FDA released final guidelines for the use of real-world data for regulatory submissions, laying out recommendations for how drug companies can best leverage electronic health records and medical claims data to support their applications. on the use of electronic health care data in pharmacoepidemiologic safety studies. This is an update on 2013’s Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data guidance, that included recommendations for documenting the design, analysis, and results of pharmacoepidemiologic safety studies to optimize FDA’s review of protocols and study reports that are submitted to FDA.

FDA noted that it is not endorsing any type or source of data, ‘This guidance does not provide recommendations on choice of study design or type of statistical analysis’. In addition, they stated, ‘This guidance is intended to provide sponsors and other interested parties with considerations when proposing to use electronic health records (EHRs) or medical claims data’ in their clinical trials. 

FDA highlighted that there are three key factors that companies should pay particular attention to:

1. Data Source.

Here, FDA noted that drug sponsors should select data sources that are ‘appropriate’ for the specific study question. 

For example, it highlighted:

• Medical claims data help support payments and may not be an accurate reflection of a specific disease or the management it requires.
• EHRs tend to vary between different healthcare centers and data in these systems may also not capture a comprehensive picture of a patient’s condition or management. 

2. Sponsor development and validation of definitions for study elements, such as exposures, outcomes and potential confounders.

Here, FDA noted that these elements should be in service of the study questions and its design and not the other way around. “The study should not be designed to fit a specific data source”, because limitations of these sources could lead to compromised conclusions. 

3. Traceability and quality of data during retrieval, curation and incorporation into final datasets.

The final guidance has set out to complement FDA’s 2013 recommendations. Overall, this additional guidance focusses on how to evaluate the relevance and reliability of real-world data in drug application submissions. The updated document also gives companies a more general view of the use of EHRs and medical claims data in clinical trials. 

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