FDA Guidance on Leveraging EHRs and Claims Data for Drug Regulatory Decisions

The FDA's Guidance document on the use of real-world data (RWD) and real-world evidence (RWE) from electronic health records and medical claims data is essential for regulatory decision-making related to drugs and biological products. It covers key aspects including data sources, study design elements, data quality, and considerations for addressing missing data.

The guidance emphasizes the importance of predefined reporting, data traceability, validation studies, and the need for protocols and final study reports to clearly describe how study elements are derived from the chosen RWD sources. It discusses the appropriateness of data sources such as Electronic Health Records (EHRs) and medical claims, noting potential changes in data over time due to claims adjudication processes.

The document also addresses the impact of healthcare system practices on the generalizability of study findings, highlighting the necessity for comprehensive data capture and continuous follow-up. It acknowledges the limitations of EHRs and medical claims data in capturing certain types of information and recommends describing the likelihood of obtaining supplemental information for the study population.

Additionally, the guidance suggests conducting quantitative bias analyses to evaluate the impact of misclassification of key covariates on study findings. It also outlines the need for careful examination of data quality throughout the data life cycle, including accrual, curation, transformation, and maintaining traceability.

Source: https://www.fda.gov/media/152503/download##~https://www.federalregister.gov/docu

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