDA to release a guidance on how to use Real-World Evidence (RWE) (for more information please se the FDA’s “Real-World Evidence Program”) in regulatory decision making. RWE is defined in the guidance as clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of Real-World Data (RWD), RWD being the data relating to patient health status or the delivery of health care routinely collected from a variety of sources. This guidance is intended to help sponsors and other interested parties determine whether or not to use electronic health records (EHR) or medical claims data (information submitted to insurers to receive payments for treatments and other interventions) when making regulatory decisions on the effectiveness or safety of a drug. This guidance extensively covers topics such as:
Selection of data sources that appropriately address the study question and sufficiently characterize study populations, exposure(s), outcome(s) of interest, and key covariates.
Not all EHRs can be used in regulatory decision making; it is important to determine the relevance and reliability of data sources before using them. The steps used in this determination process should be outlined in the study protocol.
Development and validation of definitions for study design elements (e.g., exposures, outcomes, covariates).
While some terms may seem self explanatory, it is important to clearly define what these terms mean in the context of a particular study. Any misunderstandings of these terms may cause future issues for research that references the study.
Data traceability and quality during data accrual, data curation, and incorporation into the final study-specific dataset.
Like all other forms of data that are submitted to the FDA, EHRs are also subject to audits and require a clear pathway from their origin to the results submitted. This should include the who, what, where, and when of the data capture and any manipulations that were made along the way.
The full guidance can be found on the FDA website, and is open for comment at anytime. Those who wish to comment can find the docket here. This guidance also compliments the information found in previous guidances for industry including “Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data”, “Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products”, and “Use of Electronic Health Record Data in Clinical Investigations.”
Many of Clinical Pathways previous blog entries have discussed these complimentary guidances and the use of EHRs:
FDA FINAL Guidance for Electronic Health Record Data Released!
Comment Now! RWE Using Electronic Health Record and Medical Claims Data Draft Guidance
Monitoring Electronic Health Records (EHRs): ALCOA vs. 21 CFR Part 11 Compliance
Clinical Pathways offers GxP auditing services including auditing regulatory submissions. For more information, please visit our website: https://www.clinicalpathwaysresearch.com/gxp-auditing. If your team needs training, you can also see a list of available courses in our catalog!
-The Clinical Pathways Team
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