SEPTEMBER 12, 2024
Originally published by our sister publication Specialty Pharmacy Continuum
By Gina Shaw
On July 25, the FDA finalized long-awaited guidance for pharmaceutical companies about using electronic health record and medical claims data in the process of submitting drugs for approval. The final guidance updates a draft version released in September 2021.
“This guidance does not provide recommendations on choice of study design or type of statistical analysis,” the document noted, nor does it
Originally published by our sister publication Specialty Pharmacy Continuum
By Gina Shaw
On July 25, the FDA finalized long-awaited guidance for pharmaceutical companies about using electronic health record and medical claims data in the process of submitting drugs for approval. The final guidance updates a draft version released in September 2021.
“This guidance does not provide recommendations on choice of study design or type of statistical analysis,” the document noted, nor does it endorse any specific 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 studies, according to the document.
Three critical areas of focus for the final guidance are accuracy, completeness and traceability of data, as well as how these should be measured, said Dan Riskin, MD, the CEO of real-world evidence company Verantos and a member of the Department of Health and Human Services Health IT Advisory Committee, who led an FDA demonstration project to support guidance development.
“This is an important statement from the FDA. Much of the direction from global agencies 10 and even five years ago was all around the process of data collection,” Dr. Riskin said. “But when you get into secondary use of data—which is typical in using real-world evidence—someone else has collected it, and you can’t control the process or adjust it to make it capture what you want.”
He continued, “If you have a data set that has been given to you, collected by someone else for some other use, the only way that you can know if it is high enough quality is to measure that quality. That is what the FDA says in the final guidance: ‘We’re not going to only focus on the process, we’re going to focus on measured quality.’ And that is a big, bold statement for the entire industry.”
To assess the quality of EHRs, medical claims and other real-world data, the document recommends:
Protocols submitted to the FDA should identify all data sources proposed for the study, as well as other relevant descriptive information, the guidance states, adding that each data source should be evaluated to determine whether the available information is appropriate for addressing a specific study question.
“Given that existing electronic health care data were not developed for research purposes or to support regulatory submissions to FDA, it is important to understand their potential limitations when they are used for that purpose,” it cautions, citing as one potential limitation the fact that the purpose of medical claims data is to support payment, and thus, may not accurately reflect a disease or its comprehensive management.
Although the document points out that FDA guidance documents do not establish “legally enforceable responsibilities,” Dr. Riskin said it may be difficult for study sponsors to ignore the guidance in applications. “It’s there for a reason,” he said.
Hospital and health-system pharmacies that partner with study sponsors to collect real-world data can expect an increased focus on data quality. “That’s good for everyone,” Dr. Riskin said. “And in the long term, it’s very exciting. We are going through this massive transformation in which, instead of largely relying on randomized trials to provide all the answers, we are starting to learn from everyday care.”
While a clinical trial cannot “tell us exactly what to do with this specific individual with all of these characteristics,” he concluded, “we can certainly have real-world evidence tell us—within the safe and effective options identified through trials—which is best for this patient?”
Dr. Riskin reported no relevant financial disclosures.
