The regulator on Wednesday provided recommendations to drugmakers for assessing the use of electronic health records and medical claims data to support their applications.
The FDA on Wednesday 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.
“This guidance does not provide recommendations on choice of study design or type of statistical analysis,” the FDA wrote in its guidance document, noting that it is also not endorsing any type or source of data. “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, according to the document.
The FDA named three key factors that companies should pay particular attention to. First is the source of their data. According to the regulator, drug sponsors should select data sources that are “appropriate” for the specific study question. For instance, medical claims data help support payments and may not be an accurate reflection of a specific disease or the management it requires, the agency contends.
At the same time, 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, the FDA warned.
Second, the FDA recommended that sponsors develop and validate their definitions for various study elements, such as exposures, outcomes and potential confounders. 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,” the regulator cautioned, because limitations of these sources could in turn compromise the conclusions.
Finally, drug sponsors should also ensure the traceability and quality of data during retrieval, curation and incorporation into their studies’ final dataset.
Wednesday’s final guidance advances the FDA’s recommendations from May 2013, when the regulator released its Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data.
The final guidance “complements” the FDA’s 2013 recommendations by providing additional guidance 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. However, the regulator’s final guidance is not legally enforceable.
