10/26/2021
Photo by National Cancer Institute on Unsplash
The US Food and Drug Administration (FDA) released a draft guidance on “Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision Making for Drug and Biological Products.” This draft guidance is published in line with the 21st Century Cures Act (Cures Act). Part of the Cures Act is related to the use of real-world evidence (RWE) to help support the approval of a new indication for an approved drug or to support post approval study requirements. According to the FDA, RWE is “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials” and the “clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real word data (RWD).” RWD comes from medical billing claims, electronic health records (EHR), clinical registries, patient-generated data including from in-home use, and other sources. RWE provides valuable information on the safety and efficacy of a drug, biologic, or device when it is used as intended, outside the constraints of a clinical trial.
The draft guidance covers the potential use of EHRs and medical claims in clinical trials to support FDA regulatory decisions. Items discussed are:
Data sources that address the study question and characterize clinical trial populations, exposure(s), outcome(s) of interest, and key covariates.
Development and validation of definitions for study design elements.
Data that maintains an audit trail and quality during all stages of involvement with the data.
The FDA considers key data elements to be exposure, outcomes, and covariates covering an adequate number of patients. A covariate is “a variable that is neither an exposure nor outcome of interest, but is measured to describe a population or because it may be a confounder or effect modifier to account for in study design or analysis.” The FDA considers data to be reliable if it is accurate, complete, traceable, and has an audit trail.
De-identification of data obtained in this manner may be needed. De-identification is defined by the Health Insurance Portability and Accountability Act (HIPAA) as “the process by which personal identifiers are removed from an individual’s health information.” There is a guidance document available on how to de-identify personal health information (PHI) following HIPAA: “Guidance Regarding Methods for De-identification of Protected Health Information in Accordance with the Health Insurance Portability and Accountability Act (HIPAA) Privacy Rule.” For RWE used for regulatory submissions, de-identified data needs to retain the ability to re-identify unique patients in the original source data without losing traceability.
The draft guidance provides extensive recommendations on selection of data to ensure its completeness and accuracy. Using RWE to support applications for drug or biologics can be expected to expedite the data collection process, thus leading to safe and effective products getting to market faster. For more information on the FDA’s framework for RWE, visit our blog “FDA Framework for Use of Real-World Evidence.” Additional guidance on the use of RWE can be expected. If you are preparing a regulatory submission and need assistance with utilizing RWE, Clinical Pathways can provide training as it is important to know how to apply these methods. Contact us at info@clinicalpathwaysresearch.com.
Comment now through Nov 29, 2021 HERE.
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- The Clinical Pathways Team
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